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Archive for the ‘Gene Therapy Research’ Category

$14.6M Grant to Explore a Therapy to Control HIV Without Meds – POZ

In nearly 40 years of the HIV epidemic, only two people have likely been cured of the virus. Both scenarios resulted from stem cell transplants needed to fight blood cancers such as leukemia. Inspired by these two cases, a team of scientists is studying a multipronged way to potentially control HIV without medication. It involves two different genetic alterations of immune cells and with a safer method of stem cell transplants, also referred to as bone marrow transplants, a procedure that is generally toxic and dangerous.

The research is being funded by a five-year $14.6 million grant from the National Institutes of Health. The scientists coleading the preclinical studies are Paula Cannon, PhD, a distinguished professor of molecular microbiology and immunology at the Keck School of Medicine of the University of Southern California, and Hans-Peter Kiem, MD, PhD, who directs the stem cell and gene therapy program at the Fred Hutchinson Cancer Research Center, also known as Fred Hutch. According to a Keck School of Medicine press release, the two other main partners are David Scadden, MD, a bone marrow transplant specialist and professor at Harvard University and the Harvard Stem Cell Institute, and the biotechnology company Magenta Therapeutics.

In the HIV cure scenariosinvolving the so-called Berlin and London patientsboth men received stem cell transplants from donors with a natural genetic mutation that made them resistant to HIV. Specifically, their genes resulted in immune cells that lack CCR5 receptors on their surface (HIV latches onto these receptors to infect cells). Unfortunately, this method isnt viable for the nearly 38 million people worldwide living with HIV. Not only is it expensive, toxic and riskyit involves wiping out the patients immune system and replacing it with the new immune cellsbut it also requires matched donors who are CCR5 negative. According to the press release, about 1% of the population have this mutation.

With funding from this new grant, researchers hope to overcome these challenges in several ways. First, Cannon has already developed a gene-editing method to remove the CCR5 receptors from a patients own stem cells. She now hopes to further genetically engineer stem cells so they release antibodies that block HIV.

Our engineered cells will be good neighbors, Cannon said in the press release. They secrete these protective molecules so that other cells, even if they arent engineered to be CCR5 negative, have some chance of being protected.

Fred Hutchs Kiem will use CAR-T therapya new method of genetically modifying immune cells that is emerging out of cancer researchwith the goal of creating T cells that attack HIV-infected cells.

In addition, other scientists involved in the federal grant aim to develop less toxic methods of bone marrow transplantationfor example, by reducing the amount of chemotherapy required and speeding up the process of creating the new immune system.

The research finding could translate to other illnesses, such as cancer, sickle cell anemia and autoimmune disorders.

A home run would be that we completely cure people of HIV, Cannon said. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

For the latest on the cure cases, see Famed London Man Probably Cured of HIV from earlier this year. And in related news, see $14M Federal Grant to Research CAR-T Gene Therapy to Cure HIV.

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$14.6M Grant to Explore a Therapy to Control HIV Without Meds - POZ

Sangamo Therapeutics Appoints Kenneth Hillan to Its Board of Directors – Business Wire

BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced the appointment of Dr. Kenneth Hillan, an accomplished biotechnology executive, to the Company's Board of Directors.

"We are pleased to welcome Kenneth to the Sangamo Board of Directors," said Sandy Macrae, Chief Executive Officer of Sangamo. "We believe that Kenneths 25 years of clinical development and executive leadership experience, as well as his current focus on the therapeutic applications of DNA sequencing data, will provide great benefit to Sangamo as we continue to advance our growing pipeline of genomic medicines through clinical development.

Since 2019, Dr. Hillan has served as Head of Therapeutics of 23andMe, the consumer genetics and research company, leading a dedicated discovery research and therapeutic development team that uses human genetic data to identify and pursue novel therapies for common and rare diseases. Previously, Dr. Hillan held leadership roles in the biotechnology industry. He served at Genentech from 1994 to 2011, where he led the medical and scientific strategies for its Immunology, Tissue Growth and Repair drug portfolio, and held a number of key leadership positions in research and development, including Senior Vice President of Clinical Development, Inflammation; Vice President of Immunology, Tissue Growth and Repair; Vice President of Development Sciences; and Vice President of Research Operations and Pathology. He also served as Genentechs Senior Vice President and Head of Clinical Development and Product Development Strategy in Asia-Pacific for Roche in Shanghai, China. From 2011 to 2017, he was Chief Executive Officer of Achaogen, Inc., where he was also a member of the Board of Directors. He has also served on the Board of Directors of Zymeworks since February 2017, and was a member of the Board of Directors of Relypsa from 2014 until 2016, when it was acquired by Galenica AG.

Dr. Hillan has an M.B. Ch.B. (Bachelor of Medicine and Surgery) degree from the Faculty of Medicine at the University of Glasgow, U.K. He is a Fellow of the Royal College of Surgeons (FRCS, Glasg), and a Fellow of the Royal College of Pathologists (FRCPath). Dr. Hillan has authored dozens of scientific publications and is a named inventor on almost 50 issued patents.

Sangamo also today announced that Stephen Dilly, M.B.B.S., Ph.D. will resign from the Board of Directors effective September 30, 2020.

It has been a great pleasure serving as a Sangamo director for the past decade, said Dr. Dilly. With a broad therapeutic pipeline, meaningful collaborations with global biopharmaceutical companies, and a strong balance sheet, Sangamo is well positioned as a leader in the field of genomic medicine. I'm personally very pleased to welcome Kenneth Hillan to the Board and am excited for the future success of Sangamo."

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and genome regulation. For more information about Sangamo, visit http://www.sangamo.com.

Forward Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize therapies to treat genetic diseases and the timing thereof, the anticipated benefits of Sangamos collaborations, Sangamos financial resources and expectations and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators. These risks and uncertainties are described more fully in Sangamo's Securities and Exchange Commission filings and reports, including in Sangamos Quarterly Report on Form 10-Q for the quarter ended June 30, 2020. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

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Sangamo Therapeutics Appoints Kenneth Hillan to Its Board of Directors - Business Wire

Covid-19 roundup: NIH spotlights blood thinners in its next big trial; All it took was a pandemic to get JPM out of SF – Endpoints News

After immune modulators and antibodies, blood thinners are the next class of drugs to be put through a Phase III trial organized under the NIHs ACTIV initiative.

Three different anticoagulants have been named for the master protocol: heparin, aspirin and apixaban. Informed by studies suggesting that many patients who died from Covid-19 have formed unusual blood clots throughout their bodies including in their smallest blood vessels the NIH wants to see which therapies are the most effective at preventing or reducing them and thus improving outcomes for patients.

There is currently no standard of care for anticoagulation in hospitalized COVID-19 patients, and there is a desperate need for clinical evidence to guide practice, agency head Francis Collins said in a statement. Conducting trials using multiple existing networks of research sites provides the scale and speed that will get us answers faster.

For the inpatient trial, patients will be given either a low dose or a high dose of heparin.

But hospitalized patients are just one of two groups now being recruited to ACTIV-4, which also includes an outpatient trial broken down into four cohorts given placebo, aspirin, or a low or therapeutic dose of the blood thinner apixaban. The goal there is to reduce life-threatening cardiovascular or pulmonary complications in newly-diagnosed Covid-19 patients who dont need hospital admission.

Gary Gibbons, director of the National Heart, Lung, and Blood Institute, said while heparin has shown promise, physicians really need clinical trial data to determine how much blood thinner, or even anti-platelet medication, to give.

A third trial will start soon involving patients who had moderate or severe disease but have been discharged after hospitalization. It has a slightly different design, with the key metric being whether patients develop thrombotic complications (think heart attack, stroke, blood clots in major veins or arteries and death) within 45 days of being hospitalized.

As is custom in these master protocols, researchers may switch out candidates or add in new therapies as clinical data emerge.

NHLBI is coordinating and overseeing the program, while Operation Warp Speed is providing the funding. Amber Tong

The JP Morgan Healthcare Conference just got a whole lot cheaper.

On Thursday, the bank told clients that the famed conference was going virtual. That means no more packing into ballrooms and conference halls, no more dashing between different hotel rooms for an endless circle of meetings, and crucially no more $2,000 suites and $150 rent-a-tables. Instead, there will only be what there has been for the last 6 months: Endless circles of Zoom meetings, looped in infinite recursion from San Diego to Boston to Cambridge, UK.

The virtual conference means that, after years of chatter, the conference will finally, at least for a year, leave San Francisco. In theory, though, you could still go. A room at the Westin is selling for only $276, breakfast not included. Jason Mast

Longtime FDA officials and drug companies are trying to shore up public confidence in an eventual Covid-19 vaccine, but a new poll suggests that, at least until the election, they could be facing an uphill battle.

The poll, conducted by theKaiser FamilyFoundation,found that most adults, both Republican and Democrat, are wary of taking a vaccine before the election. Six in ten adults, meanwhile, were worried that the Trump Administration would pressure the FDA to rush an approval. The poll was conducted between August 28 and September 3 days after a heavily politicized emergency use authorization for convalescent plasma stoked fears that a vaccine would be authorized under similar conditions.

Fear of political pressure was most acute among Democrats, 51% of whom said they were very worried and 85% of whom were at least somewhat worried. A majority of Republicans said they were not too worried or not at all worried, although 35% expressed some concern.

As to whether they would take a vaccine, though, the splits are reversed. Half of Democrats were willing to take a vaccine, but only 36% of Republicans said they would. These numbers are in linewith long-running differences between Democrats and Republicans on vaccination, though it does suggest that recent events may have dented views. A Gallup pollconducted in late July and early August found 81% of Democrats and 47% of Republicans were willing to take an an FDA-approved vaccine to prevent coronavirus/COVID-19 if it was available right now at no cost. Those questions, though, did not directly reference the election.

Still, most respondents did not think such an eventuality was likely. Eighty-one percent of people, regardless of party, did not expect a vaccine to come before November 3.

The polls come as officials in the US struggle to shore up confidence in any eventually approved vaccine. Earlier this week, 9 major drug companies signed a pledge to only submit vaccine data when it meets safety and efficacy standards and 8 FDA career officialspublishedan open letter in USA Today on their commitment to science. Even before the latest concerns of political interference, public health experts have talked for months about how rising rates of vaccine hesitancy could pose a barrier in pandemic response. Bill Gates has called getting people to actually take a vaccine the final hurdle.

New data highlight how criticalmessaging can be on vaccine uptake. A large study published this morning inThe Lancettracked global vaccine hesitancy from 2015 to 2019 identified misinformation as one of the key factors in places with growing skepticism of vaccines, including South Korea, Malaysia, and Georgia. Anthropologist and director of the Vaccine Confidence Project Heidi Larson, the studys author, said messaging in the US and elsewhere that focused on speed wasnt helping.

Theres a lot of anxiety about the speed of vaccine development (for COVID-19), she told Reuters. But the public is not really keen on speed theyre more keen on thoroughness, effectiveness and safety. Jason Mast

For a look at allEndpoints Newscoronavirus stories, check out ourspecial news channel.

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Covid-19 roundup: NIH spotlights blood thinners in its next big trial; All it took was a pandemic to get JPM out of SF - Endpoints News

After Novartis left, Boehringer bets up to $500M on the field of prescription smartphone apps – Endpoints News

Boehringer Ingelheim wants in on digital therapeutics.

The German pharma signed an up to $500 million collaboration with Click Therapeutics, one of a handful of major companies developing prescription computer and smartphone applications for various neurological or mental disorders. They join a small list of pharmas that have decided to test the waters in the emerging field.

Under the deal, Boehringer and Click will co-develop a digital therapeutic for schizophrenia, known as CT-155. Its a complementary effort to Boehringers more traditional schizophrenia work. The company has concentrated their neuroscience efforts there since 2018, when a Phase II failure blew up their late-stage Alzheimers efforts.

CT-155 is an excellent addition to our CNS pipeline portfolio; it reflects our patient centric approach and translates evidence showing how behavioral modification can induce positive neuronal changes into a widely accessible solution,Cornelia Dorner-Ciossek, Boehringers head of CNS disease research, said in a statement.

Boehringer is at least the third Big Pharma to dip their toes into digital therapeutics. Sanofi Ventures the French giants investment arm led a $17 million funding round for Click in 2018, the same year Mercks venture arm contributed to the video game therapeutics company Akilis $55 million Series C. Novartis also signed a partnership that year with Pear Therapeutics to commercialize Pears FDA-authorized app for substance abuse, although they pulled out of the partnership just one year later.

Boehringers new deal marks one of the first major development partnerships between a large pharma company and a digital therapeutic.

Click has already developed a commercial app for smoking cessation called Clickotine. Clickotine, like the other apps Click has in development for insomnia, migraine and major depressive disorder, among others, uses a variety of smartphone-based features to try to adjust peoples behavior. In Clickotines case, that involves personalized curriculums that offer users live coaching, guided breathing, and personal analytics. That app, though, while backed by a non-controlled study, did not have to be FDA-approved as other apps will.

Click has released little about CT-155 its description on their website reads simply confidential but in theory it could be used alongside existing schizophrenia drugs and, eventually, alongside the schizophrenia meds Boehringer now has in its pipeline. Two such compounds are now in Phase II.

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After Novartis left, Boehringer bets up to $500M on the field of prescription smartphone apps - Endpoints News

Achieving Quality Cell and Gene Therapies Outcomes Through Multidisciplinary Collaboration, Upcoming Webinar Hosted by Xtalks – PR Web

Xtalks Life Science Webinars

TORONTO (PRWEB) September 11, 2020

During the last five years, the number of cell and gene therapies (CGT) have exponentially increased, stimulated by their efficacy and potential for cure. However, CGT can expose the patients to significant toxicities such as cytokine release syndrome (CRS) and neurotoxicities (ICANS), requiring specialised and accurate management associated with precise data capture and analysis. Timely and unrestricted support is crucial for the safe progress of CGT. In addition, dealing with these living therapies represents whats been called the most complex supply chain in the history of medicine.

This webinar will present and discuss the challenges involved in supporting CGT-related clinical trials. Attendees will gain first-hand learning from members of a multidisciplinary team that was developed at ICON for the successful support of CGT clinical trials.The participants will learn the following during the presentation:

A The medical landscape:

B Operational management:

C Commercialisation and logistics

Join Anne S. Renteria, MD, Medical Director, Medical Affairs, ICON plc, Brandon Fletcher, PhD, Principal, Clinical Project Management, ICON plc and Tamie Joeckel, Business Lead, Cell and Gene Therapy Center of Excellence, ICON plc in a live webinar on Thursday, September 24, 2020 at 10am EDT (3pm BST/UK).

For more information or to register for this event, visit Achieving Quality Cell and Gene Therapies Outcomes Through Multidisciplinary Collaboration.

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.comFor information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

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Achieving Quality Cell and Gene Therapies Outcomes Through Multidisciplinary Collaboration, Upcoming Webinar Hosted by Xtalks - PR Web

Seeing the eye like never before – Science Codex

While there is no cure for blindness and macular degeneration, scientists have accelerated the process to find a cure by visualizing the inner workings of the eye and its diseases at the cellular level.

In an effort led by UW Medicine, researchers successfully modified the standard process of optical coherence tomography (OCT) to detect minute changes in response to light in individual photoreceptors in the living eye.

The results were published Sept. 9 in Science Advances.

"We have now accelerated the life cycle of vision restoration," said lead author Vimal Prabhu Pandiyan, a ophthalmology researcher at the University of Washington School of Medicine.

The study was funded in part by the National Eye Institute's Audacious Goals Initiative, which embraces bold ideas in helping people to see better.

The OCT modifications outlined in the study will help researchers who want to test therapies such as stem cells or gene therapy to treat retinal disease. They now have the tools to zoom in on the retina to evaluate whether the therapy is working.

Corresponding author Ramkumar Sabesan, a UW assistant research professor of ophthalmology, said the only way to objectively measure the eye currently is to look at a wide retinal area. Sabesan said researchers currently can attach electrodes on the cornea but it captures a large area with around 1 million cells. Now they are talking about nanometers, or one billionth of a meter - a small fraction of the size of a cell, providing orders of magnitude improvement.

"Since photoreceptors are the primary cells affected in retinal generation and the target cells of many treatments, noninvasive visualization of their physiology at high resolution is invaluable," the researchers wrote.

Cone photoreceptors are the building blocks of sight, capturing light and funneling information to the other retinal neurons. They are a key ingredient in how we process images and patterns of light falling on the retina.

Optical coherence tomography has been around since the 1990s. In this study, researchers used OCT with adaptive optics, line-scanning and phase-resolved acquisition to deliver the concept of Thomas Young's interference to the human eye. With the ability to zoom in on the retina at high speeds, they found that cone photoreceptors deform at the scale of nanometers when they first capture light and begin the process of seeing.

As Sabesan explained: "You can imagine a picture that looks visually and structurally normal. But when we interrogate the inner working of the retina at a cellular scale, we may detect a dysfunction sooner than what other modalities can do. A doctor then can prescribe medication to intervene early or follow the time-course of its repair via gene therapy or stem cell therapy in the future."

"We will now have a way to see if these therapies are acting in the way they should," Sabesan said.

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Seeing the eye like never before - Science Codex

How CRISPR is tackling the troubling immune response that’s plagued gene therapy until now – Best gaming pro

One of many major challenges facing gene therapy a approach to deal with illness by changing a affected persons faulty genes with wholesome ones is that its troublesome to securely ship therapeutic genes to sufferers with out the immune system destroying the gene, and the car carrying it, which might set off life-threatening widespread irritation.

Three a long time in the past researchers thought that gene remedy can be the last word remedy for genetically inherited illnesses like hemophilia, sickle cell anemia, and genetic illnesses of metabolism. However the know-how couldnt dodge the immune response.

Since then, researchers have been searching for methods to good the know-how and management immune responses to the gene or the car. Nevertheless, lots of the methods examined to date have not been completely successful in overcoming this hurdle.

Medicine that suppress the entire immune system, similar to steroids, have been used to dampen the immune response when administering gene remedy. However its troublesome to regulate when and the place steroids work within the physique, they usually create undesirable unwanted side effects. My colleague Mo Ebrahimkhani and I wished to deal with gene remedy with immune-suppressing instruments that have been simpler to regulate.

I am a medical doctor and synthetic biologist involved in gene remedy as a result of six years in the past my father was recognized with pancreatic cancer. Pancreatic most cancers is without doubt one of the deadliest types of most cancers, and the at present out there therapeutics often fail to avoid wasting sufferers. Consequently, novel therapies similar to gene remedy could be the one hope.

[Learn: These tech tendencies outlined 2020 to date, in keeping with 5 founders]

But, many gene therapies fail as a result of sufferers both have already got pre-existing immunity to the car used to introduce the gene or develop one in the midst of remedy. This downside has plagued the sector for many years, stopping the widespread utility of the know-how.

Historically scientists use viruses from which harmful disease-causing genes have been eliminated as automobiles to move new genes to particular organs. These genes then produce a product that may compensate for the defective genes which might be inherited genetically. That is how gene remedy works.

Although there have been examples displaying that gene therapy was helpful in some genetic illnesses, theyre nonetheless not good. Generally, a defective gene is so large that you couldt merely match the wholesome alternative within the viruses generally utilized in gene remedy.

One other downside is that when the immune system sees a virus, it assumes that its a disease-causing pathogen and launches an assault to battle it off by producing antibodies and immune response simply as occurs when individuals catch another infectious viruses, like SARS-CoV-2 or the frequent chilly.

Not too long ago, although, with the rise of a gene-editing technology called CRISPR, scientists can do gene remedy otherwise.

CRISPR can be utilized in some ways. In its main function, it acts as a genetic surgeon with a pointy scalpel, enabling scientists to discover a genetic defect and proper it throughout the native genome in desired cells of the organism. It might probably additionally restore multiple gene at a time.

Scientists can even use CRISPR to show off a gene for a brief time frame after which flip it again on, or vice versa, with out completely altering the letters of DNA that makes up our genome. Which means that researchers like me can leverage CRISPR know-how to revolutionize gene therapies within the coming a long time.

However to make use of CRISPR for both of those features, it nonetheless must be packaged right into a virus to get it into the physique. So some challenges, similar to stopping the immune response to the gene remedy viruses, nonetheless have to be solved for CRISPR-based gene therapies.

Being educated as a synthetic biologist, I teamed up with Ebrahimkhani to make use of CRISPR to check whether or not we may shut down a gene thats liable for the immune response that destroys the gene remedy viruses. Then we investigated whether or not decreasing the exercise of the gene, and dulling the immune response, would permit the gene remedy viruses to be more practical.

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CRISPR can exactly take away even single items of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene within the immune system and controls the response to micro organism and viruses, together with the frequent gene remedy viruses. We determined to briefly flip off this gene in the entire physique of lab animals.

We injected animals with a group of the CRISPR molecules that focused the Myd88 gene and seemed to see whether or not this lowered the variety of antibodies that have been produced to particularly battle our gene remedy viruses. We have been excited to see that the animals that obtained our remedy utilizing CRISPR produced much less antibodies towards the virus.

This prompted us to ask what occurs if we give the animal a second dose of the gene remedy virus. Often, the immune response towards a gene remedy virus prevents the remedy from being administered a number of occasions. Thats as a result of after the primary dose, the immune system has seen the virus, and on the second dose, antibodies swiftly assault and destroy the virus earlier than it could possibly ship its cargo.

We noticed that animals receiving multiple dose didnt present a rise in antibodies towards the virus. And, in some instances, the impact of gene remedy improved in contrast with the animals through which we had not paused the Myd88 gene.

We additionally did plenty of different experiments that proved that tweaking the Myd88 gene might be helpful in combating off different sources of irritation. That could possibly be helpful in illnesses like sepsis and even COVID-19.

Whereas we at the moment are starting to enhance this technique by way of controlling the exercise of the Myd88 gene. Our outcomes, now revealed in Nature Cell Biology, present a path ahead to program our immune system throughout gene therapies and different inflammatory responses utilizing the CRISPR know-how.

This text is republished from The Conversation by Samira Kiani, Affiliate Professor of Pathology, University of Pittsburghbeneath a Artistic Commons license. Learn the original article.

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How CRISPR is tackling the troubling immune response that's plagued gene therapy until now - Best gaming pro

Maryland Labs Developing Vaccines And Therapeutics, Attracting Federal Dollars – Patch.com

By Bruce DePuytSeptember 10, 2020

A 2016 decision to link the research labs at the University of Maryland College Park with those at the University of Maryland at Baltimore is paying dividends in the competition for federal COVID-19 research dollars, a top education official told a science advisory panel on Wednesday.

The decision was a spinoff of the University of Maryland Strategic Partnership Act, said Laurie Ellen Locasio, vice president for research at the two institutions.

"Now we'll be ranked together by the [National Science Foundation] in the U.S. rankings of research universities and together this makes us [an] over $1 billion public research enterprise," she told the Maryland Life Sciences Advisory Board.

With a multi-billion dollar research powerhouse, the privately-funded Johns Hopkins University, also operating here, Maryland is unusually well-positioned, Locasio said.

"Very few states in the country have the distinction of having two research universities with over a billion dollars in research spending per year. We've really shown up as central to this pandemic."

Martin Rosendale, the CEO of the Maryland Tech Council, said there are so many therapeutics and vaccines in development that keeping up with them all is a daunting task.

Many firms adopted a "drop everything" approach when the scope of the coronavirus challenge started to become apparent, he said.

"We just saw a lot of amazing thing happening here in Maryland so many companies were pivoting immediately to support the response to the pandemic," he said. "They were basically dropping their other work and on their own dime spending their own money evaluating their technologies, their platforms, how they could apply them to COVID-19."

Many of the big players have grabbed global headlines for their work on a possible vaccine. But smaller companies are making potentially valuable strides as well, Commerce Secretary Kelly M. Schulz told the panel.

Other firms are working to develop COVID tests that are faster and more reliable.

The Tech Council formed the Maryland COVID-19 Coalition to bring companies together, "to make sure they knew who each other were and begin a conversation," Rosendale said. Twenty-five firms were part of the coalition's first call; 40 were on the second.

Chris P. Austin, director of the National Center for Advancing Translation Sciences at NIH, told the panel that the federal government has pumped more than $20 billion into COVID research.

"Of that, we know that over $3 billion is coming here to Maryland," Rosendale said. "That's a testament to the amazing vaccine and cell-and-gene therapy industries that have grown up here."

If foundation and other non-government funds are added to the public dollars, "the number is well over $4 billion," he added.

To play off the capital region's strengths in biotech, a group of industry leaders in Maryland, Washington, D.C., and Virginia is organizing a "pandemic and bio-defense center" to help countries around the globe guard against future pandemics.

"Because of the prominence of what's going on with COVID-19 development in our region especially within Maryland with vaccine, diagnostics, and therapeutic research we are sort of the epicenter in the world right now," said Richard A. Bendis, president and CEO of BioHealth Innovation, Inc. in Rockville.

"People are starting to take notice."

Schulz said the Department of Commerce paused its planned marketing efforts in the spring to focus on the pandemic, but now is ready to gear back up again with an "Innovation Uncovered" campaign.

"We're going back to where we wanted to be at the beginning of the year, which was to push the bio- and the life sciences worlds out, because we have heard from [company executives] that Maryland needs to be seen as a strong, tight ecosystem for this industry," she said.

bruce@maryandmatters.org

For more stories from Maryland Matters, visit http://www.marylandmatters.org.

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Maryland Labs Developing Vaccines And Therapeutics, Attracting Federal Dollars - Patch.com

Gene Therapy Market to Eyewitness Massive Growth by 2026: Leading Key Players Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc….

This report focuses on the Global Gene Therapy Market trends, future forecasts, growth opportunities, key end-user industries, and market players. The objectives of the study are to present the key developments of the market across the globe.

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Reports include the following segmentation: By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions OthersBy Type of Vectors Viral vectors Non-viral vectorsBy Type of Cells Somatic cells Germline cellsBy 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

The report has been curated after observing and studying various factors that determine regional growth such as economic, environmental, social, technological, and political status of the particular region. Analysts have studied the data of revenue, production, and manufacturers of each region. This section analyses region-wise revenue and volume for the forecast period of 2015 to 2026. These analyses will help the reader to understand the potential worth of investment in a particular region.

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The major players in the market Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation Others

Global Gene Therapy MarketThis research report providesCOVID-19 Outbreakstudy accumulated to offer Latest insights about acute features of the Gene Therapy Market. The report contains different market predictions related to marketsize, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market. It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary andSWOT analysis.It presents the360-degreeoverview of the competitive landscape of the industries. Gene Therapy Market is showing steadygrowthandCAGRis expected to improve during the forecast period.

The main sources are industry experts from the global Gene Therapy industry, including management organizations, processing organizations, and analytical services providers that address the value chain of industry organizations. We interviewed all major sources to collect and certify qualitative and quantitative information and to determine future prospects. The qualities of this study in the industry experts industry, such as CEO, vice president, marketing director, technology and innovation director, founder and key executives of key core companies and institutions in major biomass waste containers around the world in the extensive primary research conducted for this study We interviewed to acquire and verify both sides and quantitative aspects.

Global Gene Therapy Market: Regional AnalysisThe report offers in-depth assessment of the growth and other aspects of the Gene Therapy market in important regions, including the U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, Taiwan, Southeast Asia, Mexico, and Brazil, etc. Key regions covered in the report are North America, Europe, Asia-Pacific and Latin America.

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The key findings and recommendations highlight vital progressive industry trends in the global Gene Therapy market, thereby allowing players to improve effective long term policies

A complete analysis of the factors that drive market evolution is provided in the report.

To analyze opportunities in the market for stakeholders by categorizing the high-growth segments of the market

The numerous opportunities in the Gene Therapy market are also given.

Report Answers Following Questions:

What are the factors driving the growth of the market?

What factors are inhibiting market growth?

What are the future opportunities in the market?

Which are the most dynamic companies and what are their recent developments within the Gene Therapy Market?

What key developments can be expected in the coming years?

What are the key trends observed in the market?

TABLE OF CONTENT

1 Report Overview

2 Global Growth Trends

3 Market Share by Key Players

4 Breakdown Data by Type and Application

5 United States

6 Europe

7 China

8 Japan

9 Southeast Asia

10 India

11 Central & South America

12 International Players Profiles

13 Market Forecast 2020-2025

14 Analysts Viewpoints/Conclusions

15 Appendix

Read Full Report: https://industrystatsreport.com/Lifesciences-and-Healthcare/Gene-Therapy-Market-Share/Summary

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Gene Therapy Market to Eyewitness Massive Growth by 2026: Leading Key Players Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc....

Cancer Gene Therapy Market Size Will Expand Post COVID-19 Covered in Forecast Report 2020-2026 – NJ MMA News

[190+ Pages PDF Report] Facts & Factors (FnF) published a market research report onCancer Gene Therapy Market: By Size, Share, Growth, Analysis Covering COVID-19 Impact and Forecast to 2020-2026that includes a research report with TOC including a list of tables and figures in its research offerings.

Cancer Gene Therapy Market Market Report: Industry Insights, 2020-2026

This multi-client research study on theCancer Gene Therapy Market marketprovides in-depth research and analysis into Cancer Gene Therapy Market industry trends, market developments and technological insights. The report provides data and analysis of Cancer Gene Therapy Market penetration across application segments across countries and regions. The report presents a strategic analysis of the Cancer Gene Therapy Markety market through key drivers, challenges, opportunities, and growth contributors.

The global Cancer Gene Therapy Market market delivers value to customers through reliable market size for 2020 on the basis of demand and price analysis. The report presents near term and long term forecast of the addressable Cancer Gene Therapy Market market size to 2026.

The report presents an introduction to the Cancer Gene Therapy Market market in 2020, analyzing the COVID-19 impact both quantitatively and qualitatively.

Request an Exclusive FREE SAMPLE REPORT of Cancer Gene Therapy Market Market:https://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

(The sample of this report is readily available on request).

The FREE PDF Sample Report Includes:

(Note: The sample of this report is updated with COVID-19 impact analysis before delivery)

Some of Following Top Market Players Profile Included in This Report:

2020 Cancer Gene Therapy Market Market: COVID-19 Impact Analysis

The global rise of COVID-19 has many businesses struggling and confused about what steps to take to minimize the economic impact. A simple look at the stock market will tell you that coronavirus has led to a volatile economy, but there are numerous other factors at play.

The long term COVID-19 impact on the business industry is largely still unknown; hence,Facts and Factors market researchanalysts have already covered the effects of COVID-19 on the business industry at a large level, as well as global and regional levels. Through our coverage below, get a better understanding of the business and economic implications coronavirus has on trends like remote working, consumer shopping behavior, global advertising spend, and essential industries like food, medical, travel, and transportation.

Download the Free Sample Report PDF Brochure Including COVID-19 Business Impact:https://www.fnfresearch.com/sample/cancer-gene-therapy-market-by-type-ex-vivo-898

Cancer Gene Therapy Market Market 2020: Research Scope & Coverage

The report coversCancer Gene Therapy Market market sizeand growth, characteristics, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the markets historic and forecast market growth by geography. The market size analysis gives the market size covering both the historic growth of the market, the impact of the COVID 19 virus, and forecasting its recovery. The report also provides a comprehensive analysis of current & future trends and emerging avenues for the growth of this market along with this research also offers an insight into the competitive landscape in terms of new technological developments, untapped segments, and value chain analysis.

The report, Cancer Gene Therapy Market Market, provides up-to-date information about market size, share shifts, and potential risks, as well as in-depth knowledge of products and services, which can help in planning and in executing category management activities. It focuses on the cost-saving aspects of procurement and on providing insights that can lead to the optimization of category spend.

Get Free Customized Copy of Cancer Gene Therapy Market Market Report @https://www.fnfresearch.com/customization/cancer-gene-therapy-market-by-type-ex-vivo-898

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Competitive Landscape

The market appears to be fragmented and with the presence of several. Thissize of Cancer Gene Therapy Market marketresearch report will help clients identify new growth opportunities and design unique growth strategies by providing a comprehensive analysis of the markets competitive landscape and offering information on the products offered by companies.

Key Takeaways from this Cancer Gene Therapy Market Market Report

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Cancer Gene Therapy Market Size Will Expand Post COVID-19 Covered in Forecast Report 2020-2026 - NJ MMA News

Global and US Gene Therapy for Ovarian Cancer Market to Witness Huge Growth by abc Major Players included in report Takara Bio, VBL Therapeutics,…

Global Coronavirus pandemic has impacted all industries across the globe, Gene Therapy for Ovarian Cancer market being no exception. As Global economy heads towards major recession post 2009 crisis, Cognitive Market Research has published a recent study which meticulously studies impact of this crisis on Global Gene Therapy for Ovarian Cancer market and suggests possible measures to curtail them. This press release is a snapshot of research study and further information can be gathered by accessing complete report. To Contact Research Advisor Mail us @ [emailprotected] or call us on +1-312-376-8303.

The research report on global Gene Therapy for Ovarian Cancer market as well as industry is a detailed study that provides detailed information of major key players, product types & applications/end-users; historical figures, region analysis, market drivers/opportunities & restraints forecast scenarios, strategic planning, and a precise section for the effect of Covid-19 on the market. Our research analysts intensively determine the significant outlook of the global Gene Therapy for Ovarian Cancer market study with regard to primary & secondary data and they have represented it in the form of graphs, pie charts, tables & other pictorial representations for better understanding.

Intravenous, Intratumoral, Intraperitoneal are some of the key types of market. All the type segments have been analyzed based on present and future trends and the market is estimated from 2020 to 2027. Based on the application segment, the global market can be classified into Ovarian Cancer (unspecified), Recurrent Ovarian Epithelial Cancer, Platinum Resistant Ovarian Cancer . The analysis of application segment will help to analyze the demand for market across different end-use industries.

Request Free Sample Copy of Gene Therapy for Ovarian Cancer Market Research [emailprotected] https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-ovarian-cancer-market-report#download_report

Amid the COVID-19 pandemic, the industry is witnessing a major change in operations.Some of the key players include Takara Bio, VBL Therapeutics, CELSION, Targovax . key players are changing their recruitment practices to comply with the social distancing norms enforced across several regions to mitigate the risk of infection. Additionally, companies are emphasizing on using advanced recruiting solutions and digital assets to avoid in-person meetings. Advanced technologies and manufacturing process are expected to play a decisive role in influencing the competitiveness of the market players.

Regional Analysis for Gene Therapy for Ovarian Cancer Market:North America (United States, Canada)Europe (Germany, Spain, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, India, Australia, and South Korea)Latin America (Brazil, Mexico, etc.)The Middle East and Africa (GCC and South Africa)

Get A Free Sample of Gene Therapy for Ovarian Cancer Market Report: https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-ovarian-cancer-market-report#download_report

NOTE: Whole world is experiencing the impact of Covid-19 pandemic due to its increasing spread hence, the report comprises of an up to date scenario of the Gene Therapy for Ovarian Cancer market report. Research analyst team of our company is understanding & reviewing the Covid19 Impact on Market and all the necessary areas of the market that have been altered due to the change caused by Covid19 impact. Get in touch with us for more precise/in-depth information of the Gene Therapy for Ovarian Cancer market.

Any query? Enquire Here For Discount (COVID-19 Impact Analysis Updated Sample): Click Here>Download Sample Report of Gene Therapy for Ovarian Cancer Market Report 2020 (Coronavirus Impact Analysis on Gene Therapy for Ovarian Cancer Market)

At the end of May, many states began lifting lockdown restrictions and reopening in order to revive their economies, despite warnings that it was still too early. As a result, by mid-July, around 33 states were reporting higher rates of new cases compared to the previous week with only three states reporting declining rates. Due to this Covid-19 pandemic, there has been disruptions in the supply chain which have made end-use businesses realize destructive in the manufacturing and business process. During this lockdown period, the plastic packaging helps the products to have longer shelf life as the public would not be able to buy new replacements for the expired products because most of the production units are closed.

About Us:Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.Contact Us: +1-312-376-8303Email: [emailprotected]Web: https://www.cognitivemarketresearch.com/

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Global and US Gene Therapy for Ovarian Cancer Market to Witness Huge Growth by abc Major Players included in report Takara Bio, VBL Therapeutics,...

VBL Presents Human Proof-of-Concept Data That Show the Potential of its Novel anti-MOSPD2 Monoclonal Antibodies for Multiple Sclerosis at the MS…

TEL AVIV, Israel, Sept. 11, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT), today presents for the first time new data demonstrating ex-vivo activity of its proprietary investigational anti-MOSPD2 mAbs in patients with relapsing-remitting and progressive multiple sclerosis (MS). VBL's study is being presented at the MS Virtual 2020, the virtual 8th Joint ACTRIMS-ECTRIMS Meeting.

Data show that VBL's anti-MOSPD2 mAbs significantly inhibited migration of monocytes isolated from all MS patients included in the study (n=33) by up to 97%, regardless of disease severity, gender or active treatment. Notably, the activity was seen not only in the monocytes from relapsing-remitting, but also those from primary progressive and secondary progressive patients with high Expanded Disability Status Scale (EDSS) scores of 5.5-6.5.

We believe our antibodies open up a completely novel mechanism for potential treatment of MS, through blocking the accumulation of monocytes/macrophages in the central nervous system, said Itzhak Mendel, Ph.D., Immunology Director of VBL Therapeutics. This mechanism is differentiated from the existing available treatments, which mostly target T and B cells and therefore, it is not surprising that our antibodies showed activity on top of any patient active therapy. Backed up by strong preclinical results, these first patient-driven proof-of-concept data reinforce the therapeutic potential of our MOSPD2 program.

VBL is currently advancing lead anti-MOSPD2 candidate VB-601 through IND-enabling studies, aiming to start a first-in-human study in 2H 2021.

For a link to VBL's presentation at the MS Virtual 2020 conference, see: LINK

About VBL's VB-600 PlatformVBL is conducting two parallel drug development programs that are exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases and cancer. Our VB-600 platform comprises classical anti-MOSPD2 monoclonal antibodies for inflammatory indications, as well as bi-specific antibody candidates for oncology.

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 may include, but are not limited to, statements regarding our programs, including MOSPD2, including their clinical development, therapeutic potential and clinical results. 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 theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. 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 Therapeuticsundertakes 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 Advisors, LLC(646) 597-6979

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VBL Presents Human Proof-of-Concept Data That Show the Potential of its Novel anti-MOSPD2 Monoclonal Antibodies for Multiple Sclerosis at the MS...

Precision Cancer Therapies Market, Share, Application Analysis, Regional Outlook, Competitive Strategies & Forecast up to 2025 |, Abbott…

LOS ANGELES, United States: QY Research as of late produced a research report titled, Global and United States Precision Cancer Therapies Market Size, Status and Forecast 2020-2026. The research report speak about the potential development openings that exist in the worldwide market. The report is broken down on the basis of research procedures procured from historical and forecast information. The global Precision Cancer Therapies market is relied upon to develop generously and flourish as far as volume and incentive during the gauge time frame. The report will give a knowledge about the development openings and controls that will build the market. Pursuers can increase important perception about the eventual fate of the market.

The global Precision Cancer Therapies market size is projected to reach US$ XX million by 2026, from US$ XX million in 2020, at a CAGR of XX% during 2021-2026.

Key Companies/Manufacturers operating in the global Precision Cancer Therapies market include: , Abbott Laboratories, Bayer HealthCare, GlaxoSmithKline, OncoGenex Pharmaceuticals, Hospira, Boehringer Ingelheim, AstraZeneca, Aveo Pharmaceuticals

Get PDF Sample Copy of the Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) :

https://www.qyresearch.com/sample-form/form/2125996/global-and-united-states-precision-cancer-therapies-market

Segmental Analysis

The report incorporates significant sections, for example, type and end user and a variety of segments that decide the prospects of global Precision Cancer Therapies market. Each type provide data with respect to the business esteem during the conjecture time frame. The application area likewise gives information by volume and consumption during the estimate time frame. The comprehension of this segment direct the readers in perceiving the significance of variables that shape the market development.

Global Precision Cancer Therapies Market Segment By Type:

Hormone TherapyImmunotherapiesTargeted TherapyMonoclonal Antibody TherapyGene Therapy Precision Cancer Therapies

Global Precision Cancer Therapies Market Segment By Application:

HospitalsDiagnostic CentersOncology ClinicsResearch Institutes

Competitive Landscape

Competitor analysis is one of the best sections of the report that compares the progress of leading players based on crucial parameters, including market share, new developments, global reach, local competition, price, and production. From the nature of competition to future changes in the vendor landscape, the report provides in-depth analysis of the competition in the global Precision Cancer Therapies market.

Key questions answered in the report:

For Discount, Customization in the Report Drop Your Query Here: https://www.qyresearch.com/customize-request/form/2125996/global-and-united-states-precision-cancer-therapies-market

TOC

1 Report Overview1.1 Study Scope1.2 Market Analysis by Type1.2.1 Global Precision Cancer Therapies Market Size Growth Rate by Type: 2020 VS 20261.2.2 Hormone Therapy1.2.3 Immunotherapies1.2.4 Targeted Therapy1.2.5 Monoclonal Antibody Therapy1.2.6 Gene Therapy1.3 Market by Application1.3.1 Global Precision Cancer Therapies Market Share by Application: 2020 VS 20261.3.2 Hospitals1.3.3 Diagnostic Centers1.3.4 Oncology Clinics1.3.5 Research Institutes1.4 Study Objectives1.5 Years Considered 2 Global Growth Trends2.1 Global Precision Cancer Therapies Market Perspective (2015-2026)2.2 Global Precision Cancer Therapies Growth Trends by Regions2.2.1 Precision Cancer Therapies Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Precision Cancer Therapies Historic Market Share by Regions (2015-2020)2.2.3 Precision Cancer Therapies Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Market Restraints 3 Competition Landscape by Key Players3.1 Global Top Precision Cancer Therapies Players by Market Size3.1.1 Global Top Precision Cancer Therapies Players by Revenue (2015-2020)3.1.2 Global Precision Cancer Therapies Revenue Market Share by Players (2015-2020)3.2 Global Precision Cancer Therapies Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.3 Players Covered: Ranking by Precision Cancer Therapies Revenue3.4 Global Precision Cancer Therapies Market Concentration Ratio3.4.1 Global Precision Cancer Therapies Market Concentration Ratio (CR5 and HHI)3.4.2 Global Top 10 and Top 5 Companies by Precision Cancer Therapies Revenue in 20193.5 Key Players Precision Cancer Therapies Area Served3.6 Key Players Precision Cancer Therapies Product Solution and Service3.7 Date of Enter into Precision Cancer Therapies Market3.8 Mergers & Acquisitions, Expansion Plans 4 Precision Cancer Therapies Breakdown Data by Type (2015-2026)4.1 Global Precision Cancer Therapies Historic Market Size by Type (2015-2020)4.2 Global Precision Cancer Therapies Forecasted Market Size by Type (2021-2026) 5 Precision Cancer Therapies Breakdown Data by Application (2015-2026)5.1 Global Precision Cancer Therapies Historic Market Size by Application (2015-2020)5.2 Global Precision Cancer Therapies Forecasted Market Size by Application (2021-2026) 6 North America6.1 North America Precision Cancer Therapies Market Size (2015-2026)6.2 North America Precision Cancer Therapies Market Size by Type (2015-2020)6.3 North America Precision Cancer Therapies Market Size by Application (2015-2020)6.4 North America Precision Cancer Therapies Market Size by Country (2015-2020)6.4.1 United States6.4.2 Canada 7 Europe7.1 Europe Precision Cancer Therapies Market Size (2015-2026)7.2 Europe Precision Cancer Therapies Market Size by Type (2015-2020)7.3 Europe Precision Cancer Therapies Market Size by Application (2015-2020)7.4 Europe Precision Cancer Therapies Market Size by Country (2015-2020)7.4.1 Germany7.4.2 France7.4.3 U.K.7.4.4 Italy7.4.5 Russia7.4.6 Nordic7.4.7 Rest of Europe 8 China8.1 China Precision Cancer Therapies Market Size (2015-2026)8.2 China Precision Cancer Therapies Market Size by Type (2015-2020)8.3 China Precision Cancer Therapies Market Size by Application (2015-2020)8.4 China Precision Cancer Therapies Market Size by Region (2015-2020)8.4.1 China8.4.2 Japan8.4.3 South Korea8.4.4 Southeast Asia8.4.5 India8.4.6 Australia8.4.7 Rest of Asia-Pacific 9 Japan9.1 Japan Precision Cancer Therapies Market Size (2015-2026)9.2 Japan Precision Cancer Therapies Market Size by Type (2015-2020)9.3 Japan Precision Cancer Therapies Market Size by Application (2015-2020)9.4 Japan Precision Cancer Therapies Market Size by Country (2015-2020)9.4.1 Mexico9.4.2 Brazil 10 Southeast Asia10.1 Southeast Asia Precision Cancer Therapies Market Size (2015-2026)10.2 Southeast Asia Precision Cancer Therapies Market Size by Type (2015-2020)10.3 Southeast Asia Precision Cancer Therapies Market Size by Application (2015-2020)10.4 Southeast Asia Precision Cancer Therapies Market Size by Country (2015-2020)10.4.1 Turkey10.4.2 Saudi Arabia10.4.3 UAE10.4.4 Rest of Middle East & Africa 11 Key Players Profiles11.1 Abbott Laboratories11.1.1 Abbott Laboratories Company Details11.1.2 Abbott Laboratories Business Overview11.1.3 Abbott Laboratories Precision Cancer Therapies Introduction11.1.4 Abbott Laboratories Revenue in Precision Cancer Therapies Business (2015-2020))11.1.5 Abbott Laboratories Recent Development11.2 Bayer HealthCare11.2.1 Bayer HealthCare Company Details11.2.2 Bayer HealthCare Business Overview11.2.3 Bayer HealthCare Precision Cancer Therapies Introduction11.2.4 Bayer HealthCare Revenue in Precision Cancer Therapies Business (2015-2020)11.2.5 Bayer HealthCare Recent Development11.3 GlaxoSmithKline11.3.1 GlaxoSmithKline Company Details11.3.2 GlaxoSmithKline Business Overview11.3.3 GlaxoSmithKline Precision Cancer Therapies Introduction11.3.4 GlaxoSmithKline Revenue in Precision Cancer Therapies Business (2015-2020)11.3.5 GlaxoSmithKline Recent Development11.4 OncoGenex Pharmaceuticals11.4.1 OncoGenex Pharmaceuticals Company Details11.4.2 OncoGenex Pharmaceuticals Business Overview11.4.3 OncoGenex Pharmaceuticals Precision Cancer Therapies Introduction11.4.4 OncoGenex Pharmaceuticals Revenue in Precision Cancer Therapies Business (2015-2020)11.4.5 OncoGenex Pharmaceuticals Recent Development11.5 Hospira11.5.1 Hospira Company Details11.5.2 Hospira Business Overview11.5.3 Hospira Precision Cancer Therapies Introduction11.5.4 Hospira Revenue in Precision Cancer Therapies Business (2015-2020)11.5.5 Hospira Recent Development11.6 Boehringer Ingelheim11.6.1 Boehringer Ingelheim Company Details11.6.2 Boehringer Ingelheim Business Overview11.6.3 Boehringer Ingelheim Precision Cancer Therapies Introduction11.6.4 Boehringer Ingelheim Revenue in Precision Cancer Therapies Business (2015-2020)11.6.5 Boehringer Ingelheim Recent Development11.7 AstraZeneca11.7.1 AstraZeneca Company Details11.7.2 AstraZeneca Business Overview11.7.3 AstraZeneca Precision Cancer Therapies Introduction11.7.4 AstraZeneca Revenue in Precision Cancer Therapies Business (2015-2020)11.7.5 AstraZeneca Recent Development11.8 Aveo Pharmaceuticals11.8.1 Aveo Pharmaceuticals Company Details11.8.2 Aveo Pharmaceuticals Business Overview11.8.3 Aveo Pharmaceuticals Precision Cancer Therapies Introduction11.8.4 Aveo Pharmaceuticals Revenue in Precision Cancer Therapies Business (2015-2020)11.8.5 Aveo Pharmaceuticals Recent Development 12 Analysts Viewpoints/Conclusions 13 Appendix13.1 Research Methodology13.1.1 Methodology/Research Approach13.1.2 Data Source13.2 Disclaimer13.3 Author Details

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Precision Cancer Therapies Market, Share, Application Analysis, Regional Outlook, Competitive Strategies & Forecast up to 2025 |, Abbott...

News briefing: Exelixis rolls the dice with 2 ADC deals tied to $35M cash upfronts; A rare antibiotic win for prominently-backed Spero – Endpoints…

Catalents Redwood Bioscience subsidiary is taking the lead role in developing ADCs for Exelixis $EXEL. In one of 2 deals announced this morning, Exelixis is turning to Catalent to do the discovery work on the ADCs, which will employ their SMARTag site-specific bioconjugation platform technology using antibodies out of the biotechs pipeline.

Catalent gets $10 million upfront to trigger the deal, with Exelixis holding worldwide development rights on anything it chooses.

In a separate ADC development deal, Exelixis is turning to NBE-Therapeutics for another pact that could steer more product candidates its way. In this deal Exelixis is paying $25 million upfront to get a 2-year alliance underway.

Exelixis is pursuing both internal drug discovery and external business development approaches to build a pipeline with the potential to make a difference for patients with cancer, saidPeter Lamb, the CSO at Exelixis. John Carroll

Back in the days when investors thought there was still good business to be made in antibiotics, Spero raised a fair bit of capital: a $30 million Series A and B led by Atlas Ventures, a $51.7 million Series C led by GV, and a $77 million IPO. In 2018, they received an up to $54 million contract with BARDA too.

The market for antibiotics has since soured, but that cash has allowed Spero to get through Phase III with its lead drug. And on Tuesday they announced positive results, showing in a 1,372-person study that their oral antibiotic tebipenem was non-inferior to the approved IV antibiotic ertapenem in treating patients with complicated urinary tract infections and acute pyelonephritis.

The drug, said trial investigator Keith Kaye, will give cUTI patients a new oral option after evolving microbiotic resistance had left them with only the IV drug.

Due to the increasing prevalence of antibiotic-resistant bacteria, many patients with cUTI now receive intravenous antibiotics as their only available treatment option, Kaye, who is also director of research in the division of infectious diseases at the University of Michigan Medical School, said in a statement. The much-anticipated data from this head-to-head comparison against an IV standard-of-care carbapenem antibiotic suggest that in many instances oral, outpatient treatment of these complicated bacterial infections is a viable option.

Spero CEO Ankit Mahadevia said it would be the first oral cUTI drug approved in 26 years. The company said it plans to start a rolling NDA and complete it by the second quarter of 2021.

An approval would be a major boon to any biotech, but when it comes to antibiotics, regulatory success doesnt necessarily translate into commercial success. Melinta and Achaoegen are well proof of that. Jason Mast

A UK-based biotech is buying outright a software developer in Brooklyn, NY, in order to bring artificial intelligence to its diagnostics tools.

APIS Assay Technologies announced the acquisition of Beogenomics on Friday, hoping to use data-mining processes to identify biomarker targets in oncology, as well as inflammatory, autoimmune and infectious diseases. The technology from Beogenomics, which has been developing both on-prem and secure cloud-based data analysis solutions, will help support the launch of a new proprietary service line. APIS mainly works in R&D and diagnostics, developing new tests for the prediction, prevention, and diagnosis of disease from discovery to regulatory approval.

The companys business model focuses on three aspects: biomarker diagnostics development, molecular diagnostic contract development, and applied bioinformatics. Max Gelman

Intravacc, a Dutch vaccines company, landed an up to $9.4 million contract from NIAID to develop a vaccine for enterovirus D68, a respiratory virus that can cause paralysis and has become increasingly common in America, Europe and Asia over the last few years.

The small biotech, which also has programs for RSV, gonorrhea and of course Covid-19, will develop an inactivated virus vaccine in Vero cells. The contract is for early product selection through Phase I. Jason Mast

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News briefing: Exelixis rolls the dice with 2 ADC deals tied to $35M cash upfronts; A rare antibiotic win for prominently-backed Spero - Endpoints...

COVID-19 Impact on Cell and Gene Therapy Market Research, Size, Growth And Trends 2020 to 2025: Keyplayer-Amgen Inc., bluebird bio, Inc. – Scientect

Chicago, United States:The global Cell and Gene Therapy Market report offers a complete research study that includes accurate estimations of market growth rate and size for the forecast period 2020-2025. It offers a broad analysis of market competition, regional expansion, and market segmentation by type, application, and geography supported by exact market figures. The all-inclusive market research report also offers Porters Five Forces Analysis and profiles some of the leading players of the global Cell and Gene Therapy Market. It sheds light on changing market dynamics and discusses about different growth drivers, market challenges and restraints, and trends and opportunities in detail. Interested parties are provided with market recommendations and business advice to ensure success in the global Cell and Gene Therapy Market.

Top Key players cited in the report:Amgen Inc., bluebird bio, Inc., Dendreon Pharmaceuticals LLC., Fibrocell Science, Inc., Human Stem Cells Institute, Kite Pharma, Inc., Kolon TissueGene, Inc., Novartis AG, Orchard Therapeutics plc., Organogenesis Holdings Inc., Pfizer, Inc., RENOVA THERAPEUTICS, .

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The final report will add the analysis of the Impact of Covid-19 in this report Cell and Gene Therapy Market

Cell and Gene Therapy Marketreports offers important insights which help the industry experts, product managers, CEOs, and business executives to draft their policies on various parameters including expansion, acquisition, and new product launch as well as analyzing and understanding the market trends.

Each segment of the global Cell and Gene Therapy market is extensively evaluated in the research study. The segmental analysis offered in the report pinpoints key opportunities available in the global Cell and Gene Therapy market through leading segments. The regional study of the global Cell and Gene Therapy market included in the report helps readers to gain a sound understanding of the development of different geographical markets in recent years and also going forth. We have provided a detailed study on the critical dynamics of the global Cell and Gene Therapy market, which include the market influence and market effect factors, drivers, challenges, restraints, trends, and prospects. The research study also includes other types of analysis such as qualitative and quantitative.

Global Cell and Gene Therapy Market: Competitive Rivalry

The chapter on company profiles studies the various companies operating in the global Cell and Gene Therapy market. It evaluates the financial outlooks of these companies, their research and development statuses, and their expansion strategies for the coming years. Analysts have also provided a detailed list of the strategic initiatives taken by the Cell and Gene Therapy market participants in the past few years to remain ahead of the competition.

Global Cell and Gene Therapy Market: Regional Segments

The chapter on regional segmentation details the regional aspects of the global Cell and Gene Therapy market. This chapter explains the regulatory framework that is likely to impact the overall market. It highlights the political scenario in the market and the anticipates its influence on the global Cell and Gene Therapy market.

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Report Highlights

Table of Contents

Report Overview:It includes six chapters, viz. research scope, major manufacturers covered, market segments by type, Cell and Gene Therapy market segments by application, study objectives, and years considered.

Global Growth Trends:There are three chapters included in this section, i.e. industry trends, the growth rate of key producers, and production analysis.

Cell and Gene Therapy Market Share by Manufacturer:Here, production, revenue, and price analysis by the manufacturer are included along with other chapters such as expansion plans and merger and acquisition, products offered by key manufacturers, and areas served and headquarters distribution.

Market Size by Type:It includes analysis of price, production value market share, and production market share by type.

Market Size by Application:This section includes Cell and Gene Therapy market consumption analysis by application.

Profiles of Manufacturers:Here, leading players of the global Cell and Gene Therapy market are studied based on sales area, key products, gross margin, revenue, price, and production.

Cell and Gene Therapy Market Value Chain and Sales Channel Analysis:It includes customer, distributor, Cell and Gene Therapy market value chain, and sales channel analysis.

Market Forecast Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

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About Us:Report Hive Research delivers strategic market research reports, statistical survey, and Industry analysis and forecast data on products and services, markets and companies. Our clientele ranges mix of United States Business Leaders, Government Organizations, SMEs, Individual and Start-ups, Management Consulting Firms, and Universities etc. Our library of 600,000+ market reports covers industries like Chemical, Healthcare, IT, Telecom, Semiconductor, etc. in the USA, Europe Middle East, Africa, Asia Pacific. We help in business decision-making on aspects such as market entry strategies, market sizing, market share analysis, sales and revenue, technology trends, competitive analysis, product portfolio and application analysis etc.

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COVID-19 Impact on Cell and Gene Therapy Market Research, Size, Growth And Trends 2020 to 2025: Keyplayer-Amgen Inc., bluebird bio, Inc. - Scientect

Grifols makes a $146M bet on a Stanford play in a controversial anti-aging field – Endpoints News

For the last couple of years, when it came to the vaguely vampiric field of young blood plasma transfusions, there was Alkahest and there was everyone else.

Since the field was briefly mocked onSilicon Valley in 2017, one startup charged $8,000 to $12,000 to pump the elderly with the plasma of young donors and one doctor pitched his clinical trial in a glitzy West Palm Beach gala where he reminded retirees they were likely to die soon, prompting FDAs chief Scott Gottlieb to warn such treatments were unproven and that some patients are being preyed upon by unscrupulous actors. But Alkahest, founded by a Genentech alumn and a Stanford neuroscientist, has promised to take a more measured, scientific approach to the still-fringe science, running phased clinical trials for their plasma-derived products and couching their press releases accordingly.

Now, the plasma giant Grifols is going all-in on their work. On Labor Day, the Spanish company announced they would buy out the remaining shares of the startup they didnt already own, spending $146 million for just over 50% of Alkahests stock.

Grifols is a roughly $15 billion company, so the buyout is not exactly a huge outlay for them. Nor is the nearly $300 million market value this deal places on Alkahest nearly enough to rank them among the industrys most valuable players. Still, the acquisition represents a major validation for a company and a young-blood field that has grown up largely on the fringes of the biomedical mainstream. And it amounts to a step forward for the anti-aging field more broadly, which has struggled to make gains despite significant big-name investment over the last few years.

The new deal is about four times what Grifols paid for the first 45% of Alkahests stock in 2015. The startup has relied on Grifols to collect plasma for its products.

We saw the promise of Alkahests understanding of aging when we made our first investment and entered into a collaboration agreement with them five years ago, Grifols CEO Vctor Grfols said in a statement. Now we see a wealth of plasma-derived and non-plasma therapeutic candidates identified by Alkahest that can significantly support the unmet needs of many diseases associated with aging.

Unlike, say, Ambrosia (the aforementioned $8,000 infusion company), Alkahest doesnt give plasma directly from young donors to the elderly. Instead, it has worked since 2014 on mapping the proteins in plasma and distilling a cocktail of roughly 400 different types of proteins it believes can make a difference in treating Alzheimers and other CNS disorders. Thats a ton of proteins compared to most biologics, which are made of 1, but it amounts to just 3% of whats in plasma. The Long Island Ice Tea of a lead drug is known as GRF-6019. The science is based on work from Stanford neuroscientist Tony Wyss-Coray, which was spun out by former Genentech scientist Karoly Nikolich.

So far, evidence for effectiveness remains scant. Last year, the company said that, in a 47-person Phase II study, patients with mild to moderate Alzheimers maintained their level of cognition for 6 months. But they still have yet to release data from that study and while this is certainly a new approach to the evasive disease, many other therapies have failed after showing promise in early trials. Their Phase I study, published inJAMA, showed little change in cognitive performance tests among 18 Alzheimers patients, although it was only powered for safety.

Alkahest is also testing GRF-6019 in severe Alzheimers patients, an oft-overlooked population, and said recently that it proved safe in a 26-person pilot study. They have studies ongoing in Parkinsons, age-related macular degeneration, and patients with end-stage renal disease and cognitive impairment.

Outside of plasma, the company is also developing an oral drug aimed at another anti-aging target: Eotaxin. Its an immunomodulatory protein, they say, that increases with age. Alkahest is in Phase II studies to see if blocking it can curb Parkinsons and AMD.

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Grifols makes a $146M bet on a Stanford play in a controversial anti-aging field - Endpoints News

New California law gives consumers more agency over data they share with genetic testing firms – Business Insider

California lawmakers passed a law that allows consumers to revoke consent for genetic testing companies like 23andMe and Ancestry to use their data, mandating the companies to destroy the DNA samples within 30 days, STAT reported in its weeklynewsletter.

California law boosts genetic data privacy. Business Insider Intelligence

For context, direct-to-consumer (D2C) DNA testing firms often give customers the opportunity to opt into research by consenting to pass along their samples: For instance, 8 million of 23andMe's network of 10 million users haveopted in to participate in research. States are taking the helm at passing D2C genetic testing regulation, while federal lawmakers remain mumcreating a patchworked legal landscape for genetic testing companies to operate in.

Privacy laws have yet to be enacted on a federal level, so state lawmakers are stepping in: Florida recently passedlegislation prohibiting insurance companies from accessing members' genetic insights, which could impact the type and cost of coverage. But as states take charge putting forth their own laws, genetic testing companies will be faced with new obstacles, and it's unclear how they'll navigate adhering to the changing legal ecosystem.

In reference to the new law passed in California, Justin Yedor, a Los Angeles-based data privacy attorney, wascitedin Bloomberg asking, "are [D2C companies] going to provide these rights strictly for Californians or are they going to extend them to all consumers regardless of jurisdiction?" Contending with new rules passed on a state-by-state basis could cause hangups in operations, exacerbating the alreadysofteningD2C genetic testing market.

While a hodgepodge of legislation across the US will be a hurdle, increased privacy laws could assuage consumers' fears and translate into more sales.In a recent YourDNA survey,40%of consumers who had never taken a DNA test cited privacy concerns as the driving reason for why they've shied away. But if companies are transparent about granting consumers more autonomy over their data and how it's handled, they may be more likely to take the plunge.

Still, we think high-flying genetic testing firms will lean more heavily on their healthcare-focused initiatives as they navigate the shifting D2C realm: Some DNA testing firms likeColorandYouScriptthe latter of which is now owned by Invitaeare powering hospitals' precision medicine initiatives, for example.

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New California law gives consumers more agency over data they share with genetic testing firms - Business Insider

Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy -…

CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C written response only meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA), to obtain OTATs concurrence on the commencement of its next clinical trial for SRP-9001 using commercial process material. SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) is Sareptas investigational gene transfer therapy for the treatment of Duchenne muscular dystrophy.

Among other items, OTAT has requested that Sarepta utilize an additional potency assay for release of SRP-9001 commercial process material prior to dosing in a clinical study. Sarepta has several existing assays and data that it believes could be employed in response to OTATs request. However, additional dialogue with the Agency is required to determine the acceptability of the potency assay approach.

We look forward to working with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study. We will provide further updates as we are able, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. Every day, thousands of children degenerate from the irreversible damage caused by Duchenne muscular dystrophy. It is for that reason that we will work relentlessly with the Division to satisfy any requests of OTAT and continue the advancement of a potentially transformative therapy for these patients.

About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin)SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sareptas belief that its existing assays and data could be employed in response to OTATs request; the acceptability of Sareptas potency assay approach by the FDA; our plan to work with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study; and the potential of SRP-9001 to be a transformative therapy for DMD patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: delays in the commencement of Sareptas next clinical study for SRP-9001 could delay, prevent or limit our ability to gain regulatory approval for SRP-9001; any inability to complete successfully clinical development could result in additional costs to Sarepta or impair Sareptas ability to generate revenues from product sales, regulatory and commercialization milestones and royalties; SRP-9001 may not result in a viable treatment suitable for commercialization due to a variety of reasons, including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy -...

$14.6M Grant to Explore a Therapy to Control HIV Without Meds – Cancer Health Treatment News

In nearly 40 years of the HIV epidemic, only two people have likely been cured of the virus. Both scenarios resulted from stem cell transplants needed to fight blood cancers such as leukemia. Inspired by these two cases, a team of scientists is studying a multipronged way to potentially control HIV without medication. It involves two different genetic alterations of immune cells and with a safer method of stem cell transplants, also referred to as bone marrow transplants, a procedure that is generally toxic and dangerous.

The research is being funded by a five-year $14.6 million grant from the National Institutes of Health. The scientists coleading the preclinical studies are Paula Cannon, PhD, a distinguished professor of molecular microbiology and immunology at the Keck School of Medicine of the University of Southern California, and Hans-Peter Kiem, MD, PhD, who directs the stem cell and gene therapy program at the Fred Hutchinson Cancer Research Center, also known as Fred Hutch. According to a Keck School of Medicine press release, the two other main partners are David Scadden, MD, a bone marrow transplant specialist and professor at Harvard University and the Harvard Stem Cell Institute, and the biotechnology company Magenta Therapeutics.

In the HIV cure scenariosinvolving the so-called Berlin and London patientsboth men received stem cell transplants from donors with a natural genetic mutation that made them resistant to HIV. Specifically, their genes resulted in immune cells that lack CCR5 receptors on their surface (HIV latches onto these receptors to infect cells). Unfortunately, this method isnt viable for the nearly 38 million people worldwide living with HIV. Not only is it expensive, toxic and riskyit involves wiping out the patients immune system and replacing it with the new immune cellsbut it also requires matched donors who are CCR5 negative. According to the press release, about 1% of the population have this mutation.

With funding from this new grant, researchers hope to overcome these challenges in several ways. First, Cannon has already developed a gene-editing method to remove the CCR5 receptors from a patients own stem cells. She now hopes to further genetically engineer stem cells so they release antibodies that block HIV.

Our engineered cells will be good neighbors, Cannon said in the press release. They secrete these protective molecules so that other cells, even if they arent engineered to be CCR5 negative, have some chance of being protected.

Fred Hutchs Kiem will use CAR-T therapya new method of genetically modifying immune cells that is emerging out of cancer researchwith the goal of creating T cells that attack HIV-infected cells.

In addition, other scientists involved in the federal grant aim to develop less toxic methods of bone marrow transplantationfor example, by reducing the amount of chemotherapy required and speeding up the process of creating the new immune system.

The research finding could translate to other illnesses, such as cancer, sickle cell anemia and autoimmune disorders.

A home run would be that we completely cure people of HIV, Cannon said. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

For the latest on the cure cases, see Famed London Man Probably Cured of HIV from earlier this year. And in related news, see $14M Federal Grant to Research CAR-T Gene Therapy to Cure HIV.

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$14.6M Grant to Explore a Therapy to Control HIV Without Meds - Cancer Health Treatment News

Gyroscope Therapeutics Announces Appointment of Sean Bohen to the Board of Directors – Business Wire

LONDON--(BUSINESS WIRE)--Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced that biotech industry veteran Sean Bohen, M.D., Ph.D., has been appointed to the Gyroscope Board of Directors, effective immediately. Dr. Bohen will also serve as Chair of the Boards Research and Development Committee. Dr. Bohen brings more than 30 years of experience in the discovery and development of new medicines. Over the course of his career, he has made significant contributions to the early- and late-stage development of numerous FDA-approved drugs.

Sean brings a wealth of experience to our board in researching potential new medicines and successfully bringing them to patients in need. He also brings expertise and knowledge in helping companies build impressive R&D and commercial units side by side. We are excited to work with him as we advance Gyroscopes programmes, said Khurem Farooq, Chief Executive Officer.

Dr. Bohen currently serves on the board of directors of Altrubio, Inc. Previously, Dr. Bohen was the Chief Medical Officer and Executive Vice President, Global Medicines Development, AstraZeneca LP, where he was responsible for AstraZenecas worldwide product development and clinical programmes. Prior to joining AstraZeneca, he was Senior Vice President of Genentech Early Development (gRED), where he led preclinical and clinical development programmes to deliver pivotal trial-ready drug candidates to Genentechs late-stage development pipeline. During this time, he also served as a clinical instructor at the Stanford University School of Medicine. He received both his Ph.D. in Biochemistry and M.D. from the University of California, San Francisco, and has been board certified in Internal Medicine and Medical Oncology.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing and delivering gene therapy beyond rare disease to treat a leading cause of blindness, dry age-related macular degeneration. Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and a Phase II clinical trial called EXPLORE.

Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

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Gyroscope Therapeutics Announces Appointment of Sean Bohen to the Board of Directors - Business Wire

Gene Therapy Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 – The Daily Chronicle

New Jersey, United States, The Gene Therapy Market report 2020 provides a detailed impression, describe the product industry scope and the market expanded insights and forecasts up to 2027. It shows market data according to industry drivers, restraints and opportunities, analyzes the market status, the industry share, size, future Trends and growth rate of the market. The Gene Therapy Market report is categorized by application, end user, technology, product / service types, and other, as well as by region. In addition, the report includes the calculated expected CAGR of chitosan acetate-market derivative from the earlier records of the Gene Therapy Market, and current market trends, which are organized with future developments.

Gene Therapy Market was valued at USD 3.69 Billion in 2019 and is projected to reach USD 24.78 Billion by 2027, growing at a CAGR of 26.9% from 2020 to 2027.

Gene Therapy Market, By Product

Viral Vectorso Adeno-associated virus vectorso Retroviral vectors Gammaretroviral vectors Lentiviral vectorso Other viral vectors (herpes simplex and adenoviral vectors) Non-viral Vectorso Oligonucleotideso Other non-viral vectors (plasmids and RNAi)

Gene Therapy Market, By Indication

Neurological Diseases Cancer Hepatological Diseases Duchenne Muscular Dystrophy Other Indications

Gene Therapy Market, By Delivery Method

Ex vivo In vivo

The report provides detailed coverage of the Gene Therapy Market, including structure, definitions, applications, and Industry Chain classifications. The Gene Therapy Market analysis is provided for the international markets including development trends, competitive landscape analysis, investment plan, business strategy, opportunities and development status of key regions. Development policies and plans are discussed and manufacturing processes and cost structures analyzed. This report also includes information on import / export consumption, supply and demand, costs, industry share, policy, Price, Sales and gross margins.

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Gene Therapy Market forecast up to 2027, with information such as company profiles, product picture and specification, capacity production, price, cost, revenue, and contact information. Upstream raw materials and equipment as well as downstream demand analyses are also carried out. The Gene Therapy Market size, development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and general research results are offered.

The Gene Therapy Market was created on the basis of an in-depth market analysis with contributions from industry experts. The report covers the growth prospects in the coming years and the discussion of the main providers.

To understand how the effects of COVID-19 are addressed in this report. A sample copy of the report is available at https://www.verifiedmarketresearch.com/product/gene-therapy-market/?utm_source=TDC&utm_medium=001

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Gene Therapy Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 - The Daily Chronicle

Prevail Therapeutics Announces Appointment of William H. Carson, MD, to Board of Directors – BioSpace

NEW YORK, Sept. 10, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc.(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the appointment ofWilliam H. Carson, M.D., to its Board of Directors.

Bill knows how to drive the development of innovative treatments including drugs for a wide range of diseases that affect the brain from concept to commercialization, saidFrancois Nader, M.D., Non-Executive Chairman of Prevails Board of Directors. We are thrilled that he will bring that expertise to bear at Prevail as we continue to develop gene therapies for patients suffering from devastating neurodegenerative diseases, including Parkinsons disease, frontotemporal dementia and Gaucher disease.

Dr. Carson was most recently the President and CEO of Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC), leading the development and regulatory approvals of Otsukas global compounds including Abilify Maintena, Rexulti, Samsca and Jynarque. He also oversaw the approval of Abilify MyCite, the first FDA-approved digital medicine. Dr. Carson joined Otsuka as Vice President of the Princeton Aripiprazole Unit in 2002, establishing the companys presence there. Before joining Otsuka, he rose through the ranks in the CNS Research and Development department at Bristol-Myers Squibb. Prior to joining the pharmaceutical industry, Dr. Carson, a board-certified psychiatrist, was an Associate Professor in the Department of Psychiatry and Behavioral Sciences at the Medical University of South Carolina.

Dr. Carson currently serves as Chairman of the Board of Directors of OPDC, and is also the Chairman of the Board of the Sozosei Foundation, a newly established Otsuka charitable organization with a main focus on decriminalization of mental illness. He is a Board Member of Excision Biotherapeutics and Trustee of the non-profit Internet2. He is Board Chair Emeritus of the Sphinx Organization, which advocates for parity and inclusion in the arts. Dr. Carson holds an A.B. Degree from Harvard College and a M.D. degree from Case Western Reserve University. He is a Distinguished Fellow of the American Psychiatric Association, the National Medical Association and the Executive Leadership Council. Dr. Carson was named to Savoy Magazines 2018 list of the Top 100 Most Influential Blacks in Corporate America.

Dr. Carson is an outstanding and important addition to our Board of Directors, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. I know that he will bring both deep medical expertise and compassion for patients to the role, as well as his stellar track record of advancing therapies through clinical development, FDA approval and commercialization.

I am honored to join Prevail at this exciting time for the company, said Dr. Carson. Prevails programs are moving forward quickly, and promising data are beginning to emerge. I am looking forward to helping the company continue to advance its mission of developing potentially disease-modifying treatments for patients with neurodegenerative disorders.

AboutPrevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinsons disease withGBA1mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia withGRNmutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr.Asa Abeliovichin 2017, through a collaborative effort withThe Silverstein Foundationfor Parkinsons with GBA and OrbiMed, and is headquartered inNewYork, NY.

Forward-Looking Statements Related to Prevail

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevails ability to develop potentially disease-modifying treatments for patients with neurodegenerative disorders. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; initial data or other preliminary analyses or results of early clinical trials may not be predictive of final trial results or of the results of later clinical trials; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on Prevails business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with theSecurities and Exchange Commission(SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period endedJune 30, 2020, filed with theSEConAugust 11, 2020, and its other documents subsequently filed with or furnished to theSEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Mary CarmichaelTenBridge Communicationsmary@tenbridgecommunications.com617-413-3543

Investor Contact:investors@prevailtherapeutics.com

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Prevail Therapeutics Announces Appointment of William H. Carson, MD, to Board of Directors - BioSpace

Reasons Industries to Thrive Post-Pandemic! Gene Therapy for Age related Macular Degeneration Market Report xyz Answers it Analysis by Key Companies…

Global Coronavirus pandemic has impacted all industries across the globe, Gene Therapy for Age related Macular Degeneration market being no exception. As Global economy heads towards major recession post 2009 crisis, Cognitive Market Research has published a recent study which meticulously studies impact of this crisis on Global Gene Therapy for Age related Macular Degeneration market and suggests possible measures to curtail them. This press release is a snapshot of research study and further information can be gathered by accessing complete report. To Contact Research Advisor Mail us @ [emailprotected] or call us on +1-312-376-8303.

The research report on global Gene Therapy for Age related Macular Degeneration market as well as industry is a detailed study that provides detailed information of major key players, product types & applications/end-users; historical figures, region analysis, market drivers/opportunities & restraints forecast scenarios, strategic planning, and a precise section for the effect of Covid-19 on the market. Our research analysts intensively determine the significant outlook of the global Gene Therapy for Age related Macular Degeneration market study with regard to primary & secondary data and they have represented it in the form of graphs, pie charts, tables & other pictorial representations for better understanding.

Subretinal, Intravitreal, Unspecified are some of the key types of market. All the type segments have been analyzed based on present and future trends and the market is estimated from 2020 to 2027. Based on the application segment, the global market can be classified into Monotherapy, Combination Therapy . The analysis of application segment will help to analyze the demand for market across different end-use industries.

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Amid the COVID-19 pandemic, the industry is witnessing a major change in operations.Some of the key players include RetroSense Therapeutics, REGENXBIO, AGTC . key players are changing their recruitment practices to comply with the social distancing norms enforced across several regions to mitigate the risk of infection. Additionally, companies are emphasizing on using advanced recruiting solutions and digital assets to avoid in-person meetings. Advanced technologies and manufacturing process are expected to play a decisive role in influencing the competitiveness of the market players.

Regional Analysis for Gene Therapy for Age related Macular Degeneration Market:North America (United States, Canada)Europe (Germany, Spain, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, India, Australia, and South Korea)Latin America (Brazil, Mexico, etc.)The Middle East and Africa (GCC and South Africa)

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NOTE: Whole world is experiencing the impact of Covid-19 pandemic due to its increasing spread hence, the report comprises of an up to date scenario of the Gene Therapy for Age related Macular Degeneration market report. Research analyst team of our company is understanding & reviewing the Covid19 Impact on Market and all the necessary areas of the market that have been altered due to the change caused by Covid19 impact. Get in touch with us for more precise/in-depth information of the Gene Therapy for Age related Macular Degeneration market.

Any query? Enquire Here For Discount (COVID-19 Impact Analysis Updated Sample): Click Here>Download Sample Report of Gene Therapy for Age related Macular Degeneration Market Report 2020 (Coronavirus Impact Analysis on Gene Therapy for Age related Macular Degeneration Market)

At the end of May, many states began lifting lockdown restrictions and reopening in order to revive their economies, despite warnings that it was still too early. As a result, by mid-July, around 33 states were reporting higher rates of new cases compared to the previous week with only three states reporting declining rates. Due to this Covid-19 pandemic, there has been disruptions in the supply chain which have made end-use businesses realize destructive in the manufacturing and business process. During this lockdown period, the plastic packaging helps the products to have longer shelf life as the public would not be able to buy new replacements for the expired products because most of the production units are closed.

About Us:Cognitive Market Research is one of the finest and most efficient Market Research and Consulting firm. The company strives to provide research studies which include syndicate research, customized research, round the clock assistance service, monthly subscription services, and consulting services to our clients. We focus on making sure that based on our reports, our clients are enabled to make most vital business decisions in easiest and yet effective way. Hence, we are committed to delivering them outcomes from market intelligence studies which are based on relevant and fact-based research across the global market.Contact Us: +1-312-376-8303Email: [emailprotected]Web: https://www.cognitivemarketresearch.com/

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Reasons Industries to Thrive Post-Pandemic! Gene Therapy for Age related Macular Degeneration Market Report xyz Answers it Analysis by Key Companies...

Md. Labs Developing Vaccines and Therapeutics, Attracting Federal Dollars – Josh Kurtz

A 2016 decision to link the research labs at the University of Maryland College Park with those at the University of Maryland at Baltimore is paying dividends in the competition for federal COVID-19 research dollars, a top education official told a science advisory panel on Wednesday.

The decision was a spinoff of the University of Maryland Strategic Partnership Act, said Laurie Ellen Locasio, vice president for research at the two institutions.

Now well be ranked together by the [National Science Foundation] in the U.S. rankings of research universities and together this makes us [an] over $1 billion public research enterprise, she told the Maryland Life Sciences Advisory Board.

With a multi-billion dollar research powerhouse, the privately-funded Johns Hopkins University, also operating here, Maryland is unusually well-positioned, Locasio said.

Very few states in the country have the distinction of having two research universities with over a billion dollars in research spending per year. Weve really shown up as central to this pandemic.

Martin Rosendale, the CEO of the Maryland Tech Council, said there are so many therapeutics and vaccines in development that keeping up with them all is a daunting task.

Many firms adopted a drop everything approach when the scope of the coronavirus challenge started to become apparent, he said.

We just saw a lot of amazing thing happening here in Maryland so many companies were pivoting immediately to support the response to the pandemic, he said. They were basically dropping their other work and on their own dime spending their own money evaluating their technologies, their platforms, how they could apply them to COVID-19.

Many of the big players have grabbed global headlines for their work on a possible vaccine. But smaller companies are making potentially valuable strides as well, Commerce Secretary Kelly M. Schulz told the panel.

Other firms are working to develop COVID tests that are faster and more reliable.

The Tech Council formed the Maryland COVID-19 Coalition to bring companies together, to make sure they knew who each other were and begin a conversation, Rosendale said. Twenty-five firms were part of the coalitions first call; 40 were on the second.

Chris P. Austin, director of the National Center for Advancing Translation Sciences at NIH, told the panel that the federal government has pumped more than $20 billion into COVID research.

Of that, we know that over $3 billion is coming here to Maryland, Rosendale said. Thats a testament to the amazing vaccine and cell-and-gene therapy industries that have grown up here.

If foundation and other non-government funds are added to the public dollars, the number is well over $4 billion, he added.

To play off the capital regions strengths in biotech, a group of industry leaders in Maryland, Washington, D.C., and Virginia is organizing a pandemic and bio-defense center to help countries around the globe guard against future pandemics.

Because of the prominence of whats going on with COVID-19 development in our region especially within Maryland with vaccine, diagnostics, and therapeutic research we are sort of the epicenter in the world right now, said Richard A. Bendis, president and CEO of BioHealth Innovation, Inc. in Rockville.

People are starting to take notice.

Schulz said the Department of Commerce paused its planned marketing efforts in the spring to focus on the pandemic, but now is ready to gear back up again with an Innovation Uncovered campaign.

Were going back to where we wanted to be at the beginning of the year, which was to push the bio- and the life sciences worlds out, because we have heard from [company executives] that Maryland needs to be seen as a strong, tight ecosystem for this industry, she said.

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Md. Labs Developing Vaccines and Therapeutics, Attracting Federal Dollars - Josh Kurtz

Gene Therapy in Oncology Market Research Report By Application, Type, Key Players, Region and Forecast to 2029 – News Monitoring

Good Growth Opportunities In Global Gene Therapy in Oncology Market: Distinguished Technology Development with Major Production Goals Analysis by 2029

According to the latest market analysis report Gene Therapy in Oncology Market in-depth study and complete information about the market size, market offers and market dynamics. Global Gene Therapy in Oncology Market provides different sections and sub-sections based on separation by type, application, key players, and end-user, segments, developments, topographical areas of this market. This extensive report also highlights key insights on the factors that drive the growth of the market as well as key challenges that are required to Gene Therapy in Oncology growth in the projection period. The report presents a full analysis of the key factors such as developments, trends, predictions, drivers, and business growth, developing trends, competitive landscape analysis. Development strategies and policies are presented as well as manufacturing processes and value structures are also examined presented in the analytical surveying report, besides the cost structures and production methods.

For Better Understanding, Download Sample PDF of Gene Therapy in Oncology Market Research Report @https://market.us/report/gene-therapy-in-oncology-market/request-sample

Global Gene Therapy in Oncology Market gives a comprehensive overview, the key features that are required to improve the growth of the market in the coming future. Gene Therapy in Oncology Market presenting information such as supply and demand situation, the competitive scenario, and the objections for market growth, market opportunities, and the threats faced by Top key players Bristol-Myers Squibb, Cold Genesys, Advantagene, Amgen, AstraZeneca, Bio-Path Holdings, CRISPR Therapeutics, Editas Medicine, Geron Corp, Idera Pharmaceuticals, Intellia Therapeutics, Johnson & Johnson, Marsala Biotech, Merck, . Many of the market players are interested in Gene Therapy in Oncology Market companies, raw material suppliers, machine suppliers, end users, traders, distributors. Building opportunities in this Gene Therapy in Oncology market and the growing progressions in the Gene Therapy in Oncology Market.It has been giving a clear understanding of primary and secondary research techniques and they are now intended towards collaborating accurate and exact data. The data gathered to structure this report is based on the data collection modules with large sample quantities.

The detailed segments and sub-segment of the market are explained below:

Top growing factors by Key Companies like:

Bristol-Myers Squibb, Advantagene, Amgen, Marsala Biotech, Idera Pharmaceuticals, Merck, Geron Corp, Intellia Therapeutics, , Editas Medicine, Johnson & Johnson, CRISPR Therapeutics, AstraZeneca, Cold Genesys and Bio-Path Holdings

Current Market Status, Trends, Types:

Ex Vivo, In Vivo

Review of Market Growth, Future Prospects, and Applications:

Hospitals, Diagnostics Centers, Research Institutes

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Global Gene Therapy in Oncology Market: Regional Segmentation

1.North America (United States, Mexico, and Canada)

2.Europe (France, UK, Germany, Russia, and Italy)

3.Asia-Pacific (Korea, India, China, Japan, and Southeast Asia)

4.South America (Brazil, Argentina, Colombia, etc.)

5.Middle East and Africa (UAE, Egypt, Saudi Arabia, Nigeria, and South Africa)

Research Objectives Of Gene Therapy in Oncology Market Report:

A comprehensive overview of the market share and growth rate by type, application.

A complete analysis of the market opportunities, product scope, market risk, market driving force.

Analyze the top companies of Gene Therapy in Oncology Market Industry, with sales, revenue, and price

The report presents a forward-looking prospect on the global gene therapy in oncology market past data, status, and expected forecast, product, revenue, consumption.

It helps to recognize rising trends, drivers, growth influencing factors in global gene therapy in oncology market and regions.

It helps to prepare Marketing Policies by understanding the rising trends developing and improving gene therapy in oncology market development.

It supports to understand competitive developments such as developments, acquisitions, agreements, and new product launches in the gene therapy in oncology market.

*Please contact us for any specific customized requirements or changes you want and we will offer you the report as your requirement.

Browse Full Report withMore Professional and Technical insights including COVID-19 Impact:https://market.us/report/gene-therapy-in-oncology-market/

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Gene Therapy in Oncology Market Research Report By Application, Type, Key Players, Region and Forecast to 2029 - News Monitoring

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