Archive for the ‘Gene Therapy Research’ Category

TEL AVIV, Israel, Sept. 10, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT) announced today that the first two patients have been enrolled in the Phase 2 clinical trial of VB-111 in combination with nivolumab (Opdivo), an immune checkpoint inhibitor, for the treatment of metastatic colorectal cancer. The study is being conducted under a Cooperative Research and Development Agreement (CRADA) between the National Cancer Institute (NCI) and VBL.

Colon cancer is one of the most common cancers worldwide, but immune-based approaches in gastrointestinal cancers have unfortunately been largely unsuccessful, said Tim F. Greten, M.D., Deputy Branch Chief & Senior Investigator of the Thoracic and GI Malignancies Branch (TGMB), Co-Director of the NCI Center for Cancer Research (CCR) Liver Cancer Program, and the principal investigator of the study. The reasons for this are unclear, but no doubt relate to the fact that, in advanced disease, GI cancer appears to be less immunogenic. The goal of this Phase 2 study is to investigate whether priming with VB-111 followed by the addition of nivolumab may induce anti-tumor immune response in metastatic colorectal cancer, for which there remains a major unmet need.

We are pleased to see beginning of enrollment in this study, despite the challenges of COVID-19, said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. We look forward to collaborating with the NCI on investigating VB-111 for the potential benefit of patients with colorectal cancer.

For additional information on the study refer to https://clinicaltrials.gov/show/NCT04166383.

For patients interested in enrolling in this clinical study, please contact NCIs toll-free number 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615) and/or the Web site: https://trials.cancer.gov

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications. VBL has developed three platform technologies: a gene-therapy based technology for targeting newly formed blood vessels with focus on cancer, an antibody-based technology targeting MOSPD2 for anti-inflammatory and immuno-oncology applications, and the Lecinoxoids, a family of small-molecules for immune-related indications. VBLs lead oncology product candidate, ofranergene obadenovec (VB-111), is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in a VBL-sponsored all comers Phase 1 trial as well as in three VBL-sponsored tumor-specific Phase 2 studies. Ofranergene obadenovec is currently being studied in a VBL-sponsored Phase 3 potential registration trial for platinum-resistant ovarian cancer.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements include, but are not limited to, statements regarding our programs, including VB-111, including their clinical development, such as the timing of clinical trials and expected announcement of data, therapeutic potential and clinical results, and our financial position and cash runway. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, the impact of the COVID-19 pandemic on our business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines and clinical results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with the U.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year ended December 31, 2019, and subsequent filings with the SEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. VBL Therapeutics undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:

Michael RiceLifeSci Advisorsmrice@lifesciadvisors.com(646) 597-6979

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VBL Therapeutics Announces Enrollment of the First Patients in the Phase 2 Clinical Trial of VB-111 in Metastatic Colorectal Cancer - GlobeNewswire

Future technologies that could transform peoples lives such as high-performance batteries for electric vehicles, advanced medical treatments and robotics will receive a 65 million government cash boost today (Thursday 10 September).

The funding will be available through the governments Industrial Strategy Challenge Fund, which has been extended today to help develop solutions to some of the biggest global challenges, including climate change and tackling diseases such as cancer and Alzheimers.

Of the investment announced today:

nearly 44 million to develop the next generation of high-performance batteries for electric vehicles and wind turbines, which could also be used for new technologies such as electric aeroplanes. The funding will also be used to complete a first-of-its-kind UK Battery Industrialisation Centre, in Coventry, West Midlands, creating 100 high-skilled jobs. Organisations across the automotive, rail and aerospace sectors will have access a unique battery production facility combining manufacturing, experimentation and innovation

15 million to enable universities, research organisations and businesses to build robots to inspect, maintain and repair nuclear power stations, satellites and wind turbines. The robotics will also be used to address new problems resulting from the pandemic, including ones that can operated remotely and make contact-free deliveries or move hospital beds.

6.5 million will be allocated to the Advanced Therapy Treatment Centre network to accelerate patient access to advanced therapies through the development of specialised infrastructure for the delivery of these products in the NHS. These cell and gene based therapies are aimed at the treatment of life-limiting and inherited diseases such as cancer, Duchenne muscular dystrophy or cystic fibrosis.

Science Minister Amanda Solloway said:

We want to build back better by putting the UK at the forefront of new technologies to create high-skilled jobs, increase productivity and grow the economy as we recover from coronavirus.

This new funding will strengthen the UKs global status in a range of areas, including battery technologies for electric vehicles and robotics, helping us develop innovative solutions to some of our biggest global challenges and creating jobs in rewarding careers right across the country.

Challenge Director for The Faraday Battery Challenge Tony Harper said:

In order for batteries to play their full environmental and economic role in achieving Net Zero we need to deploy at scale and build supply chains for todays technology, shift from strong potential to commercial dominance in a new generation of batteries and continue to build world-class scientific capability to sustain us into the future. The announcement today confirms our commitment and determination to build on the hard-won progress the UK has made in the last 3 years on all these fronts and to accelerate progress post COVID-19.

CEO of the Cell and Gene Therapy Catapult Matthew Durdy said:

The ATTC network is a fantastic example of effective government intervention and the international community recognises this as part of the UKs leadership in the field. Bringing together companies, the NHS and regulatory bodies to make the use of cell and gene therapies easier, more cost effective, and more widespread both boosts the industry and brings these life changing medicines to patients who need them.

The fact that 12% of global clinical trials in cell and gene therapy take place in the UK and half of those involve ATTCs is a testament to the success of this highly respected programme.

Andrew Tyrer, Challenge Director for Robotics for a Safer World said:

I am delighted that the government has provided an extra 15 million funding to help academics and businesses bridge the gap to: complete on-going deliverables set against the Robots for a Safer World Challenge, and also; utilise knowledge gained to the benefit of new sectors, ahead of this Autumns spending review.

Established in 2017, the Industrial Strategy Challenge Fund is delivering 2.6 billion of government investment with the aim of funding world-leading research and highly innovative businesses to address the biggest industrial and societal challenges.

Todays announcement furthers the governments commitment through its R&D Roadmap to put the UK at the forefront of transformational technologies and is part of the governments wider commitment to increase R&D investment to 2.4% of GDP by 2027.

Excerpt from:
Government investment to help build robots for nuclear plants and batteries for electric aeroplanes - GOV.UK

Research

The worlds preeminent scientists say a theory from the Broad Institutes Alina Chan is too wild to be believed. But when the theory is about the possibility of COVID being man-made, is this science or censorship?

Illustration by Benjamen Purvis

In January, as she watched the news about a novel virus spreading out of control in China, Alina Chan braced for a shutdown. The molecular biologist at the Broad Institute of Harvard and MIT started stockpiling medicine and supplies. By the time March rolled around and a quarantine seemed imminent, shed bought hundreds of dollars worth of fillets from her favorite fishmonger in Cambridge and packed them into her freezer. Then she began to ramp down her projects in the lab, isolating her experimental cells from their cultures and freezing them in small tubes.

As prepared as she was for the shutdown, though, she found herself unprepared for the frustration of being frozen out of work. She paced the walls of her tiny apartment feeling bored and useless. Chan has been a puzzle demon since childhood, which was precisely what she loved about her workthe chance to solve fiendishly difficult problems about how viruses operate and how, through gene therapy, they could be repurposed to help cure devastating genetic diseases. Staring out her window at the eerily quiet streets of her Inman Square neighborhood, she groaned at the thought that it could be months before she was at it again. Her mind wandered back to 2003, when she was a teenager growing up in Singapore and the first SARS virus, a close relative of this coronavirus, appeared in Asia. It hadnt been anything like this. That one had been relatively easy to corral. How had this virus come out of nowhere and shut down the planet? Why was it so different? she asked herself.

Then it hit her: The worlds greatest puzzle was staring her in the face. Stuck at home, all she had to work with was her brain and her laptop. Maybe they were enough. Chan fired up the kettle for the first of what would become hundreds of cups of tea, stacked four boxes on her kitchen counter to raise her laptop to the proper height, pulled back her long dark hair, and began reading all of the scientific literature she could find on the coronavirus.

It wasnt long before she came across an article about the remarkable stability of the virus, whose genome had barely changed from the earliest human cases, despite trillions of replications. This perplexed Chan. Like many emerging infectious diseases, COVID-19 was thought to be zoonoticit originated in animals, then somehow found its way into people. At the time, the Chinese government and most scientists insisted the jump had happened at Wuhans seafood market, but that didnt make sense to Chan. If the virus had leapt from animals to humans in the market, it should have immediately started evolving to life inside its new human hosts. But it hadnt.

On a hunch, she decided to look at the literature on the 2003 SARS virus, which had jumped from civets to people. Bingo. A few papers mentioned its rapid evolution in its first months of existence. Chan felt the familiar surge of puzzle endorphins. The new virus really wasnt behaving like it should. Chan knew that delving further into this puzzle would require some deep genetic analysis, and she knew just the person for the task. She opened Google Chat and fired off a message to Shing Hei Zhan. He was an old friend from her days at the University of British Columbia and, more important, he was a computational god.

Do you want to partner on a very unusual paper? she wrote.

Sure, he replied.

One thing Chan noticed about the original SARS was that the virus in the first human cases was subtly differenta few dozen letters of genetic codefrom the one in the civets. That meant it had immediately morphed. She asked Zhan to pull up the genomes for the coronaviruses that had been found on surfaces in the Wuhan seafood market. Were they at all different from the earliest documented cases in humans?

Zhan ran the analysis. Nope, they were 100 percent the same. Definitely from humans, not animals. The seafood-market theory, which Chinese health officials and the World Health Organization espoused in the early days of the pandemic, was wrong. Chans puzzle detectors pulsed again. Shing, she messaged Zhan, this paper is going to be insane.

In the coming weeks, as the spring sun chased shadows across her kitchen floor, Chan stood at her counter and pounded out her paper, barely pausing to eat or sleep. It was clear that the first SARS evolved rapidly during its first three months of existence, constantly fine-tuning its ability to infect humans, and settling down only during the later stages of the epidemic. In contrast, the new virus looked a lot more like late-stage SARS. Its almost as if were missing the early phase, Chan marveled to Zhan. Or, as she put it in their paper, as if it was already well adapted for human transmission.

That was a profoundly provocative line. Chan was implying that the virus was already familiar with human physiology when it had its coming-out party in Wuhan in late 2019. If so, there were three possible explanations.

Perhaps it was just staggeringly bad luck: The mutations had all occurred in an earlier host species, and just happened to be the perfect genetic arrangement for an invasion of humanity. But that made no sense. Those mutations would have been disadvantageous in the old host.

Maybe the virus had been circulating undetected in humans for months, working out the kinks, and nobody had noticed. Also unlikely. Chinas health officials would not have missed it, and even if they had, theyd be able to go back now through stored samples to find the trail of earlier versions. And they werent coming up with anything.

That left a third possibility: The missing phase had happened in a lab, where the virus had been trained on human cells. Chan knew this was the third rail of potential explanations. At the time, conspiracy theorists were spinning bioweapon fantasies, and Chan was loath to give them any ammunition. But she also didnt want to play politics by withholding her findings. Chan is in her early thirties, still at the start of her career, and an absolute idealist about the purity of the scientific process. Facts were facts.

Or at least they used to be. Since the start of the pandemic, the Trump administration has been criticized for playing fast and loose with factsdenying, exaggerating, or spinning them to suit the presidents political needs. As a result, many scientists have learned to censor themselves for fear that their words will be misrepresented. Still, Chan thought, if she were to sit on scientific research just to avoid providing ammunition to conspiracy theorists or Trump, would she be any better than them?

Chan knew she had to move forward and make her findings public. In the final draft of her paper, she torpedoed the seafood-market theory, then laid out a case that the virus seemed curiously well adapted to humans. She mentioned all three possible explanations, carefully wording the third to emphasize that if the novel coronavirus did come from a lab, it would have been the result of an accident in the course of legitimate research.

On May 2, Chan uploaded the paper to a site where as-yet-unpublished biology papers known as preprints are shared for open peer review. She tweeted out the news and waited. On May 16, the Daily Mail, a British tabloid, picked up her research. The very next day, Newsweek ran a story with the headline Scientists Shouldnt Rule Out Lab as Source of Coronavirus, New Study Says.

And that, Chan says, is when shit exploded everywhere.

Alina Chan, a molecular biologist at the Broad Institute, says we cant rule out the possibility that the novel coronavirus originated in a labeven though she knows its a politically radioactive thing to say. / Photo by Mona Miri

Chan had come to my attention a week before the Newsweek story was published through her smart and straightforward tweets, which I found refreshing at a time when most scientists were avoiding any serious discussion about the possibility that COVID-19 had escaped from a biolab. Id written a lot about genetic engineering and so-called gain-of-function researchthe fascinating, if scary, line of science in which scientists alter viruses to make them more transmissible or lethal as a way of assessing how close those viruses are to causing pandemics. I also knew that deadly pathogens escape from biolabs with surprising frequency. Most of these accidents end up being harmless, but many researchers have been infected, and people have died as a result.

For years, concerned scientists have warned that this type of pathogen research was going to trigger a pandemic. Foremost among them was Harvard epidemiologist Marc Lipsitch, who founded the Cambridge Working Group in 2014 to lobby against these experiments. In a series of policy papers, op-eds, and scientific forums, he pointed out that accidents involving deadly pathogens occurred more than twice a week in U.S. labs, and estimated that just 10 labs performing gain-of-function research over a 10-year period would run a nearly 20 percent risk of an accidental release. In 2018, he argued that such a release could lead to global spread of a virulent virus, a biosafety incident on a scale never before seen.

Thanks in part to the Cambridge Working Group, the federal government briefly instituted a moratorium on such research. By 2017, however, the ban was lifted and U.S. labs were at it again. Today, in the United States and across the globe, there are dozens of labs conducting experiments on a daily basis with the deadliest known pathogens. One of them is the Wuhan Institute of Virology. For more than a decade, its scientists have been discovering coronaviruses in bats in southern China and bringing them back to their lab in Wuhan. There, they mix genes from different strains of these novel viruses to test their infectivity in human cells and lab animals.

When word spread in January that a novel coronavirus had caused an outbreak in Wuhanwhich is a thousand miles from where the bats that carry this lineage of viruses are naturally foundmany experts were quietly alarmed. There was no proof that the lab was the source of the virus, but the pieces fit.

Despite the evidence, the scientific community quickly dismissed the idea. Peter Daszak, president of EcoHealth Alliance, which has funded the work of the Wuhan Institute of Virology and other labs searching for new viruses, called the notion preposterous, and many other experts echoed that sentiment.

That wasnt necessarily what every scientist thought in private, though. They cant speak directly, one scientist told me confidentially, referring to the virology communitys fear of having their comments sensationalized in todays politically charged environment. Many virologists dont want to be hated by everyone in the field.

There are other potential reasons for the pushback. Theres long been a sense that if the public and politicians really knew about the dangerous pathogen research being conducted in many laboratories, theyd be outraged. Denying the possibility of a catastrophic incident like this, then, could be seen as a form of career preservation. For the substantial subset of virologists who perform gain-of-function research, Richard Ebright, a Rutgers microbiologist and another founding member of the Cambridge Working Group, told me, avoiding restrictions on research funding, avoiding implementation of appropriate biosafety standards, and avoiding implementation of appropriate research oversight are powerful motivators. Antonio Regalado, biomedicine editor of MIT Technology Review, put it more bluntly. If it turned out COVID-19 came from a lab, he tweeted, it would shatter the scientific edifice top to bottom.

Thats a pretty good incentive to simply dismiss the whole hypothesis, but it quickly amounted to a global gaslighting of the mediaand, by proxy, the public. An unhealthy absolutism set in: Either you insisted that any questions about lab involvement were absurd, or you were a tool of the Trump administration and its desperation to blame China for the virus. I was used to social media pundits ignoring inconvenient or politically toxic facts, but Id never expected to see that from some of our best scientists.

Which is why Chan stood out on Twitter, daring to speak truth to power. It is very difficult to do research when one hypothesis has been negatively cast as a conspiracy theory, she wrote. Then she offered some earnest advice to researchers, suggesting that most viral research should be done with neutered viruses that have had their replicating machinery removed in advance, so that even if they escaped confinement, they would be incapable of making copies of themselves. When these precautions are not followed, risk of lab escape is exponentially higher, she explained, adding, I hope the pandemic motivates local ethics and biosafety committees to think carefully about how they can reduce risk. She elaborated on this in another tweet several days later: Id alsopersonallyprefer if high biosafety level labs were not located in the most populous cities on earth.

How Safe Are Bostons Biolabs?

As one of the world centers of biotech, the Hub is peppered with academic and corporate labs doing research on pathogens. Foremost among them is Boston Universitys National Emerging Infectious Diseases Laboratories (NEIDL), the only lab in the city designated as BSL-4 (the highest level of biosafety and the same level as the Wuhan Institute of Virology). It is one of just a dozen or so in the United States equipped to work with live versions of the worlds most dangerous viruses, including Ebola and Marburg. Researchers there began doing so in 2018 after a decade of controversy: Many locals objected to the risks of siting such a facility in the center of a major metropolitan area.

The good news? Before opening, NEIDL undertook one of the most thorough risk assessments in history, learning from the mistakes of other facilities. Even Lynn Klotz, a senior science fellow at the Washington, DCbased Center for Arms Control and Non-Proliferation, who advised local groups that opposed NEIDL, told the medical website Contagion that the lab likely has the best possible security protocols and measures in place.

But the reality, Klotz added, is that most lab accidents are caused by human error, and there is only so much that can be done through good design and protocols to proactively prevent such mistakes. (Or to guard against an intentional release by a disgruntled researcher, as allegedly happened in the anthrax attacks of 2001.) Rutgers molecular biologist Richard Ebright, a longtime critic of potentially dangerous pathogen research, says the risks introduced by NEIDL are not low enough and definitely not worth the negligible benefits.

Still, risk is relative. Klotz has estimated the chance of a pathogen escape from a BSL-4 lab at 0.3 percent per year, and NEIDL is probably significantly safer than the typical BSL-4 lab. And if catching a deadly pathogen is your fear, well, currently you run a good risk of finding one in your own neighborhood. Until that gets cleared up, the citys biolabs are probably among the safer spaces in town.

Chan had started using her Twitter account this intensely only a few days earlier, as a form of outreach for her paper. The social platform has become the way many scientists find out about one anothers work, and studies have shown that attention on Twitter translates to increased citations for a paper in scientific literature. But its a famously raw forum. Many scientists are not prepared for the digital storms that roil the Twitterverse, and they dont handle it well. Chan dreaded it at first, but quickly took to Twitter like a digital native. Having Twitter elevates your work, she says. And I think its really fun to talk to nonscientists about that work.

After reading her tweets, I reviewed her preprint, which I found mind-blowing, and wrote her to say so. She thanked me and joked that she worried it might be career suicide.

It wasnt long before it began to look like she might be right.

Speaking her mind, it turns outeven in the face of censurewas nothing new for Chan, who is Canadian but was raised in Singapore, one of the more repressive regimes on earth. Her parents, both computer science professionals, encouraged free thinking and earnest inquiry in their daughter, but the local school system did not. Instead, it was a pressure-cooker of a system that rewarded students for falling in line, and moved quickly to silence rebels.

That was a bad fit for Chan. You have to bow to teachers, she says. Sometimes teachers from other classes would show up and ask me to bow to them. And I would say, No, youre not my teacher. Back then they believed in corporal punishment. A teacher could just take a big stick and beat you in front of the class. I got whacked so many times.

Still, Chan rebelled in small ways, skipping school and hanging out at the arcade. She also lost interest in her studies. I just really didnt like school. And I didnt like all the extracurriculars they pack you with in Singapore, she says. That changed when a teacher recruited her for math Olympiads, in which teams of students compete to solve devilishly hard arithmetic puzzles. I really loved it, she says. You just sit in a room and think about problems.

Chan might well have pursued a career in math, but then she came up against teams from China in Olympiad competitions. They would just wipe everyone else off the board, she says. They were machines. Theyd been trained in math since they could walk. Theyd hit the buzzer before you could even comprehend the question. I thought, Im not going to survive in this field.

Chan decided to pursue biology instead, studying at the University of British Columbia. I liked viruses from the time I was a teen, she says. I remember the first time I learned about HIV. I thought it was a puzzle and a challenge. That instinct took her to Harvard Medical School as a postdoc, where the puzzle became how to build virus-like biomolecules to accomplish tasks inside cells, and then to Ben Devermans lab at the Broad Institute. When I see an interesting question, I want to spend 100 percent of my time working on it, she says. I get really fixated on answering scientific questions.

Deverman, for his part, says he wasnt actively looking to expand his team when Chan came along, but when opportunities to hire extraordinary people fall in my lap, he takes them. Alina brings a ton of value to the lab, he explains, adding that she has an ability to pivot between different topics and cut to the chase. Nowhere was that more on display than with her coronavirus work, which Deverman was able to closely observe. In fact, Chan ran so many ideas past him that he eventually became a coauthor. She is insightful, determined, and has the rare ability to explain complex scientific findings to other scientists and to the public, he says.

Those skills would prove highly useful when word got out about her coronavirus paper.

If Chan had spent a lifetime learning how to pursue scientific questions, she spent most of the shutdown learning what happens when the answers you come up with are politically radioactive. After the Newsweek story ran, conservative-leaning publications seized on her paper as conclusive evidence that the virus had come from a lab. Everyone focused on the one line, Chan laments. The tabloids just zoomed in on it. Meanwhile, conspiracists took it as hard evidence of their wild theories that there had been an intentional leak.

Chan spent several exhausting days putting out online fires with the many people who had misconstrued her findings. I was so naive, she tells me with a quick, self-deprecating laugh. I just thought, Shouldnt the world be thinking about this fairly? I really have to kick myself now.

Even more troubling, though, were the reactions from other scientists. As soon as her paper got picked up by the media, luminaries in the field sought to censure her. Jonathan Eisen, a well-known professor at UC Davis, criticized the study in Newsweek and on his influential Twitter account, writing, Personally, I do not find the analysis in this new paper remotely convincing. In a long thread, he argued that comparing the new virus to SARS was not enough to show that it was preadapted to humans. He wanted to see comparisons to the initial leap of other viruses from animals to humans.

Moments later, Daszak piled on. The NIH had recently cut its grant to his organization, EcoHealth Alliance, after the Trump administration learned that some of it had gone to fund the Wuhan Institute of Virologys work. Daszak was working hard to get it restored and trying to stamp out any suggestion of a lab connection. He didnt hold back on Chan. This is sloppy research, he tweeted, calling it a poorly designed phylogenetic study with too many inferences and not enough data, riding on a wave of conspiracy to drive a higher impact. Peppering his tweets with exclamation points, he attacked the wording of the paper, arguing that one experiment it cited was impossible, and told Chan she didnt understand her own data. Afterward, a Daszak supporter followed up his thread with a GIF of a mike drop.

It was an old and familiar dynamic: threatened silverback male attempts to bully a junior female member of the tribe. As a postdoc, Chan was in a vulnerable position. The world of science is still a bit medieval in its power structure, with a handful of institutions and individuals deciding who gets published, who gets positions, who gets grants. Theres little room for rebels.

What happened next was neither old nor familiar: Chan didnt back down. Sorry to disrupt mike drop, she tweeted, providing a link to a paper in the prestigious journal Nature that does that exact experiment you thought was impossible. Politely but firmly, she justified each point Daszak had attacked, showing him his mistakes. In the end, Daszak was reduced to arguing that she had used the word isolate incorrectly. In a coup de grce, Chan pointed out that actually the word had come from online data provided by GenBank, the NIHs genetic sequence database. She offered to change it to whatever made sense. At that point, Daszak stopped replying. He insists, however, that Chan is overinterpreting her findings.

With Eisen, Chan readily agreed to test her hypothesis by finding other examples of viruses infecting new hosts. Within days, a perfect opportunity came along when news broke that the coronavirus had jumped from humans to minks at European fur farms. Sure enough, the mink version began to rapidly mutate. You actually see the rapid evolution happening, Chan said. Just in the first few weeks, the changes are quite drastic.

Chan also pointed out to Eisen that the whole goal of a website such as bioRxiv (pronounced bioarchive)where she posted the paperis to elicit feedback that will make papers better before publication. Good point, he replied. Eventually he conceded that there was a lot of interesting analysis in the paper and agreed to work with Chan on the next draft.

The Twitter duels with her powerful colleagues didnt rattle Chan. I thought Jonathan was very reasonable, she says. I really appreciated his expertise, even if he disagreed with me. I like that kind of feedback. It helped to make our paper better.

With Daszak, Chan is more circumspect. Some people have trouble keeping their emotions in check, she says. Whenever I saw his comments, Id just think, Is there something I can learn here? Is there something hes right about that I should be fixing? Ultimately, she decided, there was not.

By late May, both journalists and armchair detectives interested in the mystery of the coronavirus were discovering Chan as a kind of Holmes to our Watson. She crunched information at twice our speed, zeroing in on small details wed overlooked, and became a go-to for anyone looking for spin-free explications of the latest science on COVID-19. It was thrilling to see her reasoning in real time, a reminder of why Ive always loved science, with its pursuit of patterns that sometimes leads to exciting revelations. The website CNET featured her in a story about a league of scientists-turned-detectives who were using genetic sequencing technologies to uncover COVID-19s origins. After it came out, Chan added scientist-turned-detective to her Twitter bio.

Shes lived up to her new nom de tweet. As the search for the source of the virus continued, several scientific teams published papers identifying a closely related coronavirus in pangolinsanteater-like animals that are heavily trafficked in Asia for their meat and scales. The number of different studies made it seem as though this virus was ubiquitous in pangolins. Many scientists eagerly embraced the notion that the animals might have been the intermediate hosts that had passed the novel coronavirus to humans. It fit their preexisting theories about wet markets, and it would have meant no lab had been involved.

As Chan read the pangolin papers, she grew suspicious. The first one was by a team that had analyzed a group of the animals intercepted by anti-smuggling authorities in southern China. They found the closely related virus in a few of them, and published the genomes for that virus. Some of the other papers, though, were strangely ambiguous about where their data was coming from, or how their genomes had been constructed. Had they really taken samples from actual pangolins?

Once again, Chan messaged Shing Hei Zhan. Shing, somethings weird here, she wrote. Zhan pulled up the raw data from the papers and compared the genomes they had published. Individual copies of a virus coming from different animals should have small differences, just as individuals of a species have genetic differences. Yet the genomes in all of the pangolin papers were perfect matchesthe authors were all simply using the first groups data set. Far from being ubiquitous, the virus had been found only in a few pangolins who were held together, and it was unclear where they had caught it. The animals might have even caught it from their own smuggler.

Remarkably, one group of authors in Nature even appeared to use the same genetic sequences from the other paper as if it were confirmation of their own discovery. These sequences appear to be from the same virus (Pangolin-CoV) that we identified in the present study.

Chan called them out on Twitter: Of course its the same Pangolin-CoV, you used the same dataset! For context, she later added, Imagine if clinical trials were playing fast and loose with their patient data; renaming patients, throwing them into different datasets without clarification, possibly even describing the same patient multiple times across different studies unintentionally.

She and Zhan posted a new preprint on bioRxiv dismantling the pangolin papers. Confirmation came in June when the results of a study of hundreds of pangolins in the wildlife trade were announced: Not a single pangolin had any sign of a coronavirus. Chan took a victory lap on Twitter: Supports our hypothesis all this time. The pangolin theory collapsed.

Chan then turned her Holmesian powers on bigger game: Daszak and the Wuhan Institute of Virology. Daszak had been pleading his case everywhere from 60 Minutes to the New York Times and has been successful in rallying sympathy to his cause, even getting 77 Nobel laureates to sign a letter calling for the NIH to restore EcoHealth Alliances funding.

In several long and detailed tweetorials, Chan began to cast a cloud of suspicion on the WIVs work. She pointed out that scientists there had discovered a virus that is more than 96 percent identical to the COVID-19 coronavirus in 2013 in a mineshaft soon after three miners working there had died from a COVID-like illness. The WIV didnt share these findings until 2020, even though the goal of such work, Chan pointed out, was supposedly to identify viruses with the potential to cause human illnesses and warn the world about them.

Even though that virus had killed three miners, Daszak said it wasnt considered a priority to study at the time. We were looking for SARS-related virus, and this one was 20 percent different. We thought it was interesting, but not high risk. So we didnt do anything about it and put it in the freezer, he told a reporter from Wired. It was only in 2020, he maintained, that they started looking into it once they realized its similarity to COVID-19. But Chan pointed to an online database showing that the WIV had been genetically sequencing the mine virus in 2017 and 2018, analyzing it in a way they had done in the past with other viruses in preparation for running experiments with them. Diplomatic yet deadpan, she wrote, I think Daszak was misinformed.

For good measure, almost in passing, Chan pointed out a detail no one else had noticed: COVID-19 contains an uncommon genetic sequence that has been used by genetic engineers in the past to insert genes into coronaviruses without leaving a trace, and it falls at the exact point that would allow experimenters to swap out different genetic parts to change the infectivity. That same sequence can occur naturally in a coronavirus, so this was not irrefutable proof of an unnatural origin, Chan explained, only an observation. Still, it was enough for one Twitter user to muse, If capital punishment were as painful as what Alina Chan is doing to Daszak/WIV regarding their story, it would be illegal.

Daszak says that indeed he had been misinformed and was unaware that that virus found in the mine shaft had been sequenced before 2020. He also says that a great lab, with great scientists, is now being picked apart to search for suspicious behavior to support a preconceived theory. If you believe, deep down, something fishy went on, then what you do is you go through all the evidence and you try to look for things that support that belief, he says, adding, That is not how you find the truth.

Many of the points in Chans tweetorials had also been made by others, but she was the first reputable scientist to put it all together. That same week, Londons Sunday Times and the BBC ran stories following the same trail of breadcrumbs that Chan had laid out to suggest that there had been a coverup at the WIV. The story soon circulated around the world. In the meantime, the WIV has steadfastly denied any viral leak. Lab director Yanyi Wang went on Chinese television and described such charges as pure fabrication, and went on to explain that the bat coronavirus from 2013 was so different than COVID that it could not have evolved into it this quickly and that the lab only sequenced it and didnt obtain a live virus from it.

To this day, there is no definitive evidence as to whether the virus occurred naturally or had its origins in a lab, but the hypothesis that the Wuhan facility was the source is increasingly mainstream and the science behind it can no longer be ignored. And Chan is largely to thank for that.

In late spring, Chan walked through the tall glass doors of the Broad Institute for the first time in months. As she made her way across the gleaming marble foyer, her sneaker squeaks echoed in the silence. It was like the zombie apocalypse version of the Broad; all the bright lights but none of the people. It felt all the weirder that she was wearing her gym clothes to work.

A few days earlier, the Broad had begun letting researchers back into their labs to restart their projects. All computer work still needed to be done remotely, but bench scientists such as Chan could pop in just long enough to move along their cell cultures, provided they got tested for the virus every four days.

In her lab, Chan donned her white lab coat and took inventory, throwing out months of expired reagents and ordering new materials. Then she rescued a few samples from the freezer, took her seat at one of the tissue-culture hoodsstainless steel, air-controlled cabinets in which cell engineers do their workand began reviving some of her old experiments.

She had mixed emotions about being back. It felt good to free her gene-therapy projects from their stasis, and she was even more excited about the new project she and Deverman were working on: an online tool that allows vaccine developers to track changes in the viruss genome by time, location, and other characteristics. It came out of my personal frustration at not being able to get answers fast, she says.

On the other hand, she missed being all-consumed by her detective work. I wanted to stop after the pangolin preprint, she says, but this mystery keeps drawing me back in. So while she waits for her cell cultures to grow, shes been sleuthing on the sideonly this time she has more company: Increasingly, scientists have been quietly contacting her to share their own theories and papers about COVID-19s origins, forming something of a growing underground resistance. Theres a lot of curiosity, she says. People are starting to think more deeply about it. And they have to, she says, if we are going to prevent future outbreaks: Its really important to find out where this came from so it doesnt happen again.

That is what keeps Chan up at nightthe possibility of new outbreaks in humans from the same source. If the virus emerged naturally from a bat cave, there could well be other strains in existence ready to spill over. If they are closely related, whatever vaccines we develop might work on them, too. But that might not be the case with manipulated viruses from a laboratory. Someone could have been sampling viruses from different caves for a decade and just playing mix-and-match in the lab, and those viruses could be so different from one another that none of our vaccines will work on them, she says. Either way, We need to find where this came from, and close it down.

Whatever important information she finds, we can be sure Chan will share it with the world. Far from being shaken by the controversy her paper stirred, she is more committed than ever to holding a line that could all too easily be overrun. Scientists shouldnt be censoring themselves, she says. Were obliged to put all the data out there. We shouldnt be deciding that its better if the public doesnt know about this or that. If we start doing that, we lose credibility, and eventually we lose the publics trust. And thats not good for science. In fact, it would cause an epidemic of doubt, and that wouldnt be good for any of us.

Link:
Was COVID-19 Manmade? Meet the Scientist Behind the Theory - Boston magazine

SOUTH SAN FRANCISCO, Calif., Sept. 10, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (NASDAQ: CBIO) today announced thatNassim Usman, Ph.D., president and chief executive officer and Clinton Musil, chief financial officer ofCatalyst Biosciences, will participate in fireside chats at two investor conferences in September: The Morgan Stanley Global Healthcare Conference and the Cantor Virtual Global Healthcare Conference. Details for each can be found below.

An archived webcast of the presentations will be available for 90 days on theEvents and Presentationspage under the Investors section of the Companys website.

About Catalyst BiosciencesCatalyst is a research and clinical development biopharmaceutical company focused on addressing unmet needs in rare hematologic and complement-mediated disorders. Our protease engineering platform includes two late-stage clinical programs in hemophilia; a research program on engineering of subcutaneous (SQ) complement inhibitors; and a partnered preclinical development program with Biogen for dry age-related macular degeneration (AMD). The product candidates generated by our protease engineering platform have improved functionality and potency that allow for: SQ administration of recombinant coagulation factors and complement inhibitors; low-dose, high activity gene therapy constructs; and less frequently dosed intravitreal therapeutics. Our most advanced product candidate is marzeptacog alfa (activated) (MarzAA), a next-generation SQ FVIIa entering a Phase 3 registration study in late 2020. Our next late-stage product candidate is dalcinonacog alfa (DalcA), a next-generation SQ FIX, which has demonstrated efficacy and safety in a Phase 2b clinical trial in individuals with Hemophilia B. We have a discovery stage Factor IX gene therapy construct - CB 2679d-GT - for Hemophilia B, that has demonstrated superiority compared with the Padua variant in preclinical models. Finally, we have a global license and collaboration agreement with Biogen for the development and commercialization of anti-complement Factor 3 (C3) pegylated CB 2782.

Forward-Looking StatementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential benefits of products based on Catalysts engineered protease platform, plans to enroll the first patients in a Phase 3 registration study of MarzAA in late 2020, the potential for MarzAA and DalcA to effectively and therapeutically treat hemophilia subcutaneously, the superiority of CB 2679d-GT over other gene therapy, and the development of anti-complement Factor 3 (C3) pegylated CB 2782 by Biogen. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that trials and studies may be delayed as a result of the COVID-19 pandemic and other factors, that trials may not have satisfactory outcomes, that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of DalcA or MarzAA, including the generation of neutralizing antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Companys products will be higher than anticipated, including as a result of delays in development and manufacturing resulting from COVID-19 and other factors, the risk that Biogen will terminate Catalysts agreement, competition and other risks described in the Risk Factors section of the Companys quarterly report filed with the Securities and Exchange Commission on August 6, 2020, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

Contact:Ana KaporCatalyst Biosciences, Inc.investors@catbio.com

The rest is here:
Catalyst Biosciences to Participate in Two Upcoming Investor Conferences - GlobeNewswire

New York, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Biologic Therapeutics Market 2020-2024" - https://www.reportlinker.com/p02448159/?utm_source=GNW Our reports on biologic therapeutics market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the strong R&D pipeline, targeted therapeutic mechanism of biologics, and increasing investment in development of biologics. In addition, strong R&D pipeline is anticipated to boost the growth of the market as well. The biologic therapeutics market analysis includes product segment and geographical landscapes

The biologic therapeutics market is segmented as below: By Product Antibody therapeutics Vaccines Cell therapy Gene therapy Other therapies

By Geographic Landscapes North America Europe Asia ROW

This study identifies the introduction of biosimilars as one of the prime reasons driving the biologic therapeutics market growth during the next few years. Also, increasing incidence of chronic diseases and immunological disorders and patent expiry of major biologics will lead to sizable demand in the market.

The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our biologic therapeutics market covers the following areas: Biologic therapeutics market sizing Biologic therapeutics market forecast Biologic therapeutics market industry analysis

Read the full report: https://www.reportlinker.com/p02448159/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Biologic Therapeutics Market is expected to grow by $ 190.94 bn during 2020-2024 progressing at a CAGR of 12% during the forecast period -...

The diabetes drug metforminderived from a lilac plant thats been used medicinally for more than a thousand yearshas been prescribed to hundreds of millions of people worldwide as the frontline treatment for type 2 diabetes. Yet scientists dont fully understand how the drug is so effective at controlling blood glucose.

Now, researchers at the Salk Institute say they have shown the importance of specific enzymes in the body for metformins function. In addition, the new work showed that the same proteins, regulated by metformin, controlled aspects of inflammation in mice, something the drug has not typically been prescribed for.

Apart from clarifying how metformin works, the research AMPK regulation of Raptor and TSC2 mediate Metformin effects on transcriptional control of anabolism and inflammation, which appears in Genes & Developmentreportedly has relevance for many other inflammatory diseases.

Despite being the frontline therapy for type 2 diabetes, the mechanisms of action of the biguanide drug metformin are still being discovered. In particular, the detailed molecular interplays between the AMPK and the mTORC1 pathway in the hepatic benefits of metformin are still ill-defined. Metformin-dependent activation of AMPK classically inhibits mTORC1 via TSC/RHEB. But several lines of evidence suggest additional mechanisms at play in metformin inhibition of mTORC1, write the investigators.

Here we investigated the role of direct AMPK-mediated serinephosphorylation of RAPTOR in a new RaptorAA mouse model, in which AMPK phospho-serine sites Ser722 and Ser792 of RAPTOR were mutated to alanine. Metformin treatment of primary hepatocytes and intact murine liver requires AMPK regulation of both RAPTOR and TSC2 to fully inhibit mTORC1, and this regulation is critical for both the translational and transcriptional response to metformin.

Transcriptionally, AMPK and mTORC1 were both important for regulation of anabolic metabolism and inflammatory programs triggered by metformin treatment. The hepatic transcriptional response in mice on high fat diet treated with metformin was largely ablated by AMPK-deficiency under the conditions examined, indicating the essential role of this kinase and its targets in metformin action in vivo.

These findings let us dig into precisely what metformin is doing at a molecular level, says Reuben Shaw, PhD, a professor in Salks Molecular and Cell Biology Laboratory and the senior author of the new paper. This more granular understanding of the drug is important because there is increasing interest in targeting these pathways for not only diabetes but immune diseases and cancer.

Researchers have known for 20 years that metformin activates a metabolic master switch, a protein called AMPK, which conserves a cells energy under low nutrient conditions, and which is activated naturally in the body following exercise. Twelve years ago, Shaw discovered that in healthy cells, AMPK starts a cascade effect, regulating two proteins called Raptor and TSC2, which results in a block of the central pro-growth protein complex called mTORC1 (mammalian target of rapamycin complex 1).

These findings helped explain the ability of metformin to inhibit the growth of tumor cells, an area of research that began to generate excitement after Shaw and others connected AMPK to a bona fide cancer gene in the early 2000s.

But in the intervening years, many additional proteins and pathways that metformin regulates have been discovered, drawing into question which of the targets of metformin are most important for different beneficial consequences of metformin treatment. Indeed, metformin is currently entering clinical trials in the United States as a general anti-aging treatment because it is effects are so well established from millions of patients and its side effects are minimal.

But whether AMPK or its targets Raptor or TSC2 are important for different effects of metformin remains poorly understood.

In the new work, in mice, Shaw and his colleagues genetically disconnected the master protein, AMPK, from the other proteins, so they could not receive signals from AMPK, but were able to otherwise function normally and receive input from other proteins.

When these mice were put on a high-fat diet triggering diabetes and then treated with metformin, the drug no longer had the same effects on liver cells as it did in normally diabetic animals, suggesting that communication between AMPK and mTORC1 is crucial for metformin to work.

By looking at genes regulated in the liver, the researchers found that when AMPK couldnt communicate with Raptor or TSC2, metformins effect on hundreds of genes was blocked. Some of these genes were related to lipid metabolism, helping explain some of metformins beneficial effects. But surprisingly, many others were linked to inflammation.

Metformin, the genetic data showed, normally turned on anti-inflammatory pathways and these effects required AMPK, TSC2 and Raptor.

We didnt go looking for a role in inflammation, so for it to come up so strongly was surprising, says Salk postdoctoral fellow and first author Jeanine Van Nostrand, PhD.

People suffering from obesity and diabetes often exhibit chronic inflammation, which further leads to additional weight gain and other maladies including heart disease and stroke. Therefore, identifying an important role for metformin and the interrelationship between AMPK and mTORC1 in control of both blood glucose and inflammation reveals how metformin can treat metabolic diseases by multiple means.

Metformin and exercise elicit similar beneficial outcomes, and research has previously shown that AMPK helps mediate some of the positive effects of exercise on the body, so among other questions, Shaw and Van Nostrand are interested in exploring whether Raptor and TSC2 are involved in the many beneficial effects of exercise, as well.

If turning on AMPK and shutting off mTORC1 are responsible for some of the systemic benefits of exercise, that means we might be able to better mimic this with new therapeutics designed to mimic some of those effects, says Shaw, who holds the William R. Brody Chair.

In the meantime, the new data suggest that researchers should study the potential use of metformin in inflammatory diseases, particular those involving liver inflammation. The findings also point toward AMPK, Raptor and TSC2 more broadly as potential targets in inflammatory conditions, suggesting the need for a deeper investigation of metformin, as well as newer AMPK agonists and mTOR inhibitors, the researchers say.

See more here:
Inflammation in the Liver Reversed by Metformin - Genetic Engineering & Biotechnology News

LONDON--(BUSINESS WIRE)--The global biologic therapeutics market size is expected to grow by USD 190.94 billion as per Technavio. This marks a significant market growth compared to the 2019 growth estimates due to the impact of the COVID-19 pandemic in the first half of 2020. Moreover, steady growth is expected to continue throughout the forecast period, and the market is expected to grow at a CAGR of 12%. Request Free Sample Report on COVID-19 Impacts

Read the 120-page report with TOC on "Biologic Therapeutics Market Analysis Report by Product (Antibody therapeutics, Vaccines, Cell therapy, Gene therapy, and Other therapies) and Geography (North America, Europe, Asia, and ROW), and the Segment Forecasts, 2020-2024".

https://www.technavio.com/report/biologic-therapeutics-market-industry-analysis

The market is driven by the strong R&D pipeline. In addition, the introduction of biosimilars is anticipated to boost the growth of the Biologic Therapeutics Market.

The efficiency of biologics in the treatment of severe infections, malignancies, and immunological and hormonal disorders is encouraging manufacturers to invest in R&D for the development of biologics. Monoclonal antibodies constitute one of the fastest growing segments amongst biological therapies. To date, 88 monoclonal antibodies have been approved for different indications. The pipeline of vendors such as AbbVie, Amgen, Johnson & Johnson, and F. Hoffmann-La Roche clearly indicates huge investments in R&D. Apart from manufacturers that are actively involved in research on biologics, many research institutes are also engaged in developing novel biologics through industrial collaborations. Thus, the strong R&D pipeline is expected to drive the biologic therapeutics market growth during the forecast period.

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Major Five Biologic Therapeutics Companies:

AbbVie Inc.

AbbVie Inc. operates in only one business segment that focuses on the discovery, development, manufacture, and commercialization of drugs for the treatment of various indications. The company's key offerings in the market include HUMIRA, CREON, and SURVANTA.

AstraZeneca Plc

AstraZeneca Plc generates revenue by developing and manufacturing pharmaceutical products. The company offers IMFINZI, which is a human immunoglobulin G1 kappa monoclonal antibody that blocks the interaction of PD-L 1 with the PD-1 and CD80 molecules.

Bristol-Myers Squibb Co.

Bristol-Myers Squibb Co. operates in only one segment. This segment is responsible for the discovery, licensing, manufacture, marketing, distribution, and sales of medicines required to cure serious diseases. The company's key offerings include OPDIVO and YERVOY.

F. Hoffmann-La Roche Ltd.

F. Hoffmann-La Roche Ltd. has business operations under two segments: pharmaceuticals and diagnostics. The company offers atezolizumab, a monoclonal antibody, sold under the brand name TECENTRIQ for treating various cancers.

GlaxoSmithKline Plc

GlaxoSmithKline Plc operates its business through three segments: pharmaceuticals, consumer healthcare, and vaccines. The company's key offerings include NUCALA, BENLYSTA, and BEXXAR.

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Link:
Global Biologic Therapeutics Market 2020-2024: COVID-19 Business Continuity Plan | Evolving Opportunities With AbbVie Inc. and AstraZeneca Plc |...

NEW YORK, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (NASDAQ: FBIO) (Fortress), an innovative revenue-generating company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, today announced that Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer, will participate in three virtual investor conferences in September 2020. The company will also host virtual one-on-one meetings during the conferences.

Details of the events are as follows:

Live webcasts of the presentation and fireside chats will be available on the Events page of the News & Media section of Fortresss website: http://www.fortressbiotech.com. Archived replays of the webcasts will be available for approximately 30 days following each presentation and fireside chat.

About Fortress BiotechFortress Biotech, Inc. (Fortress) is an innovative biopharmaceutical company that was recently ranked number 10 in Deloittes 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, based on percentage of fiscal year revenue growth over a three-year period. Fortress is focused on acquiring, developing and commercializing high-potential marketed and development-stage drugs and drug candidates. The company has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and at partners it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is driven by a world-class business development team that is focused on leveraging its significant biopharmaceutical industry expertise to further expand the companys portfolio of product opportunities. Fortress has established partnerships with some of the worlds leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including Alexion Pharmaceuticals, Inc., AstraZeneca, City of Hope, Fred Hutchinson Cancer Research Center, InvaGen Pharmaceuticals Inc. (a subsidiary of Cipla Limited), St. Jude Childrens Research Hospital and Nationwide Childrens Hospital. For more information, visit http://www.fortressbiotech.com.

Forward-Looking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. As used below and throughout this press release, the words we, us and our may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; risks relating to the COVID-19 outbreak and its potential impact on our employees and consultants ability to complete work in a timely manner and on our ability to obtain additional financing on favorable terms or at all; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our Securities and Exchange Commission filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

Company Contacts:Jaclyn Jaffe and William BegienFortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

Originally posted here:
Fortress Biotech to Participate in Three September 2020 Virtual Investor Conferences - GlobeNewswire

New York, Sept. 09, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Precision Medicine Market: Focus on Ecosystem, Technology, Application, Country Data (21 Countries), and Competitive Landscape - Analysis and Forecast, 2020-2030" - https://www.reportlinker.com/p05965013/?utm_source=GNW By Application: Cancer, Infectious Disease, Neurology, Cardiovascular, Endocrinology, Gastroenterology and Other Applications By Region: North America, Europe, Asia-Pacific, Latin America, and Rest-of-the-World

Cross Segmentation

Applied Sciences: By Product, By Technology, By End User, By Region Precision Diagnostics: By Product, By Technology, By End User, By Region Precision Therapeutics: By Product, By Technology, By End User, By Region Digital Health and IT: By Product, By Technology, By End User, By Region

Regional Segmentation

North America: U.S. and Canada Europe: Germany, France, U.K., Italy, Spain, and Rest-of-Europe Asia-Pacific: Japan, China, India, Australia, and Rest-of-Asia-Pacific Latin America: Brazil, Mexico, and Rest-of-Latin America Rest-of-the-World

Growth Drivers

Advancement of Sequencing Technologies Rising Prevalence of Chronic Diseases Growing Demand for Preventive Care Shifting the Significance in Medicine from Reaction to Prevention Reducing Adverse Drug Reactions Through Pharmacogenomics Test Potential to Reduce the Overall Healthcare Cost Across the Globe

Market Challenges

Unified Framework for Data Integration Limited Knowledge about Molecular Mechanism/ Interaction Lack of Robust Reimbursement Landscape Regulatory Hurdles

Market Opportunities

Targeted Gene Therapy Expansion into the Emerging Markets Collaborations and Partnerships Across Value Chain to Accelerate the Market Entry

Key Companies Profiled

Abbott Laboratories, Almac Group Ltd, Amgen Inc., ANGLE plc, Astellas Pharma Inc., Astra Zeneca PLC, ASURAGEN INC., Bio-Rad Laboratories, Inc., bioMrieux SA., Bristol-Myers Squibb Company, Cardiff Oncology, CETICS Healthcare Technologies GmbH, Danaher Corporation, Eli Lilly and Company Limited, Epic Sciences, Inc., F. Hoffmann-La Roche Ltd, GE Corporation, Gilead Sciences, Inc., GlaxoSmithKline Plc, Illumina, Inc., Intomics A/S, Johnson & Johnson Company, Konica Minolta, Inc., Laboratory Corporation of America, MDx Health, Inc., Menarini Silicon Biosystems, Inc., Merck KGaA, Myriad Genetics, Inc., Novartis AG, Oracle Corporation, Partek, Inc., Pfizer, Inc., QIAGEN N.V., Quest Diagnostics Inc, Randox Laboratories Ltd., Sanofi S.A., Sysmex Corporation, Teva Pharmaceuticals Industries Ltd., Thermo Fisher Scientific, Inc.

Key Questions Answered in this Report: What are the estimated and projected numbers for the global precision medicine market for 2020 and 2030? What are the drivers, challenges, and opportunities that are influencing the dynamics of the market? What is the competition layout of the market? What are the parameters on which competition mapping is carried out in the study? Which key development strategies are being followed and implemented by major players to help them sustain in the market? How are different segments of the market expected to perform during the forecast period from 2020 to 2030? The segments included in the comprehensive market study are: o product type o region o technology o application Which leading players are currently dominating the marke,t and what is the expected future scenario? Which companies are anticipated to be highly disruptive in the future, and why? How can the changing dynamics of the market impact the market share of different players operating in the market? What are the strategic recommendations offered in the study?

Market Overview

Precision medicine refers to the medicine developed as per an individuals genetic profile.It provides guidance regarding the prevention, diagnosis, and treatment of diseases.

The segmentation of the population is done depending on the genome structure of the individuals and their compatibility with a specific drug molecule.In the precision medicine market, the application of molecular biology is to study the cause of a patients disease at the molecular level, so that target-based therapies or individualized therapies can be applied to cure the patients health-related problems.

This industry is gaining traction due to the increasing awareness about healthcare among individuals, integration of smart devices such as smartphones and tablets into healthcare, and increasing collaborations and agreements of IT firms with the diagnostics and biopharmaceutical companies for the development of precision diagnostic tools.

The current precision medicine market is mainly dominated by several majors, such as Abbott Laboratories, Almac Group Ltd, Amgen Inc., ANGLE plc, Astellas Pharma Inc, Astra Zeneca PLC, ASURAGEN INC., Bio-Rad Laboratories, Inc., bioMrieux SA., Bristol-Myers Squibb Company, Cardiff Oncology, CETICS Healthcare Technologies GmbH, Danaher Corporation, Eli Lilly and Company Limited, Epic Sciences, Inc., F. Hoffmann-La Roche Ltd, GE Corporation, Gilead Sciences, Inc., GlaxoSmithKline Plc, Illumina, Inc. Intomics A/S, and Johnson & Johnson Company, Konica Minolta, Inc.

Within the research report, the market is segmented on the basis of product type, ecosystem application, and region, which highlight value propositions and business models useful for industry leaders and stakeholders. The research also comprises country-level analysis, go-to-market strategies of leading players, future opportunities, among others, to detail the scope and provide 360-degree coverage of the domain.

Competitive Landscape Major players, such as Abbott Laboratories, Almac Group Ltd, Amgen Inc., ANGLE plc, Astellas Pharma Inc, Astra Zeneca PLC, ASURAGEN INC., Bio-Rad Laboratories, Inc., bioMrieux SA., Bristol-Myers Squibb Company, Cardiff Oncology, CETICS Healthcare Technologies GmbH, Danaher Corporation, Eli Lilly and Company Limited, Epic Sciences, Inc., F. Hoffmann-La Roche Ltd, GE Corporation, Gilead Sciences, Inc., GlaxoSmithKline Plc, Illumina, Inc., Intomics A/S, Johnson & Johnson Company, Konica Minolta, Inc., Laboratory Corporation of America MDx Health, Inc., Menarini Silicon Biosystems, Inc., Merck & Co., Inc., Myriad Genetics, Inc., Novartis AG., Oracle Corporation, Partek, Inc., Pfizer, Inc., QIAGEN N.V., Quest Diagnostics Inc., Randox Laboratories Ltd., Sanofi SA, Sysmex Corporation, Teva Pharmaceuticals Industries Ltd., Thermo Fisher Scientific, Inc. including among others, led the number of key developments witnessed by the market. On the basis of region, North America is expected to retain a leading position throughout the forecast period 2020-2030, followed by Europe. Countries Covered North America U.S. Canada Europe Germany France Spain U.K. Italy Rest-of-Europe Asia-Pacific Japan China India Australia Rest-of-Asia-Pacific (RoAPAC) Latin America Brazil Mexico Rest-of-Latin America (RoLA) Rest-of-the-World Israel Saudi Arabia United Arab Emirates South Africa RussiaRead the full report: https://www.reportlinker.com/p05965013/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Global Precision Medicine Market: Focus on Ecosystem, Technology, Application, Country Data (21 Countries), and Competitive Landscape - Analysis and...

The global Cancer Gene Therapy Market, which is extensively assessed in the report contemplates the best need development angles and how they could affect the market over the figure residency under thought. The experts have taken careful endeavors to thoroughly evaluating every development factor of the market, other than indicating how certain market restrictions could represent a danger to players in the coming years. In addition, the report additionally gives data on top patterns and openings and how players could take advantage of them to take up the difficulties in the Cancer Gene Therapy Market. This could be a helpful rule for players to concrete their situation in the business or make a rebound in the market.

The Leading Market Players Covered in this Report are : Adaptimmune, GlaxoSmithKline, Bluebird bio, Merck, Celgene, Shanghai Sunway Biotech .

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Impact of Covid-19 in Cancer Gene Therapy Market:The utility-owned segment is mainly being driven by increasing financial incentives and regulatory supports from the governments globally. The current utility-owned Cancer Gene Therapy are affected primarily by the COVID-19 pandemic. Most of the projects in China, the US, Germany, and South Korea are delayed, and the companies are facing short-term operational issues due to supply chain constraints and lack of site access due to the COVID-19 outbreak. Asia-Pacific is anticipated to get highly affected by the spread of the COVID-19 due to the effect of the pandemic in China, Japan, and India. China is the epic center of this lethal disease. China is a major country in terms of the chemical industry.

Key Businesses Segmentation of Cancer Gene Therapy MarketOn the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, sales volume, Cancer Gene Therapy market share and growth rate of Cancer Gene Therapy foreach application, including-

On the basis of product,this report displays the sales volume, revenue (Million USD), product price, Cancer Gene Therapy market share and growth rate ofeach type, primarily split into-

Cancer Gene Therapy Market Regional Analysis Includes: Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia) Europe(Turkey, Germany, Russia UK, Italy, France, etc.) North America(the United States, Mexico, and Canada.) South America(Brazil etc.) The Middle East and Africa(GCC Countries and Egypt.)

Key Highlights of the Table of Contents:Cancer Gene Therapy Market Study Coverage:It includes key market segments, key manufacturers covered, the scope of products offered in the years considered, global Cancer Gene Therapy market and study objectives. Additionally, it touches the segmentation study provided in the report on the basis of the type of product and applications.Cancer Gene Therapy Market Executive summary:This section emphasizes the key studies, market growth rate, competitive landscape, market drivers, trends, and issues in addition to the macroscopic indicators.Cancer Gene Therapy Market Production by Region:The report delivers data related to import and export, revenue, production, and key players of all regional markets studied are covered in this section.Cancer Gene Therapy Market Profile of Manufacturers:Analysis of each market player profiled is detailed in this section. This segment also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

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Key Questions Answered: To gain insightful analyses of the Cancer Gene Therapy market and have a comprehensive understanding of the global market and its commercial landscape. Assess the production processes, major issues, and solutions to mitigate the development risk. To understand the most affecting driving and restraining forces in the market and its impact on the global Cancer Gene Therapy market. Learn about the Cancer Gene Therapy market strategies that are being adopted by leading respective organizations. To understand the future outlook and prospects for the market. Besides the standard structure reports, we also provide custom research according to specific requirements.

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Cancer Gene Therapy Market Is Expected to Foresee an Outstanding Growth by 2020-2021 - Owned

MarketResearch.Bizhas added the most recent research reportWorldwide Gene Therapy Market, this report assists with breaking down top makers, districts, and in like manner covers Industry deals channel, wholesalers, brokers, vendors, Research Findings, and Conclusion.

Global Gene Therapy Market report goals to convey a 360-degree perspective available as far as front line innovation, drivers, limitations, and up and coming patterns with sway examination of these patterns available during the estimate time frame. Further, the Gene Therapy Market report additionally shields vital Chaptericipants profiling with SWOT examination, key advancements of items/administrations from the previous five years.

>Company Profiles <

Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

|Click To get SAMPLE PDF of Gene Therapy Market (Including Full TOC, Table & Figures)

Main Points Covered in the Report:

The total market measurements both as far as revenue and volume.

Complete examination about the Gene Therapy market elements, which incorporates the development factors, restrictions, difficulties, and openings.

Detailed data about the key business players, their essential skills, and the Gene Therapy market share.

Strengths of the purchasers and providers which will help customers for improving their decision-making skills.

The most recent 2020 version of this report reserves all provides to give further remarks on the most recent situations, downturn, and effect of COVID-19 on the whole business. It additionally gives subjective data on when the business can reevaluate the objectives the business is taking to address the circumstance and potential activities.

Note In request to give more precise market conjecture, every one of our reports will be refreshed before conveyance by considering the effect of COVID-19.

Connect with our Analyst to understand the CORONA Virus/COVID-19 impact and be smart in redefining Business Strategies @https://marketresearch.biz/report/gene-therapy-market/covid-19-impact

Gene Therapy Market Segmentation Outlook:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Regions Covered in the Global Gene Therapy Market:

The Middle East and Africa (GCC Countries and Egypt)

North America (the United States, Mexico, and Canada)

South America (Brazil etc.)

Europe (Turkey, Germany, Russia UK, Italy, France, etc.)

Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

Key Questions Answered

What is the size and CAGR of the worldwide Gene Therapy market?

Which are the main portions of the worldwide Gene Therapy market?

What are the key driving elements of the most productive regional market?

What is the nature of competition in the worldwide Gene Therapy market?

How will the worldwide Gene Therapy market advance in the coming years?

What are the fundamental systems received in the worldwide Gene Therapy market?

The study analyses the accompanying key business aspects:

Analysis on Strategies of Leading Players:Market players can utilize this investigation to increase upper hand over their rivals in the Gene Therapy and Equipment market.

Study on Key Market Trends:This segment of the report offers a more profound investigation of the most recent and future patterns of the Gene Therapy and Equipment market.

Market Forecasts:Buyers of the report will approach exact and approved evaluations of the absolute market size as far as worth and volume. The report likewise gives utilization, creation, deals, and different gauges for the Gene Therapy and Equipment market.

Regional Growth Analysis:All significant areas and nations have been canvassed in the report. The local examination will help market players to take advantage of unexplored local markets, get ready explicit procedures for target districts, and think about the development of every single provincial market.

Segmental Analysis:The report gives exact and solid figures of the piece of the pie of significant sections of the Gene Therapy and Equipment market. Market members can utilize this examination to make key interests in key development pockets of the Gene Therapy and Equipment market.

Major Points From TOC:

Chapter One:Overview of Gene Therapy

Chapter Two:Global Gene Therapy Competition Exploration by Top Players

Chapter Three:Top Players Profiles

Chapter Four:Gene Therapy Market Size by Type and Application

Chapter Five:United States Gene Therapy Development Status and Outlook

Chapter Six:EU Gene Therapy Market Development Status and Outlook

Chapter Seven:Japan Gene Therapy Market Status and Outlook

Chapter Eight:China Gene Therapy Market Development Status and Outlook

Chapter Nine:India Gene Therapy Market Outlook

Chapter Ten:Southeast Asia Gene Therapy Market viewpoint

Chapter Eleven:Market Forecast by Type and Application

Chapter Twelve:Gene Therapy Industry Dynamics

Chapter Thirteen:Market Effect Factors Analysis

Chapter Fourteen:Conclusion

....For Detailed InformationClick Here For Complete TOC

A Brief Of The Market Division:

According to the product type, the Gene Therapy market is ordered into In-house, Outsource, Hotels and Small Caterers. In addition, the market share overall industry of each and every item alongside the anticipated valuation is referenced in the report.

Realities identified with the items business value, development rate over the timespan, just as income is available in the report.

Discussing applications, the Gene Therapy market is partitioned into Economy Class, Business Class and First Class. The market share of each product application in tandem with the revenue that every single application may register is present in the report.

Factors And Difficulties Depicted In The Report:

Data about the drivers influencing the commercialization size of the Gene Therapy market just as their effect on the income chart of this vertical is available in the report.

Most recent patterns driving the Gene Therapy market alongside the difficulties in the industry is included in the report.

Marketing Strategies In The Report:

A few strategies that are actualized by the investors with respect to the item showcasing is given in the report.

According to the report, brief with respect to the business channels picked by the organizations are available in the report.

Sellers of these items couple with the brief of clients for the equivalent is referenced in the report.

Investigation Of The Competitors In The Business:

A diagram of the producers presents in the Gene Therapy market containing with as far as possible just as deals zone is associated with the report.

Subtleties of each competitor comprising of organization profile just as their scope of products depicted is initiated in the report.

Information identified with the product deals, income age, value models just as gross edges is portrayed in the report.

The report also speaks about several other information such as evaluation of the competitive landscape, information identified with the market concentration rate and concentration ratio in the upcoming years.

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SAN DIEGO and BOSTON, Sept. 02, 2020 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, Sorrento) and SmartPharm Therapeutics, Inc. (SmartPharm) announced today that Sorrento has completed the acquisition of SmartPharm, a gene-encoded protein therapeutics company developing non-viral DNA and RNA gene delivery platforms for COVID-19 and rare diseases with broad potential application in enhancing any antibody-centric therapeutics.

The platform in synergy with Sorrentos industry-leading fully human G-MAB antibody library has the potential to be the engine for the next-generation, cost-effective in vivo production of antibody therapeutics in patients. By encoding the antibody sequence into a plasmid, a single injection into someones muscle could potentially lead the person to make their own antibodies in vivo for months, instead of relying on repeat administrations of an externally manufactured antibody.

We are very encouraged by the preclinical data generated thus far by our STI-2020dna plasmid candidate against COVID-19, said Henry Ji, Ph.D., CEO of Sorrento Therapeutics. But beyond STI-2020dna the integration of the plasmid DNA technology with our existing antibody products has the potential to make antibody therapy much more accessible and affordable for patients, and is applicable to a multitude of indications ranging from cancer to infectious diseases.

The current SmartPharm R&D and senior management team will remain in place and is expected to integrate into the Sorrento research, development, and corporate infrastructure.

The merger was completed on September 1, 2020 and at such time, SmartPharm became a wholly owned subsidiary of Sorrento. The total value of the consideration payable to the holders of capital stock of SmartPharm in the merger was $19.4 million, subject to certain adjustments for net working capital, indebtedness, transaction expenses and cash. Upon completion of the merger, SmartPharm stockholders became entitled to receive an aggregate of approximately 1.76 million shares of Sorrento common stock based on a price per share equal to $10.60.

About SmartPharm Therapeutics

SmartPharm Therapeutics, Inc. is a privately held, development stage biopharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of serious or rare diseases with the vision of creating Biologics from Within. SmartPharm is currently developing a novel pipeline of non-viral, gene-encoded proteins for the treatment of conditions that require biologic therapy such as enzyme replacement and tissue restoration. SmartPharm commenced operations in 2018 and is headquartered in Cambridge, MA, USA. For more information, please visit http://www.smartpharmtx.com.

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to turn malignant cancers into manageable and possibly curable diseases. Sorrento's multimodal, multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies ("G-MAB library"), clinical stage immuno-cellular therapies ("CAR-T", "DAR-T"), antibody-drug conjugates ("ADCs"), and clinical stage oncolytic virus ("Seprehvir"). Sorrento is also developing potential coronavirus antiviral therapies and vaccines, including COVIDTRAP, ACE-MAB, COVI-MAB, COVI-GUARD, COVI-SHIELD and T-VIVA-19; and diagnostic test solutions, including COVI-TRACK and COVI-TRACE. Sorrento's commitment to life-enhancing therapies for patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule, resiniferatoxin ("RTX"), and ZTlido (lidocaine topical system) 1.8% for the treatment of post-herpetic neuralgia. RTX is completing a phase IB trial for intractable pain associated with cancer and a phase 1B trial in osteoarthritis patients. ZTlido was approved by the FDA on February 28, 2018.

For more information, visit http://www.sorrentotherapeutics.com

Forward-Looking Statements

This press release and any statements made for and during any presentation or meeting contain forward-looking statements related toSorrento Therapeutics, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995 and subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the potential effects that the acquisition of SmartPharm may have on Sorrentos business and product candidate pipeline; the data read-outs related to ongoing studies for COVID-19 using antibodies and gene-encoded antibodies; the potency and potential therapeutic capabilities of gene-encoded antibodies and STI-2020dna, and their respective impact on SARS-CoV-2; the expected length of any therapeutic benefit or antiviral protection provided by gene-encoded antibodies and STI-2020dna; the potential administration and applications for a range of disease indications of gene-encoded antibodies and STI-2020dna, alone or in combination; the status of preclinical testing for STI-1499 and STI-2020dna; the therapeutic potential of gene-encoded antibodies, and STI-2020dna for SARS-CoV-2 and COVID-19; the potential costs and cost-effectiveness associated with STI-2020dna and other DNA plasmids; Sorrentos ability to produce antibody candidates against pathogens and cancer cells; Sorrentos ability to transition from product development to full scale manufacturing and commercialization; Sorrentos ability to advance SmartPharms non-viral gene therapy technology and its gene-encoded platform technology; Sorrentos ability to combine SmartPharms technology with Sorrentos technology and manufacturing capabilities; and Sorrentos potential position in the biopharmaceutical industry. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements, include, but are not limited to: risks related to Sorrento's technologies and prospects with newly acquired technologies, including the acquisition of SmartPharm and the utilization of SmartPharms Gene-Encoded Therapeutics (GET) platforms for the treatment and prevention of coronavirus infections and other pathogens and cancer cells; risks related to seeking regulatory approvals and conducting clinical trials; the clinical and commercial success of the treatment and prevention of coronavirus infections using gene-encoded antibodies; the viability and success of using gene-encoded antibodies for treatments in anti-viral therapeutic areas, including coronavirus; clinical development risks, including risks in the progress, timing, cost and results of clinical trials and product development programs; risk of difficulties or delays in obtaining regulatory approvals; risks that prior study and trial results may not be replicated in future studies and trials; risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval; risks related to seeking regulatory approvals and conducting clinical trials; risks of manufacturing drug product; risks related to leveraging the expertise of its employees, subsidiaries, affiliates and partners to assist the company in the execution of its strategies; risks related to the global impact of COVID-19 and other risks that are described in Sorrento's most recent periodic reports filed with theSecurities and Exchange Commission, including Sorrento's Annual Report on Form 10-K for the year endedDecember 31, 2019, and subsequent Quarterly Reports on Form 10-Q filed with theSecurities and Exchange Commission, including the risk factors set forth in those filings. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law.

Media and Investor Relations

Alexis Nahama, DVM (SVP Corporate Development)Telephone: 1.858.203.4120Email: mediarelations@sorrentotherapeutics.com

Sorrento and the Sorrento logo are registered trademarks of Sorrento Therapeutics, Inc.G-MAB, COVI-GUARD, COVI-SHIELD, COVIDTRAP, T-VIVA-19, COVI-MAB, ACE-MAB, COVI-TRACK, and COVI-TRACE are trademarks of Sorrento Therapeutics, Inc.

ZTlido is a trademark owned by Scilex Pharmaceuticals Inc.All other trademarks are the property of their respective owners. 2020 Sorrento Therapeutics, Inc. All Rights Reserved.

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Sorrento Announces the Closing of Its Acquisition of SmartPharm to Build Next Generation G-MAB-Encoded Plasmid DNA For Cost-Efficient and In Vivo...

Shareholders and others will have the opportunity to engage with CEOs from AIM ImmunoTech, Heat Biologics, Avalon GloboCare, Oregenisis, and ImmunoPrecise Antibodies

As the coronavirus (COVID-19) pandemic persists and could possibly strengthen again this fall, dozens of biopharmaceutical companies worldwide are racing to develop life-saving therapies and vaccines.

Five of those companies will participate in the virtual One2One Investor Forum entitled New Approaches to COVID-19: Hidden Breakthroughs to be held on September 10 at 1pm ET.

The virtual panel discussion will include Tom Equels, CEO of AIM ImmunoTech Inc (NYSEAMERICAN:AIM); Jeff Wolf, CEO of Inc (); Dr David Jin, CEO of (); Vered Caplan, CEO of Oregenisis Inc (); and Dr Jennifer Bath, CEO of () (OTCQB:IPATF).

The virtual One2One Investor Forum will be moderated by broadcast journalist Christine Corrado of Proactive Investors. The event, co-sponsored by investorrelations firm Crescendo Communications, is an online variant of the popular conference format Proactive has run for more than a decade and enables communication between investors and management from some of the world's most cutting-edge businesses.

is a clinical-stage biopharmaceutical company focused on developing first-in-class therapies to modulate the immune system, including multiple oncology product candidates and a novel COVID-19 vaccine.

The North Carolina-based company heads into the forumjust after releasing some promising preclinical results from a study of a potential vaccine based on its already-developed gp96 platform which is designed to activate immune responses against cancer or pathogenic antigens. The company also just won a second US patent protection for the platform.

Heat said the study found the vaccine significantly increases the frequency of systemic and tissue-specific CD8+ T-cells by conferring cellular immunity thats essential against any viral infection, including SARS-CoV-2, the virus that causes the COVID disease.

Also, the company said the vaccine elicits a robust immune response directed against the Spike protein of SARS-CoV-2, generating both helper CD4+ T-cells that aid in antibody production as well as virus killing cytotoxic CD8+ T cells.

The company said the generation of a durable cellular immune response driven by memory CD8+ T cells also may protect against re-infection. Heat's vaccine is designed to prophylactically "train" the immune system to induce this response in patients, especially in individuals most prone to severe infection such as the elderly, those with comorbidities, or others who are immuno-suppressed.

"We believe that these results are a powerful reinforcement that this platform represents a unique and relevant paradigm for novel vaccine development, capable of inducing cellular immune responses in epithelial tissues such as the lungs, said CEO Wolf in a recent statement

This publication demonstrates the potential utility and versatility of our vaccine platform to address SARS-CoV-2, relevant mutations and other pathogens of interest."

Florida-based AIM ImmunoTech focuseson the research and development of therapeutics to treat immune disorders, viral diseases, and multiple types of cancers.

But it also has developed the drug Ampligen, which the company said decreasedthe infectious viral yield of SARS-CoV-2 by 90% in a recent in vitro model.

"We are pleased with these results, as they establish Ampligen's bio-activity against SARS-CoV-2 as well as support our decision to test Ampligen in humans as an intranasal prophylaxis and early-onset therapy against COVID-19," said CEO Equels in a recent statement.

In conjunction with the Institute for Antiviral Research at Utah State University, the company tested Ampligen and found that a dramatic reduction in viral yield could be achieved at clinically achievable dosage levels. These results support Ampligens goal of developing an intranasal prophylactic approach using Ampligen to prevent the coronavirus. Ampligen has already been tested on the SARS-CoV-1 (SARS) virus, showing antiviral promise.

Also, the company recently signed a trilateral material transfer and research agreement with Japan's National Institute of Infectious Diseases and a Japanese pharmaceutical company to test Ampligen as a potential vaccine adjuvant for COVID-19. Ampligen has been shipped to Japan, to be followed up with an additional shipment.

Furthermore, the company has plans for a Phase 1/2a trial of Ampligen with Roswell Park Comprehensive Cancer Center to commence patient enrollment soon. The trial will examine Ampligen in combination with interferon alfa-2b in cancer patients with COVID-19.

AIM also has the financial runway to advance the development of Ampligen, which is also being studied as an immuno-modulator to treat melanoma, colorectal and ovarian cancers. It amassed $40.3 million as of June 30, nearly 5 times more than the $8.8 million it had at the end of 2019.

Avalon GloboCare, which specializes in developing cell-based technologies to combat cancer, has been marshaling its resources to help fight the pandemic on three fronts: marketing a test kit for a partner company, developing therapeutics to fight cytokine storms (bodys immune attacks its own cells), and creating a vaccine.

The company also has established the Avalon Combat Covid-19 Taskforce to accelerate scientific/clinical development to help combat the Covid-19 pandemic through a strategic combination of therapeutic and vaccine approaches.

Last month, the New Jersey-based company won distribution rights to market Cellex Incs coronavirus antibody-based rapid test. Cellex recently earned Emergency Use Authorization from the US Food and Drug Administration to build and sell the kits. Both are working as well to source manufacturing facilities and to develop next-generation COVID-19 detection kits.

For COVID therapeutics, Avalon is working with the Massachusetts Institute of Technologyto co-develop a therapeutic platform against the cytokine storm, which causes lung damage. Cytokines are small protein molecules in the body required to regulate and maintain proper physiological functions, but when released in excess, the cytokine stormdamages vital tissues and organs.

Avalons AVA-Trap therapeutic program is currently entering animal model testing followed by expedited clinical studies with the goal of providing an effective therapeutic option to combat COVID-19 and other life-threatening conditions involving cytokine storms.

In early September, the company launched its new allogeneic mesenchymal stromal cell (MSC) therapeutic platform as a potential therapy for COVID-19 and bone marrow transplant-related complications of acute graft versus host disease (aGVHD).

Avalon also recently formed a partnership with the University of Natural Resources and Life Sciences (BOKU) in Vienna to co-develop a surface-layer vaccine for SARS-CoV-2.

The vaccine is expected to both decrease the severity of a SARS-CoV-2 infection preventing the more severe respiratory inflammation and organ damage and building immunity against the virus. Avalon plans to complete the proof-of-concept pre-clinical studies in 2020, followed by a first-in-human clinical study of this candidate vaccine during 2021.

Inc (), which develops cell and gene therapy platforms, is collaborating with Holdings Inc () to develop and potentially obtain FDA marketing approval of Ranpirnase to treat patients suffering from severe acute respiratory syndrome.

Maryland-based recently acquired the assets of Tamir Biotechnology Inc, including Ranpirnase, a broad spectrum anti-viral agent. Ranpirnase catalyzes the degradation of RNA, and mediates several essential biological activities, including the regulation of cell proliferation, maturation, differentiation, and cell death.

Over 1,000 patients have been dosed with Ranpirnase in previous cancer/mesothelioma clinical trials.Ranpirnase has already demonstrated a strong safety and tolerability profile.Ranpirnase also has shown preclinical antiviral activity in serious viral diseases, such as cytomegalovirus (CMV), influenza, HIV, Ebola, and SARS.

has conducted in vitro studies of Ranpirnase at the University of Tennessee Health Sciences Center Regional Biocontainment Laboratory and George Mason University National Center for Biodefense and Infectious Diseases against the SARS CoV-2 virus. The in vitro studies demonstrated that Ranpirnase was significantly effective in killing the virus with an eight-fold average decrease in the number of plaque-forming units when cultures with Ranpirnase were compared to the controls treated with a placebo.

Based on these initial pre-clinical results, a pre-IND meeting request to fast track Ranpirnase for the treatment of SARS-COV-2 has been submitted by to the FDA.

"We believe this platform could address a significant need in the market and we look forward to leveraging expertise in the development and clinical trial oversight, as well as relationships with government agencies, to possibly accelerate the development of Ranpirnase and its use as a potential treatment of patients with SARS-COV-2,said CEO Caplan recently.

() (OTCQB:IPATF) has developed a broad portfolio of therapeutic antibodies that bind to multiple regions of the SARS-CoV-2 spike protein.

In July, the Vancouver company announced that its DeepDisplay phage technology confirmedthat a subset of six of these antibodies that were analyzed in combinationresultedin near-complete neutralization of SARS-CoV-2 pseudovirus infection.

As we characterize IPAs approximately seventy-five functional lead candidates it is clear that our extensive program has generated therapeutics with broadly diverse profiles, providing substantial opportunities for multi-epitope targeted strategies, said CEOBath in a recent statement.

After selecting approximately sixteen hundred antibodies with the most desirable binding profiles to various spike protein formats, we down-selected our leads based on very specific criteria, all of which are key in our ongoing determination of ideal candidates.

The company continues to further analyze multiplesubsets of antibodies, including neutralizing antibodies demonstrated to bind to the S1 and S2 domains of the SARS-CoV-2 spike protein. Furthermore, the company has begun steps for the expression of the lead antibodies for pre-clinical and clinical manufacturing.

For ImmunoPrecise, PolyTope is the name of its scientifically rigorous and comprehensive approach employed in its anti-COVID-19 programs, as company scientists lead extensive efforts toward the development of effective coronavirus preventions and therapies.

The company noted that its lead antibodies against COVID-19 (or SARS-CoV-2) have not yet been studied in human clinical trials and, as a result, ImmunoPrecise is not making any express or implied claims that its products have been proven to have the ability to eliminate, cure or contain both the virus and disease at this time.

Contact the author: patrick@proactiveinvestors.com

Follow him on Twitter @PatrickMGraham

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Biopharmaceuticals battling COVID-19 pandemic to connect with investors via Proactive One2One Investor Forum on September 10 - Proactive Investors USA...

Bleeding disorders usually include symptoms in which delay in the blood clotting process can be observed. Two main types of blood disorders include von Willebrand disease and hemophilia, both of which are hereditary in nature. Von Willbrand is one of the most common congenital bleeding disorders in humans, caused as a result of deficiency of Von Willebrand factor (VWF) which assists in clot clotting process.

There are three types of Von Willebrand disorders classified on the basis of level of VWF synthesis. Von Willebrand disorders occurrence is almost equal in both sexes, and affects about 1% of the general population according to the Centers for Disease Control and Prevention (CDC). The most common complications of the disorder include heavy menstrual bleeding, excessive gum bleeding and abnormal bleeding after minor injuries. Hemophilia, on the other hand, is a disorder in which clotting factors essential for clotting phenomenon are inadequate or absent. In the U.S. about 400 newborns suffer from hemophilia every year according to statistics published by the CDC. The two main types of hemophilia are hemophilia A with deficiency of clotting factor VIII and hemophilia B with deficiency of clotting factor IX. The National Heart, Lung and Blood Institute, U.S. details that approximately 7 out of 10 individuals who suffer from hemophilia A have severe form of the disorder.

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Global Bleeding Disorder Therapeutics market Witness Most Promising Rise in Demand

Geographically, global Bleeding Disorder Therapeuticsmarket is segmented into North America, Europe, Asia Pacific and Rest of the world (RoW) regions. Currently, North America is leading the global Bleeding Disorder Therapeutics(CSCs) market and is followed by Europe. Factors such as highly developed research infrastructure, well defined regulatory norms, availability of research funds, availability of skilled research and healthcare professionals and supportive economy are driving the North American Bleeding Disorder Therapeuticsmarket towards growth. Asia Pacific is lucrative market for cancer stem cells. Governments in the Asia Pacific countries mainly, India and China are taking initiative to boost the healthcare and biotechnology industry in the respective countries and thus, research and development activities in these countries are swiftly increasing.

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The study offers an in-depth assessment of various customers journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

Key Players of Bleeding Disorder Therapeutics Market Report:

Companies are also trying to discover gene therapy which can cure the disorder. Some of the players conducting lineal trials are Biogen Idec, Novo Nordisk, Bayer Healthcare, Xenetic Biosciences, Cangene Corporation, CSL Behring, Alnylam and Pfizer. The global bleeding disorder therapeutics market is highly competitive and fragmented. Technological advances will expand the boundaries of the market by adding new products. In 2014, Baxter International, Inc. agreed to acquire Chatham Therapeutics. The acquisition will give Baxter Chathams developmental gene therapy program which is directed toward of treatment of hemophilia.

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Bleeding Disorder Therapeutics Market: Report Analysis Global Market Revenue and Share by Manufacturers Boosting the Healthcare Industry Worldwide -...

Investment Thesis

Relay Therapeutics share price performance since IPO

(Source: TradingView)

Investors looking to place a bet on the next big biotech trend may want to take a close look at Relay Therapeutics (RLAY), a novel drug discovery startup with a strong management team, access to one of the world's most powerful supercomputers, and nearly $1 billion in funding, having completed the third-largest biotech IPO of all time in mid-July.

Relay's upsized IPO raised $400 million after the company sold 20 million shares for $20 - above the marketed range of $18-19 - to complement the $520 million it had already raised across 3 private funding rounds from backers including the SoftBank Vision Fund, Google Ventures, the Biotechnology Value Fund, and Third Rock Ventures. Shares traded up by 75% on the first day of trading, closing at $44, but have declined slightly and are currently available at a price of $35.3.

Relay's high-profile backers are supporting a brains trust comprising computational chemists, biophysicists, data scientists and drug development experts with access to the Anton 2 supercomputer developed by D.E. Shaw Research, whose founder, David E. Shaw, is also one of Relay's founders, alongside Matthew Jacobson, a faculty member in the Department of Pharmaceutical Chemistry at the University of California, Dorothee Kern, Professor of Biochemistry at Brandeis University, and Mark Murcko, a Senior Lecturer at MIT and formerly chief technology officer at Vertex Pharmaceuticals (VRTX).

The company has developed a proprietary platform, Dynamo, which it is using to pioneer a novel drug discovery paradigm based on generating insights into protein motion, a concept the company refers to as Motion Based Drug Design ("MBDD"), leveraging techniques including crystallography, cryoEM, molecular simulations and machine learning.

These techniques allow Relay to gain access to harder to reach targets with small molecules, such as the allosteric sites on proteins, which are responsible for regulating many of a protein's activities. The company's initial focus is on oncology, specifically the treatment of solid tumours, although Relay is confident its techniques may be adapted to treat genetic and other diseases.

Relay's pipeline is early-stage, and consists of 3 candidates, designed to inhibit protein tyrosine phosphatase SHP2, tyrosine kinase receptor FGFR2 and cellular signalling pathway regulator PI3K, respectively, with SHP2 inhibitor RLY-1971 currently undergoing a phase 1 dose escalation study.

Although it is hard to assign a target price to such an early-stage biotech, there are reasons to believe that an investment into Relay at this time may result in gains over the long term.

Thanks to advances in technology, allosteric drug discovery looks set to become an increasingly important and potentially lucrative market within the pharma industry.

Traditionally, drug developers target the more accessible active site of proteins, known as the orthosteric site, but most of the "low-hanging fruit" targets have already been claimed, and treatments developed by early-stage biotechs and Big Pharma, meaning drug developers are having to widen their search and look deeper and with more penetration at alternative targets.

For example, in July 2018, biotech Revolution Medicines (RVMD) licensed an allosteric SHP2 inhibitor to Novartis (NVS) for a $50 million upfront fee, plus up to $500 million of milestone payments, and in 2016, Gilead (GILD) paid $400 million up front and agreed to milestone payments worth $800 million to acquire Nimbus Therapeutics' allosteric Acetyl-CoA Carboxylase (ACC) inhibitor program.

Research suggests that the allosteric sites on proteins offer drug developers the chance to identify and develop safer and more effective treatments, but these treatments are also more expensive to develop. What may give Relay a significant head start is its powerful technology (as described in the video below), which can be leveraged to identify targets with a theoretically higher chance of treatment success, and its advantageous funding position.

With one candidate already in the clinic and two set to follow, investors ought to benefit from regular news flow in the shape of data readouts, investigational new drug ("IND") and biologics license applications ("BLAs"), and potentially, lucrative development and commercial agreements with major pharmas, whilst the field's high barriers of entry ought to keep the competition at bay.

In the rest of this article, I will take a closer look at Relay's team, candidates, strategy and addressable market. Backing Relay may require a leap of faith and patience, but the opportunity to acquire shares at a discount to their IPO price may not exist for long, whilst the financial strength of Relay's backers limits the downside case, in my view, although the company's high cash burn of $84 million in FY19 and $26 million in the first 3 months of 2020 should be noted.

Relay Therapeutics is based in Cambridge, Massachusetts, and was spun out of the biotech accelerator Third Rock Ventures in 2015. Third Rock has backed numerous Nasdaq-listed biotechs, such as bluebird bio (BLUE), Editas Medicine (EDIT), Global Blood Therapeutics (GBT), Sage Therapeutics (SAGE) and Constellation Pharmaceuticals (CNST), with decidedly mixed success to date.

Listed Third Rock Ventures seeded companies share price performance over 5-year period

(Source: TradingView)

I would put the disappointments down to the experimental nature of many of the companies. Sage, for example, looked set to commercialise a novel CNS treatment until a high-profile trial failure decimated its share price, while bluebird bio operates in the gene therapy space, which has failed to make the impact to date that many experts felt it would.

It is still early days for all of these companies, however, and we can also see from the chart above that all of these stocks are highly volatile, providing plenty of opportunities for risk-on investors who time the market right to make strong short- to medium-term gains. Relay is likely to reflect the same trend.

Relay has moved quickly to build a strong management team which is led by President and CEO Sanjiv J Patel, who joined from Allergan, where he was Chief Strategy Officer. Don Bergstrom, ex Merck, Sanofi and Mersana Therapeutics, where he specialised in antibody-drug conjugates, leads Relay's R&D, whilst Pat Walters, who spent 20 years at Vertex Pharmaceuticals, heads up Relay's Computation & Informatics division. Relay's General Counsel is Brian Adams, who joined from Keryx Pharmaceuticals.

The remainder of the senior management team have decades of biotech experience and an impressive array of skill sets, however I highlight the 4 staff above, as I see leadership, the drug-development / clinical trial process, computation and legal as perhaps the most important elements of Relay's strategy.

In order to succeed, the company will need to leverage its technology and the skill of its team to identify and purchase optimised compounds, and ensure the clinical trial process runs smoothly and that excellent relationships with the FDA and other authorities are maintained, whilst protecting its IP, particularly as and when drugs begin to approach the commercialisation stage.

Relay Therapeutics Presentation at AWS Events

(Source: YouTube)

The above video provides an insight into how Relay uses its Dynamo technology to search for and purchase the compounds it then uses to drug proteins.

Initially, the company uses a variety of different techniques to try to model the motion of proteins, attempting to create a more rounded and dynamic set of insights into its activity than can be achieved using the more traditional method of static imaging. In a recent interview, Relay founder Dorothee Kern explained:

In textbooks, the drug simply binds the protein, and then, game over, but in reality, binding is a series of conformational changes, with the compound changing the energy landscape of a protein. The idea is that different compounds can cause different changes. One molecule might shift the protein in a direction that shuts down the active site, while another might turn it on.

The company tries to look at both wild-type and mutant forms of the protein and uses, for example, room temperature X-ray crystallography and Cryo-EM, and the Anton Supercomputer to create a "movie" of how the protein might behave over time, based on calculating the forces between millions of atoms over a minute (slices of 2.5 x10-15 seconds). Compared to other drug developers, Relay says in its IPO prospectus that its simulations are based on a time scale that is 100x longer.

A 10 microsecond simulation of a 1 million atom benchmark protein (satellite tobacco mosaic virus), which requires one day of processing on the Anton 2, would require 271 days on conventional hardware (Nvidia V100). Quote from IPO prospectus.

Relay virtual screening process

(Source: Relay Therapeutics presentation at AWS Events)

Having identified a target protein, Relay then screens tens of billions of possible compounds, which are evaluated and scored according to their chances of being successful, based on characteristics such as potency, binding capabilities, bioavailability and selectivity. The company then purchases a selection of the top-ranked compounds from a "synthesis on demand" provider and proceeds to evaluate them in vitro.

Relay's platform compared to conventional drug discovery programs

(Source: IPO prospectus)

Relay has now developed 2 ready-for-the-clinic, plus 1 experimental candidate. Since the company's process is repeatable, its technology ought to improve over time as in vitro studies and human trials progress, which could lead to the development of drug franchises across a number of indications.

This gives Relay different options. It is sufficiently well-funded to progress candidates independently, which would pay off if one of its candidates is approved and starts to generate blockbuster (+$1 billion per annum) sales, but this is a risky and expensive process that could leave the company empty-handed, having burned through hundreds of millions of dollars.

The alternative option is to use the model favoured by many drug developers, which is to secure commercial partnerships with larger pharmas, who fund drug development programmes in part or often in full after a certain stage of progress has been reached, in return for the rights to market and sell the drug, with the drug-developer receiving a percentage - usually in the low double digits - of all sales, as well as milestone payments when certain key development stages are reached, i.e., a pivotal trial, submission of IND, etc.

The downsides here are the smaller revenues the company will generate (~90% less than when going it alone), and when a pharma signs an agreement in principle to partner on, for e.g., 3 candidates, but opts against taking up the option on candidates 2 and 3 when they are developed, which leaves the developer with nothing to show for its early-stage development work.

A good example of these types of companies and arrangements can be found in the field of RNAi-based treatments, for e.g., Dicerna Pharmaceuticals (DRNA), which has co-development agreements in place with Novo Nordisk (NVO), Roche (OTCQX:RHHBY), Eli Lilly (LLY), Boehringer Ingelheim and Alexion (ALXN), or Arrowhead (ARWR), which works alongside Janssen, a subsidiary of Johnson & Johnson (JNJ) and Amgen (AMGN).

Relay current candidates

(Source: Company website)

Relay's current candidates all address solid tumours and inhibit the signalling pathways of specific proteins to prevent the proliferation of cancerous cells, meaning they may be adapted to treat a wide range of cancers, including many of the most prevalent, for e.g., lung, breast, colorectal, liver and kidney.

RLY-1971 is the most advanced candidate, and like the Revolution candidate I mentioned earlier that was acquired by Novartis, it is an allosteric inhibitor of the protein tyrosine phosphatase ("SHP2"), which helps cancerous cells to spread by relaying messages downstream from receptor tyrosine kinases ("RTKs"). To quote founder Kern again:

You can view an allosteric binder as a knob where you can dial in how much activation or inhibition you want. Nature has built these allosteric networks in for regulation. Lets take advantage of them.

RLY-1971 has only made it as far as a phase 1 dose escalation study with advanced or metastatic solid tumors, enrolling 52 participants, with the primary endpoint being to establish a recommended for a phase 2 trial, but the secondary outcome measures - plasma concentration levels, objective response rate ("ORR") and Disease Control Rate ("DCR") - and other outcome measures, including duration of response ("DOR"), progression free survival ("PFS") and tumor mutations, are highly intriguing and offer plenty of share price catalysts if early signs suggest RLY-1971 has an impactful effect on these measures.

SHP2 monotherapy and combination therapy addressable patient populations

(Source: Relay IPO prospectus)

RLY-4008 is an oral, small molecule, selective inhibitor of FGFR2, which is one of four members of the FGFR family. Whilst it's known that targeting FGFR2 in patients with intrahepatic cholangiocarcinoma has demonstrated clinical proof of concept, developers have only been able to produce non-selective, pan-FGFR inhibitors to date, which cause collateral damage by also inhibiting FGFR1, which causes hyperphosphatemia.

Prevalence of hyperphosphatemia in non-selective FGFR inhibitors

(Source: Relay IPO prospectus)

Relay has used its computational models to discover motion-based differences between the two protein types, making it easier to actively target FGFR2 without inhibiting the other family members. This is a great example of how the company's processing power can make the difference when targeting the more complex allosteric site over the orthosteric.

Finally, RLY-PI3K1047 is another signalling pathway regulator, targeting PI3K, which research has indicated may be the most frequently mutated kinase in cancer. Using Dynamo, Relay has been able to model the first full-length protein structure of PI3K, which is important, since prior attempts to inhibit the protein's functions have usually resulted in intolerable levels of toxicity. If the protein can be better understood and mapped, it may unlock the secret to a more effective and safer anti-tumor treatment.

Market value indication of PI3K inhibition

(Source: Relay IPO prospectus)

Relay says it is already leveraging Dynamo to produce 3 more precision oncology programs, and with most of the pieces in place - management, funding, technology and access to funding - required to operate a company successfully, there seems to be little standing in the way of the company rolling out many more candidates over the coming years.

Although there will be volatility in the short term, patience may be the key to holding Relay stock. The company's strategy is predicated upon developing computational analysis of proteins and their functions to new and higher levels of complexity, and generating superior insights, hence it does not want to get pressured into releasing candidates before they are ready purely in order to satisfy the market's demand.

Relay's cash burn - currently at ~$100 million per annum (based on Q120 spend of $26 million) is high and likely to increase substantially as the number of candidates increases and clinical trials begin in earnest. Having listed, fresh funding is likely to come from, and therefore dilute, investors once the current ~$800 million or so of funding is exhausted. This should not happen for at least 18 months at the earliest, however.

It can be very tricky for a layperson investor to get to grips with different drug developers' technologies and decide which are likely to succeed and which aren't, but it is possible (although not always advisable) to look at who else is invested in the company's success.

Relay Chairman and founding investor Alexis Borisy was the founder of Foundation Medicine, acquired by Roche for $5 billion. Another board member, Linda A Hill, is Professor of Business Administration at the Harvard Business School, whilst another, Douglas Ingram, is President and CEO of Sarepta Therapeutics, a commercial-stage biotech that has grown its share price by 344% over the past 5 years.

I would advise against an investment in Relay if you are not won over by the technology side of the business, but despite the fact it is still relatively unproven, and it may be years or even decades away from reaching its peak (based on the ongoing struggles of, for e.g., CAR-T therapy, gene therapy, and RNAi to make a significant impact on the global oncology or other disease markets), it seems a reasonable thesis to me that examining the activities of proteins in more detail can ultimately result in the development of superior treatments.

Since there ought to be no shortage of short-term catalysts as Relay and Dynamo start to reach full-throttle, and based on the overall performance of other Third Rock Venture seeded companies (which suggests a strong culture of good management, oversight and financial planning), I would consider Relay to be a reasonable investment opportunity at a price of $35.3, and as any experienced biotech investor will know, a positive set of data that suggests above standard-of-care efficacy can easily move a stock price by anything from 10% - >100%.

Longer term, Relay appears (in my view) to have an above average chance of graduating to a mid-stage biotech with commercialised products, which gives the current share price plenty of upside potential, in my view.

Gain access to all of the market research and financial analytics used in the preparation of this article plus exclusive content and pharma, healthcare and biotech investment recommendations and research / analytics by subscribing to my channel, Haggerston BioHealth.

Disclosure: I/we have no positions in any stocks mentioned, but may initiate a long position in RLAY over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Excerpt from:
Relay Therapeutics: Heavily Backed Biotech Brains Trust Looks A Long-Term Buy - Seeking Alpha

Manufacturing cell therapies is technically and financially demanding; as a result, despite therapy developers gradually strengthening their in-house expertise, they are also becoming increasingly reliant on contract service providers

Roots Analysis is pleased to announce the publication of its recent study, titled, Cell Therapy Manufacturing Market (3rd Edition), 2019 2030.

The report features an extensive study of the current market landscape and future opportunities associated with cell therapy manufacturing. It focuses on both contract manufacturers, as well as developers with in-house manufacturing facilities, offering in-depth analyses of the various business entities that are engaged in this domain, across different global regions. Amongst other elements, the report includes:

Read Detailed Analysis: https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html

A detailed market forecast, featuring analysis of the current and projected future opportunity across key market segments (listed below)

Type of therapy

Source of cells

Scale of operation

Purpose of manufacturing

Key geographical regions

Key companies covered in the report

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About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

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Cell Therapy Manufacturing Market is estimated to reach close to USD 11 Billion by 2030 - Scientect

The FDAs accelerated approvals program is on shaky ground after a series of surprise rejections, poor communication and COVID-19-focused staff.

This is according to analysis by an anonymous FDA consultant out of regulatory firm Prevision Policy, speaking to analysts at Cowen, who see the recent spate of surprising complete response letters for BioMarin, Intercept, Gilead/Galapagos and Tricida as mounting evidence that the regulator may be backing off accelerated approvals, at least outside of cancer, where speedy reviews are still the norm, and question whether complete response letters are becoming more likely.

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BioMarins rejection for its hemophilia A gene therapy valrox seemed to be a big surprise to the biotech, and it said the FDAs qualms over durability came out of the blue. Having previously agreed with the Agency on the extent of data necessary to support the BLA, the FDA introduced a new recommendation for two years of data from the company's ongoing 270-301 study (phase 3) to provide substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint, the company said in a statement earlier this month.

The Agency first informed the company of this recommendation in the complete response letter having not raised this at any time during development or review.

Some of these issues, the Prevision consultant said in a discussion with Cowen, may be down to the virtual FDA meetings being made amid COVID-19, which is also lending to problems with communication and could go someway to explaining what happened with BioMarins complete response letter, as well as Tricidas.

With all meetings being held virtually, it is understandable that messages are not as clearly communicated as in the past, the consultant said. In fact, Tricidas management suggests that the inability to hold in person meetings played some part in the FDAs issuance of the CRL to [metabolic acidosis drug] veverimer.

There is also an issue with FDA personnel. Accelerated Approval proponent Commissioner Scott Gottlieb left FDA at the start of 2019. His successor, Stephen Hahn, lacked the regulatory experience to engage deeply on issues like AA upon joining the agency, and his agenda has been dominated by the COVID response ever since.

Center for Drug Evaluation & Research Director Janet Woodcock, a highly effective advocate for the accelerated approval pathway, has also been taken out of the direct line of review oversight, moving to a temporary post in the Commissioners office working on COVID response activities.

Its not time to panic, the Prevision consultant said, as: Overall, Prevision thinks that the FDA remains highly functional with a healthy pace of approvals despite the disruptions. However, against that backdrop, the ability of sponsors to use the accelerated approval pathway outside of oncology does bear monitoring.

This comes against the backdrop of the FDA approving 37 new molecular entities so far this year, a strong number for any year, let alone one with a pandemic.

See more here:
FDA commish finding his feet, COVID-19 and chaotic communication hitting speedy approvals - FierceBiotech

Executive Summary

Leading foundations that are seeking breakthroughs in curing diseases requires a specific approach. Often, such organizations are founded and led by patients with their disease or their loved ones. But while passion and urgency are important, they are not enough. They must articulate and live the strategy. And they must stay current with the science.

Nicola Mendelsohn, an accomplished advertising industry executive who leads Facebook in Europe. Lynn OConnor Vos, a pediatric-nurse-turned-communications guru. Michael Milken, a financier with a penchant for philanthropy. What do they have in common? Theyre leading progress towards curing very specific diseases: follicular lymphoma, muscular dystrophy, and prostate cancer.

At the Harvard Business School Kraft Precision Medicine Accelerator, our mission is to identify ways to accelerate cures. Over the past four years, weve studied cure-seeking organizations and identified that a groups success hinges on three critical success factors: the development of a comprehensive strategy, the establishment of the right leader and leadership team, and the deployment of a sustainable funding model.

In a previous HBR piece, we talked strategy, articulating the challenges that cure-seeking organizations face and how to build a plan to overcome those obstacles. But to bring your strategy from ideation into reality, you need the right leader (or leadership team) who can execute the strategy and drive results.

Across the entire nonprofit sector, more than 80% of organizations struggle with leadership. Leading an organization toward breakthroughs and eventually, cures is even more difficult. Often, cure-seeking organizations are founded by the people with the most pressing needs for progress: patients and their loved ones. But while passion and urgency are important, leaders must understand the complex health care system and have strong business acumen in order to accelerate progress. They must also be able to work within an ecosystem that is vast and growing one that includes clinicians, researchers, payers, data scientists, technologists, patient advocates and more.

Thats a lot to ask of one person or a small team, and its why so many organizations grapple with having the right leadership. By observing a broad array of leaders, weve seen where some fall short and have identified three key consistent approaches that successful leaders follow that allow them to accelerate cures.

1. Live the strategy. Leaders must guide the organization in deciding on the strategy, prioritize and resource the strategy, relentlessly communicate the strategy, and live the strategy each day.

Living the strategy means using it as the basis for making decisions and prioritizing key initiatives. Less effective foundation leaders often have difficulty saying no; they can be indecisive, seeking to please all stakeholder and donors. More effective leaders keep their eye on the prize of accelerating cures. They realize that not every good idea fits with the organizations strategy and not every goal can be accomplished simultaneously. Consequently, successful nonprofit leaders determine which programs require immediate focus and are adept at keeping their team concentrated on these goals.

Effective leaders also live the strategy by constantly communicating it internally and externally. Strategic communication begins within the organization, meaning that the leader ensures the entire team understands the strategic goals and timelines for deliverables. All communication is grounded in strategy.

Its also crucial for an organizations leader to communicate the vision and strategy externally whether through meetings, speaking engagements, public relations, or social media. Conferences such as the J.P. Morgan Healthcare Conference and the Milken Institutes Future of Health Summit convene many of the most successful organizations in one place, which provides a platform for leaders to network, share their missions, and lay the groundwork for future partnerships and investments.

2. Surround yourself with experts. Most leaders of cure-seeking foundations realize that their foundation acting alone will not produce a cure. A cure will only be developed by orchestrating a focused ecosystem. Less effective leaders try to have the organization do too much internally, dont engage the right experts or partners, and dont precisely define each players scope of work. In contrast, effective leaders define the experts and partners the organization needs and engage in thorough due diligence and careful contracting to choose the right partners and put proper incentives in place.

In creating the right team and ecosystem, the most successful CEOs build a leadership group of experts with diverse skill sets and backgrounds. This includes medical, data and technology, fundraising, and marketing and communication experts. But since hiring the right talent can be challenging for revenue-constrained organizations, leaders must find creative ways to engage outside experts their organizations need. These outside experts include:

A well-designed advisory board. As youd expect, the most effective advisory boards weve observed have members who are medical and scientific experts. But they also include business leaders and technologists who can advise on a broader array of issues such as optimal clinical-trial design, the best ways to enroll patients, and new funding models. People will be motivated to join such boards when they are well run and when all participants are motivated to cure this specific disease.

Consultants. They can be extremely valuable in strategy development and business planning.

External partners for specific tasks. For example, one leading foundation has outsourced work to a partner focused on science and biopsies, another that pulls data from electronic medical records, and yet another with analytics expertise. This organization has also partnered with a firm for phlebotomy to gather blood samples from patients at their homes and a public relations agency to generate attention around new initiatives. Each partner has been chosen for its expertise in specific area and contracts have been negotiated with great attention to detail. While this may sound obvious to business leaders, nonprofit foundations dont always use such processes.

3. Stay current and connected to the relevant science. Leaders of disease-focused organizations must constantly be attuned to the entire cure-seeking ecosystem and landscape and to scientific developments related to their specific disease. With science and technology evolving at an increasingly rapid pace, so, too, are new opportunities for innovations and breakthroughs. Some of these innovations happen outside of your immediate disease space; leaders who have a broad view of the landscape will spot opportunities earlier.

For example, recent advancements in gene therapy are showing enormous potential to change the way we treat diseases such as Duchenne muscular dystrophy, cystic fibrosis, and Huntingtons. To be an early mover, a leader must ensure their foundation stays abreast of such advancements by constantly engaging with other organizations that are working towards cures including academic medical centers, pharmaceutical companies, and technology startups. This will help a leader and his or her foundation assess the potential benefits and risks surrounding such innovations and determine if and when to invest the organizations resources in supporting it.

For traditional nonprofit foundations, where the main activities are to support patients and engage in advocacy, organizations can be run by good managers with operational and executional skills. But foundations that truly aspire to drive cures require skilled strategic leaders. These bold leaders must be able to develop and articulate a clear strategy, must have the discipline to execute the strategy in a world with numerous distractions, and must be able to form and orchestrate an aligned, collaborative ecosystem with the right experts and partners.

With the right strategy and the leadership, disease-focused foundations give themselves the best possible chance to fulfill their mission and develop life-saving cures for patients.

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What It Takes to Lead a Disease Research Foundation - Harvard Business Review

An Overview of the Impact of COVID-19 on the Gene Therapy Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms, and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

Gene Therapy Market report covers a detailed competitive outlook including the market share and company profiles of the key participants operating in the global market. The Gene Therapy Market report provides an in-depth overview of Product Specification, technology, product type, and production analysis considering major factors such as Revenue, Cost and Gross Margin.

Get FREE Sample PDF Including COVID-19 Impact Analysis: https://www.coherentmarketinsights.com/insight/request-pdf/1774

The global Gene Therapy market has defined the present market scenario in an orderly way, emphasizing the industrial development, prominent players engaged in the current Market, chapter wise market specifications, industrial procedures, that will assist our readers to aim towards the Gene Therapy Market industry perspective and promote stability with cost-effectiveness.

The competitive scenarios of the Gene Therapy market provide details by leading manufacturers such as (GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.), including capacity, production, price, revenue, cost, gross, gross margin, growth rate, import, export, market share, and technological developments.

Gene Therapy Market report 2020-2026 focuses on the major drivers and restraints for the key players. Gene Therapy research report also provides granular analysis of the market share, segmentation, revenue forecasts, and geographic regions of the market. The market research report is a professional and in-depth study on the current state of Industry.

Gene Therapy Market Report Scope:

The depth industry chain includes analysis value chain analysis, porter five forces model analysis, and cost structure analysis

The report covers the Global market of Gene Therapy Market. It also describes present situation, historical background, and future forecast

Comprehensive data showing Gene Therapy Market sale, consumption, trade statistics, and prices in the recent years is provided in this Market report

The report indicates a wealth of information on Market

Market forecast for the next five years, including market volumes and prices, is also provided.

Raw Material Supply and Downstream Consumer Information is also included in this Market report.

Following are Main Points Covered in Gene Therapy Market Report:

Introduction and Market Overview

Market Scope and Market Size Estimation

Market Concentration Ratio and Market Maturity Analysis

Global Gene Therapy Value and Growth Rate from 2020-2026 (Market Segmentation, Types of, Applications, Research Regions)

Market Dynamics (Drivers, Limitations, Opportunities)

Industry News and Policies by Regions (Industry News, Industry Policies)

Industry Chain Analysis (Upstream Raw Material Suppliers, Analysis, Major Players of, Major Players Manufacturing Base and Market Share of Gene Therapy in 2020, Major Players Product Types in 2020)

Gene Therapy Manufacturing Cost Structure Analysis (Production Process Analysis, Manufacturing Cost Structure of Gene Therapy, Raw Material Cost of Gene Therapy, Labor Cost of Gene Therapy)

Market Channel Analysis of Gene Therapy

Major Downstream Buyers of Gene Therapy Analysis

The Major TOC Of Gene Therapy Market Includes:

Market overview: Definition, brief introduction of Major Applications

Production Market Analysis: Price, revenues, cost, and gross margin analysis

Major manufactures production and sales: Regional Major Manufacturers Production and Sales Market Comparison Analysis

Sales Market analysis of Gene Therapy Market: By volume, sales revenue and major Manufacturers Performance in past

Consumption Market Analysis: Global Consumption Volume Analysis, Regional Consumption Market Analysis, Regional Market Performance, and Market Share

Major Application Analysis: Down Stream Customers Analysis

New Project Investment Feasibility Analysis: New Project SWOT Analysis and Project Investment Feasibility Analysis.

Gene Therapy Market Report provide business insights and consulting to industries, organizations, or even individuals to make strategic business decisions and achieve sustainable growth in their respective market domain.

Key Topics Covered:

Part 01: Executive SummaryPart 02: Scope of The ReportPart 03: Research MethodologyPart 04: Market Landscape Market Ecosystem Market Characteristics Market Segmentation Analysis

Part 05: Market Sizing Market Definition Market Sizing 2020 Market Size And Forecast 2020-2026

Part 06: Five Forces Analysis Bargaining Power of Buyers Bargaining Power of Suppliers Threat of New Entrants Threat of Substitutes Threat of Rivalry Market Condition

Part 07: Customer Landscape Part 08: Regional Landscape Geographical Segmentation Regional Comparison Americas Market Size And Forecast 2020-2026 EMEA Market Size And Forecast 2020-2026 APAC Market Size And Forecast 2020-2026

Part 09: Decision Framework Part 10: Drivers And Challenges Market Drivers Market Challenges

Part 11: Market Trends Part 12: Vendor Landscape Overview Landscape Disruption Vendors Covered Vendor Classification Market Positioning Of Vendors

About Us:

Coherent Market Insights is a global market intelligence and consulting organization focused on assisting our plethora of clients achieve transformational growth by helping them make critical business decisions. We are headquartered in India, having office at global financial capital in the U.S. and sales consultants in United Kingdom and Japan. Our client base includes players from across various business verticals in over 150 countries worldwide. We pride ourselves in catering to clients across the length and width of the horizon, from Fortune 500 enlisted companies, to not-for-profit organization, and startups looking to establish a foothold in the market. We excel in offering unmatched actionable market intelligence across various industry verticals, including chemicals and materials, healthcare, and food & beverages, consumer goods, packaging, semiconductors, software and services, Telecom, and Automotive. We offer syndicated market intelligence reports, customized research solutions, and consulting services.

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Analysis Covid 19: Gene Therapy Market 2020 Inclinations Top Companies GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc,...

A New Research Published byGMAon theGlobal Gene Therapy for Ovarian Cancer Market (COVID 19 Version)in various regions to produce more than 200+ page reports. This study is a perfect blend of qualitative and quantifiable information highlighting key market developments, industry and competitors challenges in gap analysis and new opportunities and may be trending in theGlobal Gene Therapy for Ovarian Cancer Market. Some are part of the coverage and are the core and emerging players being profiledTakara Bio, VBL Therapeutics, CELSION, Targovax.

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TheGlobal Gene Therapy for Ovarian Cancer Marketstudy offers a comprehensive overview of the current market and forecasts by 2020-2029 to help identify emerging business opportunities on which to capitalize.

TheGlobal Gene Therapy for Ovarian Cancer Marketreport provides an in-depth review of industry dynamics inGene Therapy for Ovarian Cancer, including existing and potential developments to represent prevailing consumer pockets of investment.

The report provides details concerning key drivers, constraints and opportunities and their effect on theGene Therapy for Ovarian Cancerreport.

Industry players strategic analysis and industry position in theGlobal Gene Therapy for Ovarian Cancer Market;

The report elaborates on the SWOT analysis and Porters Five Forces model.

The market-study value chain review gives a good view of the positions of the stakeholders.

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Quantitative data:

Breakdown of market data by main region & application / end-user

By typeIntravenous, Intratumoral, Intraperitoneal, ,

Global Gene Therapy for Ovarian Cancer MarketReport-specific sales and growth rates for applicationsOvarian Cancer (unspecified), Recurrent Ovarian Epithelial Cancer, Platinum-Resistant Ovarian Cancer, , (historical & forecast)

Global Gene Therapy for Ovarian Cancer MarketProfits by sector and growth rate (history and forecast)

Global Gene Therapy for Ovarian Cancer Marketsize and rate of growth, application and type (Past and Projected)

Global Gene Therapy for Ovarian Cancer MarketSales income, volume and growth rate Y-O-Y (base year)

Qualitative data: Includes factors affecting or influencing market dynamics and market growth. To list some names in related sections

Industry overview

GlobalGlobal Gene Therapy for Ovarian Cancer Marketgrowth driver

GlobalGlobal Gene Therapy for Ovarian Cancer Markettrends

Incarceration

Global Gene Therapy for Ovarian Cancer MarketOpportunity

Market entropy ** [specially designed to emphasize market aggressiveness]

Fungal analysis

Porter Five Army Model

Research Methodology:

Primary Research:

We interviewed various key sources of supply and demand in the course of thePrimary Researchto obtain qualitative and quantitative information related to this report. Main sources of supply include key industry members, subject matter experts from key companies, and consultants from many major firms and organizations working on theGlobal Gene Therapy for Ovarian Cancer Market.

Secondary Research:

Secondary Researchwas performed to obtain crucial information about the business supply chain, the company currency system, global corporate pools, and sector segmentation, with the lowest point, regional area, and technology-oriented perspectives. Secondary data were collected and analyzed to reach the total size of the market which the first survey confirmed.

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1) Who are the key Top Key players in the GlobalGlobal Gene Therapy for Ovarian Cancer Market Report?

Following are list of players:Takara Bio, VBL Therapeutics, CELSION, Targovax.

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Geographically, this report is divided into several main regions, consumption, revenue (million USD) andGlobal Gene Therapy for Ovarian Cancer Marketshare and growth rate in these regions, from 2019 to 2029 (predicted), covering North America, Europe, Asia-Pacific, etc.

3) What is the projected market size & market growth rate for the 2019-2029 periodGlobal Gene Therapy for Ovarian Cancer Marketindustry?

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Yes. Additional granularity / market segmentation may be included depending on data availability and difficulty of survey. However, you should investigate and share detailed requirements before final confirmation to the customer.

5) What Is impact of COVID 19 on GlobalGlobal Gene Therapy for Ovarian Cancer Market industry?

Before COVID 19Global Gene Therapy for Ovarian Cancer MarketMarket Size Was XXX Million $ & After COVID 19 Excepted to Grow At a X% & XXX Million $.

TOC for GlobalGlobal Gene Therapy for Ovarian Cancer MarketResearch Report is:

Section 1: Global Market ReviewGlobal Gene Therapy for Ovarian Cancer Market(20132029)

Defining

Description

Classified

Applications

Facts

Chapter 2: Market Competition by Players/Suppliers 2013 and 2019

Manufacturing Cost Structure

Raw Material and Suppliers

Manufacturing Process

Industry Chain Structure

Chapter 3: Sales (Volume) and Revenue (Value) by Region (2013-2019)

Sales

Revenue and market share

Chapter 4, 5 and 6: GlobalGlobal Gene Therapy for Ovarian Cancer Marketby Type, Application & Players/Suppliers Profiles (2013-2019)

Continued..

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Gene Therapy for Ovarian Cancer Market SWOT Analysis including key players Takara Bio, VBL Therapeutics, CELSION, Targovax - The Daily Chronicle

The study, published in The Journal of Cell Biology, was conducted by CNIO researchers in collaboration with researchers from the Complutense University of Madrid and the Autonomous University of Barcelona.

Our results indicate that a new therapy may be developed to prevent the development of pulmonary fibrosis associated with ageing, says CNIOs Maria Blasco, principal investigator of the study

Lung tissue of patients with pulmonary fibrosis does not regenerate because the cells involved in lung generation have damaged telomeres, the ends of the chromosomes. The new study describes a gene therapy that activates the enzyme that repairs telomeres.

Idiopathic pulmonary fibrosis is a potentially lethal disease for which there is currently no cure and that is associated with certain mutations or advanced age. TheTelomeres and Telomerase Groupat the Spanish National Cancer Research Centre (CNIO) had previously developed an effective therapy for mice with fibrosis caused by genetic defects. Now they show thatthe same therapy can successfully be used to treat mice with age-related fibrosis.

With respect to humans, our results indicate that it may be possible todevise a treatment to prevent the development of pulmonary fibrosis associated with ageing, saysMaria Blasco, principal investigator of the study.

The treatment tested in mice is agene therapy that activates the production of telomerase in the body. Telomerase is an enzyme that repairs the telomeres at the end of chromosomes. According to Blasco, this therapy was highly effective in animal models and no side effects were observed.

The CNIO team had already shown in previous studies that thegenetic factors are associated with telomere dysfunction. Telomeres are structures that, like end caps, protect the ends of chromosomes in all cells.

Back in 2015, the team generated an animal model for pulmonary fibrosis a mouse that, among other characteristics, lacked the telomerase gene. In this mouse model, alveolar type II cells or type II pneumocytes important for lung tissue regeneration eventually die as a result of telomere dysfunction. As a consequence, the mice develop aggressive pulmonary fibrosis because the respiratory epithelium cannot renew itself periodically; this periodic regeneration keeps the tissue healthy and free from possible damage caused by harmful airborne substances.

Age-related fibrosis

The mouse model that lacks the telomerase gene faithfully mimics the human disease caused by mutations affecting the telomeres. However, specific mutations are found in relatively few pulmonary fibrosis cases. In the vast majority of patients, nothing points to a specific mutation, but all patients have something in common:an advanced age.

Indeed, telomeres can become defective through the mere process of ageing. The team led by Blanco have done ground-breaking research on telomeres and the ageing process. Telomeres are protein structures that cap the ends of chromosomes; they shorten with every cell division. After many rounds of cell division over the lifetime of an individual, they become so short that they can no longer protect the chromosomes. The cells interpret this as an error and stop dividing so that the tissue cannot regenerate anymore.

In the paper now published, the researchers show that telomere dysfunction associated with ageing occurs in alveolar type II cells, which play a primary role in lung tissue regeneration.The team have thus found the molecular basis of the link between pulmonary fibrosis and ageing, a link that is clearly seen in the clinical setting.

In addition to regenerating tissue, these cells produce and release a lipid-protein complex called pulmonary surfactant that facilitates the mechanical work done by the lungs. Lung tissue must expand when we breathe in, six to ten times per minute, which means a great deal of physical effort. Pulmonary surfactant plays an important role in lubricating lung tissue, retaining its elasticity, and reducing the amount of work required to expand and contract it. If type II pneumocytes fail to regenerate, the surfactant is not produced, which results in lung stiffness and fibrosis, saysJess Prez-Gilof the Complutense University of Madrid, who participated in the study and whose team are experts in this field.

We have observed averyclear relation between telomere status in type II pneumocytes, pulmonary surfactant production and fibrosis development in animals, Prez-Gil adds. Here we address the effects on telomeres at the molecular level, biological and physical changes in cells and tissues, and the consequences for the health of the animal, the whole organism., indicatesSergio Pieiro, first author of the study.

A therapy for all types of fibrosisIn 2018, the CNIO group developed a gene therapy that reversed pulmonary fibrosis in mice lacking the telomerase gene. This therapy was based on activating telomerase expression temporarily. A virus used as a telomerase gene carrier was injected intravenously into the mice. The effect alveolar type II cells with long telomeres was temporary, but lung tissue regeneration was successfully induced.

The same therapy was now used in aging mice. And it worked in them too. The telomerase-activating gene therapy prevented the development of fibrosis in all mice, including the ones without genetic alterations that only underwent physiological ageing, Blasco explains.

This extends the possibility of a cure for pulmonary fibrosis to virtually all cases of fibrosis,as the researchers conclude in their paper: These findings contribute to a better understanding of the importance of [the telomerase gene] as a potential target for future therapeutic approaches in idiopathic pulmonary fibrosis.

The study was funded by the Spanish Ministry of Science, Innovation and Universities, the Carlos III Health Institute, the Community of Madrid, the Botn Foundation and Banco Santander through Santander Universidades, and World Cancer Research.

Development of the initial stages of pulmonary fibrosis associated with ageing in non-treated mice (left), that can be prevented in mice treated with the telomerase gene therapy (right). Credit: CNIO.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Towards a Gene Therapy for Reversal of Aging-Associated Pulmonary Fibrosis - Technology Networks

CMI published a business research report on Hemophilia Gene Therapy Market: Global Industry Analysis, Size, Share, Growth, Trends, and Forecasts 20202026. Hemophilia Gene Therapy Market with 150+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. The information is gathered based on modern floats and requests identified with the administrations and items.

The global Hemophilia Gene Therapy Market analysis further provides pioneering landscape of market along with market augmentation history and key development involved in the industry. The report also features comprehensive research study for high growth potential industries professional survey with market analysis. Hemophilia Gene Therapy Market report helps the companies to understand the market trends and future market prospective,opportunities and articulate the critical business strategies.

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Geographical segmentation of Hemophilia Gene Therapy Market involves the regional outlook which further covers United States, China, Europe, Japan, Southeast Asia and Middle East & Africa. This report categorizes the market based on manufacturers, regions, type and application.

Hemophilia Gene Therapy Market: Competitive Landscape

Leading players operating in the global Hemophilia Gene Therapy Market include:BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics

Scope of the Report

The key features of the Hemophilia Gene Therapy Market report 2020-2026 are the organization, extensive amount of analysis and data from previous and current years as well as forecast data for the next five years. Most of the report is made up from tables, charts and figures that give our clients a clear picture of the Hemophilia Gene Therapy Market. The structure of Hemophilia Gene Therapy Market by identifying its various segments and sub-segments to help understanding the report.

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As the report proceeds further, it covers the analysis of key market participants paired with development plans and policies, production techniques, price structure of the Hemophilia Gene Therapy Market. The report also identifies the other essential elements such as product overview, supply chain relationship, raw material supply and demand statistics, expected developments, profit and consumption ratio.

Important Hemophilia Gene Therapy Market Data Available In This Report:

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An Overview of the Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

National governments and international bodies are focused on adopting collaborative efforts to encourage financial institutions to meet the financial needs of customers and members affected by the coronavirus. However, there are some sectors that have remained unscathed from the impact of the pandemic and there are some that are hit the hardest.

We, at Coherent Market Insights, understand the economic impact on various sectors and markets. Using our holistic market research methodology, we are focused on aiding your business sustain and grow during COVID-19 pandemics. With deep expertise across various industries-no matter how large or small- and with a team of highly experienced and dedicated analysts, Coherent Market Insights will offer you an impact analysis of coronavirus outbreak across industries to help you prepare for the future.

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Hemophilia Gene Therapy Market: Analysis of Rising Business Opportunities with Prominent Investment Ratio by 2026 - Bulletin Line

Jeff Galvin believes his Rockville cell and gene technology company has found a breakthrough treatment for people living with HIV

By David Goldstein

| Published: 2020-08-17 08:31

When Jeff Galvin was 13 years old, he came across a lone computer in the basement of Muzzey Junior High School in Lexington, Massachusetts. It was actually a teletype machine attached to a minicomputer. This was, after all, the 1970s.

He doesnt remember why it was there, but he got permission to use it and taught himself how to program. My first love affair, Galvin recalls. My head exploded with the possibility that you had this thing that never got tired. You just fed it electricity.

At 15, he was taking classes at Massachusetts Institute of Technology (MIT) in nearby Cambridge and teaching computer science to high school and college students on weekends. This may be starting to sound like Good Will Hunting, the 1997 film in which actor Matt Damon plays an MIT janitor who secretly solves complex equations on a classroom blackboard, but theres a difference. He was a math genius, Galvin says. I was a highly passionate, excited kid who had just seen the most amazing toy in history.

In his 20s, Galvin was an early recruit to Silicon Valley in those heady nascent days of the 1980s when it was fast becoming the high-tech Xanadu. His time in California included six years at Apple, but it was a visit to the National Institutes of Health in 2007 that took his career in a much different direction.

Now 61, hes the founder and CEO of a Rockville cell and gene technology company, and he believes his company stands on the cusp of a medical breakthrough. Galvins team of molecular biologists at American Gene Technologies (AGT) thinks it has developed a gene therapy procedure that can cure HIV, the virus that leads to AIDS. According to the U.S. Department of Health and Human Services, 1.1 million people are currently living with HIV in this country; the World Health Organization estimates that 75 million people across the globe have been infected with the virus since the AIDS epidemic began in 1981. About 32 million have died.

Galvin says he made HIV his target because a cure for the virus has remained elusive. People with HIV must take a daily regimen of medications that control infections and suppress the virus, but dont eliminate it. Over time, these drugs can cause everything from nausea and fatigue to more serious conditions affecting the kidneys, heart and central nervous system, according to NIH.

He believes the gene therapy procedure AGT has developed, in which genes are transferred to modify cells or tissue through the use of viral vectors, could actually be a platform to cure any number of diseases.

What cant we cure with this? Galvin says during a call to the head of a financial securities firm one afternoon in February. Hes seated at a large oval table in the AGT conference room with the phone on speaker.

Galvin spends much of his time wooing potential investors. What he brings to the task is his salesmans personality: a bit over the top, a healthy dollop of bravado, and an inclination to push the envelope. And his companys HIV treatment is getting a serious lookthe U.S. Food and Drug Administration is determining whether the procedure is safe enough for human testing.

Galvin says the gene and cell therapy industry has been exploding in recent years, causing a slowdown in the regulatory process for those kinds of treatments. But AGT is hopeful that the FDA will approve the human clinical trials by this fall. I cant imagine we cant cure almost everything in the world, he says on the call. Were going to send chemotherapy and radiation the way of bloodletting and leeches.

Galvin is a ball of energy and nonstop talker. Politics. Facebooks troubled relationship with privacy. Hell opine as long as someone will listen and he doesnt have a pressing appointment. He is 6 feet tall, has a genial smile and an eagerness to engage. And he burns with the passion of the committed.

Were down here in Rockville, Maryland. Lots of good opportunities down here, he says during the pitch, which goes on for more than an hour. Right now [we have] the beginnings of what is turning into a revolution in pharmaceuticalswere talking about the creation of probably another $3 to $5 trillion industry over the next 10 to 15 years. I think this is bigger than the dot-com boom. I think it doesnt have quite the same bubble. This isnt the kind of thing that will all go off the cliff simultaneously like dot-com because theres real science behind it. And if it works out, it can be monetized.

Galvin is a cheerleader for Montgomery Countys growth as a hub for health technology. AGT is just off Interstate 270 at Exit 6B in the life sciences corridor, where it percolates among a cluster of tech, biotech and pharmaceutical companies, as well as educational centers. Branches of the University of Maryland and Montgomery College help incubate new startups. Thats the atmosphere that got AGT off the ground in 2008.

In addition to HIV, AGT is developing therapies for several types of cancerous tumors that affect the breasts, lungs and prostate. The company hopes that its work on a treatment for liver cancer will be approved for human clinical trials in 2022. The lab is also working on a gene therapy for phenylketonuria, known as PKU, a rare inherited metabolic disorder.

Theres this great economic engine which is evolving that will make Maryland the next Silicon Valley, Galvin tells the head of the financial securities firm. I call it DNA Valley.

Galvin, it should be noted, is not a scientist. Hes an economist by training, a 1981 graduate of Harvard University. Hes a computer prodigy by pedigreehis mother was one of the rare female computer software experts in the 1960s, and his father is an MIT-trained electrical engineer who did national security work.

Galvin has this thing about disruptive technologies, he says, systems that upend the old way of doing something and change the culture in a significant way. Like how Apple co-founder Steve Jobs simplified and popularized the computer mouse. Or how GPS changed the way we get from here to there. Thats how he sees AGT and gene therapy. Ive been through a lot of technologies: computers, software, the internet, apps, IT, Galvin says.

So I understand how these technologies grow. Gene and cell therapies are bigger than any of those, and its much more emotional because its your health.

He has a fluency in arcane subjects that arent connected to his own skills. This is Galvins explanation for how HIV infects a cell through a protein known as CCR5, located on the surface of white blood cells, and why AGT believes it has developed a defense: One element of our cell product is the removal of CCR5 from the surface of CD4+ T cells. Howeverwe have also added siRNAs against conserved regions of the vif and tat HIV genes for additional protection against R5 viruses as well as extending protection to CXCR4 versions of HIV.

Heres how he puts it in plain English: We have removed thedoor handle (CCR5) that HIV uses to get into cells. Most forms of HIV use that common surface protein in cells to infect the cell, but some forms do not need that handle. AGT has added specially designed genes to our HIV treatment that are capable of producing substances inside the cell that protect against several known mutations of HIV that do not require that handle. AGT is the first company to provide this type of broad protection to the various known versions of HIV.

HIV is an insidious virus that infects a patients T cells, a type of white blood cell that helps the body fight off infection. In Montgomery County, 3,489 people were infected with HIV between 2009 and 2018, with 123 new cases in 2018, according to the Maryland Department of Health. AGTs gene therapy approach modifies the HIV-specific T cells so they can resist infections and do their job of protecting the body from pathogens that cause disease.

AGT does this by using viral vectors; the viruses are cracked open to remove the bad genes and replaced with newly modified genes that will improve the cell. Instead of a virus with the intention of infecting you and going to the next person, its been tamed to do only one part of that process, says C. David Pauza, a molecular biologist and longtime researcher in gene cloning and HIV who serves as AGTs chief science officer. We put things in it we want it to deliver and it makes one infection and stopsand doesnt go any farther. According to Galvin, once HIV T cells are able to carry out their protective work as intended, HIV patients would eventually become permanently immune to the virus and hopefully have no need to continue taking antiretroviral drugs.

AGTs concept is not new, according to Carl Dieffenbach, director of the Division of AIDS at NIHs National Institute of Allergy and Infectious Diseases. What has improved are the vectors, he says. Its reasonable to continue to watch this. Whats unknown, Dieffenbach says, is the human reaction. Once the therapy is tried on people, what you dont know is how this will actually behave.

Tami Howie, an attorney who represents tech and biotech companies, first met Galvin in 2017, when she was CEO of the Maryland Technology Council. He is one of the rare people who epitomizes the convergence in tech and biotech, she says. Hes totally cutting edge.

But messing around with the human genome can be fraught with risk. We know there are viruses out there that get into your body and activate genes that do all kinds of good things and bad things, Pauza says. [One] good thing is a very old virus thats in most of us that controls the efficiency of pregnancy in women. But then you can get other viruses that go in and they stimulate things to be made inappropriately and trigger horrible results.

To Galvin, no risk, no reward. When he made public late last year that AGT was developing a cure for HIV, he was criticized by some in the HIV/AIDS community for peddling false hope. But Galvin has no regrets and is sympathetic to their concerns. Its reasonable to experience a gut reaction to the word cure when humanity has been struggling against HIV for decades, he says. I dont fault anyone for working to protect their community. We are always clear that we will only know for sure once we prove it in a human trial, he says. He calls AGTs work the future of medicine.

Its going to be typical that many of the diseases that strike you are going to be cured by gene and cell therapy, he tells the prospective investor. And we plan to lead that revolution becausewere going to prove were the most efficient competitor in it by curing HIV this year.

If hes right, that would be seismic.

Enormous, Pauza says.

The oldest of three children, Galvin didnt get a lot of attention when he was young so he tended to get into a lot of trouble, he says. I figured out how things worked early, so it was hard to lock me in the house.

As a child, hed come home with a bloody hand, having found a razor blade, or wander into a snowstorm looking for twigs for the fireplace. His father, Aaron, says Galvin was a handful because his mind was so active. We were on a first-name basis with all the emergency wards, he says with a chuckle.

Galvin showed his entrepreneurial spirit early on, his father says. When he was 6 and the familys house in Lexington was under construction, the boy collected dirt from the excavation site and sifted it through a window screen and into plastic bags. Then he pulled his little red wagon up and down the street, selling topsoil for 50 cents a bag.

Harvard didnt offer a degree in computer science when he went there, so Galvin majored in economics and took all the computer classes he could. After graduation, he left for California to take a job with Hewlett-Packard, but he didnt like the corporate culture and went to work for Apple. Apple was more like me, Galvin says. It was in love with what computers could do.

He stayed on the West Coast for parts of the next three decades. Silicon Valley was on fire when I was there, says Galvin, whose sister, Laurie, and brother, Mark, also pursued careers in the tech world. He describes the atmosphere there as a group of people with the right ideas andpushing things at light speed. There was such a clarity of vision and purpose. There was no limit on what we could do there.

It was a fast life, though. As a marketing manager and later director of international marketing for Claris, an Apple spinoff, Galvin was always on the move: Europe, the Middle East, Indonesia, New Zealand. While in Paris on a trip to show clients how to service Apple hardware, he fell asleep at the wheel while driving through a tunnel at 70 mph after having been up for three days. When he brushed a curb, he awoke and was able to right the car. But it was a chilling experience.

How lucky can you get? Galvin says.

In 2001, after nearly two decades of 16-hour workdays, he decided he was done. At 42, hed made investments in startups, real estate, software and internet companies. I looked at my bank account and realized I didnt have to work anymore, Galvin says.

He bought a house on Maui and traveled between there and his home in San Carlos in Silicon Valley. He started dating, thinking he should settle down before he got too old. He and his wife, Cherry, married in 2004.

The couple enjoyed a carefree life in the tropics. But after five years, Galvin was bored out of my mind, so they moved back to California. He decided to tiptoe back into the game by looking for a project where he could become an angel investor, someone who puts money behind a startup often in exchange for ownership equity. The word got out, and Galvin received a proposal from a postdoctoral researcher at a lab at NIH headed by Dr. Roscoe Brady, a renowned biochemist and pioneer in the treatment of enzyme deficiencies. Galvin visited the lab in 2007 and met Brady, who explained the science behind viral vectors. It was Galvins eureka moment.

When I learned that there was a mechanism to update the DNA in a human cell, my head practically exploded, Galvin says. He reasoned that if you look at a cell as the human bodys computer, and the DNA contained in the cell as the operating system, you could employ viral vectors to convert viruses into updates for the human computer and thereby correct defects.

DNA is the instruction set for the cell, Galvin says. Your genes are just instructions to make enzymes and proteins that then react in the cell. Basically, your cell is an organic computer. You change the software, you change the cell.

Brady was retiring and NIH was closing his lab. Galvin says NIH gave him the intellectual property, free of charge, on the condition that he continue the research. I felt like he was close to making major breakthroughs, Galvin says. So he hired two of Bradys research assistants and signed Brady on as a scientific adviser. Thats how AGT was born.

In the early days, Galvin continued to live on the West Coast and funded the work out of his own pocket. His mother, Frayda, was suffering from Stage 4 pancreatic cancer. She died in 2009 after battling the disease for 15 months. Knowing what she went through bolstered Galvins conviction that AGTs work could help people. In 2010, he realized he needed to be in Rockville full time. Over the next few years, he received $1 million in grants from NIH, which was interested in innovative approaches to gene and cell therapies. Galvin says obtaining funding that way was easier than trying to lure investors, and he kicked in an additional $2 million.

AGT began as a small but determined undertaking. An early supporter was Dr. Robert Redfield, a virologist who now heads the Centers for Disease Control and Prevention and became visible during the White House coronavirus briefings in the spring. He served as an adviser to AGT beginning in 2011 and subsequently chaired the companys clinical advisory board for its HIV gene therapy program until 2018. By that time, AGT had been granted patents for its HIV therapy and had enlisted NIH as a research partner in its search for a cure. Fast forward to today, Galvin says, we have curative therapies for HIV.

Galvins day usually begins in my sweats, he says, in the kitchen of his Rockville condo where he has a three-screen computer. He calls it command central. He can work uninterrupted for about three hours, and hes usually at AGT by noon. He presides over a three-story warren of offices where employees handle regulatory affairs, marketing, finance and other administrative business.

Its in the labs, spread over 11,500 square feet on the third floor, where the microbiologists and other scientists do the research and delicate work of separating genes, the link between one generation and the next. Behind glass walls and clad in blue protective gowns and gloves, they employ an array of biological tools as they work with T cells, viral vectors and other microscopic particles. Their findings spill out of white data machines spaced at regular intervals along the work counters.

Talking up AGT requires Galvin to travel a lot. Before the coronavirus pandemic, he was often on trains to Manhattan for meetings and dinners with prospective investors, and flying around the country for conferences and other events. But the world has new rules these days. Video conference calls and Zoom meetings have become the new way of doing business.

To relax, Galvin plays Xbox or watches The Simpsons. He enjoys how the shows writers parody American culture.

I can tell these people feel the same way about the world as I do, he says. He also reads or catches up on the news. On his nightstand in February was Sapiens: A Brief History of Humankind, a New York Times bestseller.

His wife, Cherry, prefers warm weather, so she spends her winters at the couples home in Silicon Valley. They got married late in life, Galvin says, and dont have children. Cherrys sister, a single mother, lived with the couple and in 2004 sent for her daughter, who was 6 years old and staying with her grandmother in Wuhan, China. Now 21, Galvins niece, Jesse, recently graduated from the University of Washington, where she majored in molecular and cellular biology. Interning at AGT during her high school summers might have had something to do with that. I love my niece like a daughter, Galvin says.

Galvin says hes having the most fun hes ever had. Still, securing funding for new therapies and medicines can be a struggle if youre not a pharmaceutical giant. Investors generally want a quick return, and human trials for a new drug or therapy can be costly.

To be a successful entrepreneur and forging new territory, you get a lot of arrows in the back. A lot of people say no way, says Drew Palin, a physician and chief innovation officer of Intellivisit, a Madison, Wisconsin, online medical diagnosis company. Hes also one of Galvins investors. He has had to raise lots of nickels and dimes that allowed him to be a little more patient and persistent. To do that, you have to have a lot [of] drive, a lot of personal passion and a way to survive.

When Galvin isnt pitching AGTs upside to venture capitalists, blue-chip finance houses like JPMorgan Chase, big financial institutions like Citibank, and angel investors like Palin, hes championing the promise of genetic engineering.

Im basically an evangelical person that is spending his entire day either connecting people with our mission and trying to engage them [in] some way to support it or propel it, or inspiring people to achieve greatness within the mission, Galvin says.

Should AGTs experimental HIV treatment work and eventually be approved for commercial sales, Galvin says it would likely be licensed to a large pharmaceutical company with global reach to ensure the products wide availability.

Galvin doesnt have any idea how much the treatment could cost, but predicted it would be less expensive than what insurance companies now pay to cover the costs of daily antiretroviral treatments, medicine for the side effects and appointments with doctors. Hes not chasing the dollar. Hes chasing every life he can save, Tami Howie says. He figures every minute hes not working, people are dying.

Galvins fundraising has pushed investments to $40 million, and his staff has grown to 30. His commitment to AGT, however, put a strain on both his marriage and retirement nest egg. Both have survived and recovered, he says, but it took sustained engagement with high risk and giant potential downfall for me.

Now he weighs the possibility that it all could pay off. If we get a handful of cured HIV patientsI think the [National] Mall is going to look like the days of the AIDS quilt because these people have been suffering for so long, Galvin says, and I think the emotional response is going to be quite profound.

David Goldstein is a former political and investigative reporter in Washington, D.C., for McClatchy Newspapers and The Kansas City Star.

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Rockville cell and gene company may have found treatment for HIV - BethesdaMagazine.com

Global MRNA Vaccines and Therapeutics Market Research Report 2020: COVID-19 Outbreak Impact Analysis

Brand Essence Market Research has developed a concise study on the MRNA Vaccines and Therapeutics market to depict valuable insights related to significant market trends driving the industry. The report features analysis based on key opportunities and challenges confronted by market leaders while highlighting their competitive setting and corporate strategies for the estimated timeline.

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mRNA Vaccines and Therapeutics Market is valued at USD 3.39 Billion in 2018 and expected to reach USD 6.39 Billion By 2025 with the CAGR of 9.5% over the forecast period. Increase in licensing agreements with increased prevalence of chronic disorders is likely to grow the mRNA vaccines and therapeutics market.

mRNA (messenger RNA) is a genetic material which carries the genetic information from DNA to ribosome and where it specifies the sequence of amino acids for the protein synthesis. mRNA vaccines and therapeutics are prepared with the help of advanced technology and offers the most efficient and cost effective options with reduced side effects with increased immunogenicity. The demand for mRNA vaccines & therapeutics has increased due to the increase in various diseases such as cancer, infectious disease such as ebola virus, zika virus and many more.

mRNA Vaccines and Therapeutics Market report is segmented on type, mechanism of action, application and by regional & country level. Based upon type, mRNA vaccines and therapeutics market is segmented into Standardized Therapeutic Cancer mRNA Vaccines, Individualized Therapeutic Cancer mRNA Vaccines, Therapeutic Infectious Disease mRNA Vaccines, and mRNA Therapeutic for Other Diseases. On the basis of mechanism of action, the market is segmented into Bioengineered Vaccine, Gene Therapy (Gene Silencing/Suppression), Gene Transcription (Protein Generation), Cell Therapy, Monoclonal Antibody, and Others. Based upon application, mRNA vaccines and therapeutics market is classified into Infectious Disease, Cancer, and Other.

The regions covered in this mRNA Vaccines and Therapeutics market reports are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, market of mRNA Vaccines and Therapeutics market is sub divided into U.S., Mexico, Canada, UK, France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for mRNA Vaccines and Therapeutics Market Reports Some major key players for mRNA Vaccines and Therapeutics market are Moderna Therapeutics, Translate Bio, BioNTech, Argos Therapeutics, eTheRNA, Sangamo Therapeutics, In-Cell-Art, CureVac, Ethris, and Tiba Biotechnology among others

Increase in licensing agreements with increased prevalence of chronic disorders is the key factor which helps mRNA Vaccines and Therapeutics Market to grow.- Over the period of time there has been tremendous increase in the prevalence of cronic disorders and infectious diseases on a large scale. Furthermore, constant increase in the various infections disease has increased the demand for effective and efficient vaccines and therapeutics for the treatment. There has been significant increase in the licensing agreement between manufacturers for the development of mRNA vaccine and therapeutics. Moreover, increase in approval of from various regulatory bodies such as FDA, CE and others are also expected to drive the growth of mRNA vaccines and therapeutic market. Increased adoption of mRNA vaccines and therapeutics and reduced manufacturing time has also promoted the market growth. The increase in research and development activities coupled with advanced technology is expected to create ample opportunities on a long run. However, high cost involved in the development of mRNA vaccine and therapeutic market is expected to hinder the growth of mRNA vaccines and therapeutics market in the near future.

Geographically, this report split global into several key Regions, revenue (Million USD) The geography (North America, Europe, Asia-Pacific, Latin America and Middle East & Africa) focusing on key countries in each region. It also covers market drivers, restraints, opportunities, challenges, and key issues in Global MRNA Vaccines and Therapeutics Market.

bKey Benefits for MRNA Vaccines and Therapeutics Market Reports Global market report covers in-depth historical and forecast analysis. Global market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level. Global market report helps to identify opportunities in market place. Global market report covers extensive analysis of emerging trends and competitive landscape.mRNA Vaccines and Therapeutics Market Segmentation By Type Standardized Therapeutic Cancer mRNA VaccinesIndividualized Therapeutic Cancer mRNA VaccinesTherapeutic Infectious Disease mRNA VaccinesmRNA Therapeutic for Other Diseases

By Mechanism Of ActionBioengineered VaccineGene Therapy (Gene Silencing/Suppression)Gene Transcription (Protein Generation)Cell TherapyMonoclonal AntibodyOthers

By ApplicationInfectious DiseaseCancerOther

By Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto Africa

North America is expected to dominate the mRNA Vaccines and Therapeutics Market.- North America is expected to account for the major market share over the forecast period owing to the presence of developed economies such as U.S., Canada, and others. Increase adoption of advanced vaccines and therapeutics due to the high disposable income and increased prevalence of chronic and infectious diseases are expected to drive the growth of mRNA vaccines and therapeutics market over the forecast period. Europe is expected to account the second largest market share owing to the increase in clinical trials, research and development activities coupled with advanced technology. Asia Pacific is expected to develop significantly and maintain its dominance over the forecast period due to the presence of developing economies such as India, China and others. Moreover, increase in infectious disease and increased adoption of advanced medicines and vaccines due to the developing healthcare facilities. Latin America, Middle East and Africa is expected to show substantial growth over the forecast period.

MRNA Vaccines and Therapeutics Market Key Players: Moderna Therapeutics Translate Bio BioNTech Argos Therapeutics eTheRNA Sangamo Therapeutics In-Cell-Art CureVac Ethris Tiba Biotechnology Others

This comprehensive report will provide:

Enhance your strategic decision making Assist with your research, presentations and business plans Show which emerging market opportunities to focus on Increase your industry knowledge Keep you up-to-date with crucial market developments Allow you to develop informed growth strategies Build your technical insight Illustrate trends to exploit Strengthen your analysis of competitors Provide risk analysis, helping you avoid the pitfalls other companies could make Ultimately, help you to maximize profitability for your company.

Our Market Research Solution Provides You Answer to Below Mentioned Question:

Which are the driving factors responsible for the growth of market? Which are the roadblock factors of this market? What are the new opportunities, by which market will grow in coming years? What are the trends of this market? Which are main factors responsible for new product launch? How big is the global & regional market in terms of revenue, sales and production? How far will the market grow in forecast period in terms of revenue, sales and production? Which region is dominating the global market and what are the market shares of each region in the overall market in 2017? How will each segment grow over the forecast period and how much revenue will these segment account for in 2025? Which region has more opportunities?

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Our industry professionals are working reluctantly to understand, assemble and timely deliver assessment on impact of COVID-19 disaster on many corporations and their clients to help them in taking excellent business decisions. We acknowledge everyone who is doing their part in this financial and healthcare crisis.

The Essential Content Covered in the GlobalMRNA Vaccines and Therapeutics Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

The report provides a 6-year forecast (2020-2026) assessed based on how the MRNA Vaccines and Therapeutics market is predicted to grow in major regions like USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Strategic Points Covered in TOC:

Chapter 1:Introduction, market driving force product scope, market risk, market overview, and market opportunities of the global MRNA Vaccines and Therapeutics market.

Chapter 2:Evaluating the leading manufacturers of the global MRNA Vaccines and Therapeutics market which consists of its revenue, sales, and price of the products.

Chapter 3: the competitive nature among key manufacturers, with market share, revenue, and sales.

Chapter 4:Presenting global MRNA Vaccines and Therapeutics market by regions, market share and revenue and sales for the projected period.

Chapters 5, 6, 7, 8 and 9:To evaluate the market by segments, by countries and by manufacturers with revenue share and sales by key countries in these various regions.

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MRNA Vaccines and Therapeutics Market research, Industry Outlook, Current Trends and Forecast by 2025 - Scientect

PITTSBURGH, Aug. 17, 2020 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (Nasdaq:KRYS), a fully integrated gene therapy company driven by its proprietary, engineered herpes simplex virus type 1 vector (HSV-1) platform, today announced that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to KB407, currently in preclinical development for the treatment of cystic fibrosis (CF).

We are pleased to receive Orphan Drug Designation for KB407 to treat cystic fibrosis as this is an important step forward in our efforts to address the continued unmet need in this devastating disease, said Suma M. Krishnan, founder and chief operating officer of Krystal Biotech. We are excited by the results of the in vitro data thus far, as presented at ASGCT earlier this year, and we look forward to sharing in vivo animal data later this year.

The FDAs Office of Orphan Drug Products grants Orphan Drug Designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan Drug Designation may allow Krystal Biotech to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of KB407, tax credits for certain clinical research costs and a waiver of the Prescription Drug User Fee Act (PDUFA) filing fees, subject to certain conditions.About KB407 for Cystic FibrosisKB407 is an inhaled, repeat-dose gene therapy product currently in the preclinical phase with plans to file an IND in 2021. In pre-clinical studies to date, KB407 has been able to successfully transduce human CF patient-derived epithelial cells and deliver functional cystic fibrosis transmembrane conductance regulator (CFTR) in vitro in 2D and 3D organotypic systems. Additional data has shown that the therapy is amendable to non-invasive inhaled administration in vivo, as indicated by successful delivery to the lungs through the use of a clinically relevant nebulizer in rodent healthy and diseased small animal models.

AboutKrystal BiotechKrystal Biotech, Inc. (NASDAQ:KRYS) is a gene therapy company dedicated to developing transformative medicines to treat diseases caused by protein or gene dysfunction. For more information, please visit http://www.krystalbio.com.

Forward-Looking StatementsAny statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., including but not limited to statements about the development of Krystals product candidates, such as plans for the design, conduct and timelines of ongoing clinical trials of beremagene geperpavec (B-VEC), KB105, KB104, KB301 and KB407; the clinical utility of B-VEC, KB105, KB104, KB301 and KB407, and Krystals plans for filing of regulatory approvals and efforts to bring B-VEC, KB105, KB104, KB301 and KB407 to market; the market opportunity for and the potential market acceptance of B-VEC, KB105, KB104, KB301 and KB407; plans to pursue research and development of other product candidates; the sufficiency of Krystals existing cash resources; the unanticipated impact of COVID-19 on Krystals business operations, pre-clinical activities and clinical trials; and other statements containing the words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, likely, will, would, could, should, continue, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including B-VEC, KB105, KB104, KB301 and KB407, the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption Risk Factors in Krystals annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystals views as of any date subsequent to the date of this release.

CONTACTS:

Investors:Ashley R. RobinsonLifeSci Advisorsarr@lifesciadvisors.com

Media:Darren Opland, PhDLifeSci Communicationsdarren@lifescicomms.com

Source: Krystal Biotech, Inc.

Read more:
Krystal Biotech's KB407 Granted Orphan Drug Designation by the FDA to Treat Patients With Cystic FibrosisThe company is on track to file an IND for...