Archive for the ‘Gene Therapy Research’ Category

The coronavirus pandemic has triggered a race for disruptive innovation in diagnostics, new therapies and novel vaccines, but the global life sciences industry was already expected to reach more than $2 trillion in gross value by 2023, up from $1.6 trillion before the crisis.

The pharmaceutical sector has long been attractive for family office investors, either directly or as part of their diversified portfolio. More than a quarter (27%) of families surveyed by Campden Wealth with UBS for The Global Family Office Report 2019 said they directly invested in health care and social assistance. It was the fourth most popular sector for families behind technology (49%), real estate and rental/leasing (42%) and finance and insurance (30%).

Dr Annalisa Jenkins MBBS, MRCP is a life sciences thought leader with more than 25 years of biopharmaceutical industry experience. The former surgeon lieutenant commander in the British Royal Navy during the Gulf Warsaid new entrepreneurial healthcare and biotech investment opportunities were opening for family investors from the disruptive revolutions in technology, stem cell therapy and mental health.

In an interview held before the coronavirus outbreak, the Chair of the Court of the London School of Hygiene and Tropical Medicine at the University of London, and chair or board member of Cellmedica, Vium and Cocoon Biotech, among others, tells CampdenFB which areas of investment she is personally interested in, the innovative trends she is seeing and what family investors need to know before entering the sector.

What is your outlook for investments in 2020?

Im hugely optimistic moving into the next decade. Whilst I think that one can always recognise that the geopolitical environment that we now operate in has markedly evolved in the last few years, whether that be the notion of risk, uncertainty, unpredictability, theres no doubt that has significantly evolved. Some of the driving factors behind that include global leadership, the climate evolution were living through, but ultimately the world of investment and the opportunities continue to be exciting.

When one looks across the field of health and innovation, were living in the so-called Fourth Industrial Revolution, and there is probably no sector that exemplarises that more than the health tech and life science sector. Today I would say the innovation coming out of our academic universities is as broad and as advanced as I have seen in the last 30 years of my career. I think the ability to access capital is just remarkable on a global basis, there are trillions of dollars that are out there in different parts of the economy, looking for innovation and looking for leaders to invest in. And, of course, we have a technology revolution that is ongoing, particularly driven by our ability to generate, curate and process data and deliver insights which are going to transform how we access and deliver healthcare and think about keeping people on a global basis well and healthy for longer and deliver improved therapeutics to reduce suffering.

I dont think I could be really more optimistic entering this new decade when it comes to thinking about the combination of equity with innovation and disruption and the evolution of the world I operate in.

What are the headwinds for investments in healthcare and life science?

The biggest concerns are around the well-publicised debates on pricing of technologies, particularly in the US, and how to ensure every individual can get equal access to health. Those two areas will continue to be debated and drive short-term cyclical uncertainty. There is no doubt global leadership, and that includes the US, recognise that for stable societies, access to universal healthcare and the ability of populations to be happy, healthy and flourishing lies as the centre of their political ambition.

Which healthcare investments are you excited about?

The cross-cutting themes that I am particularly excited about when it comes to technology are the ability for machine-learning and AI [artificial intelligence] to generate algorithms and therefore products that can be placed into the hands of individuals for them to manage, track and improve their health.

For example, there are products coming along in the area of mental health and wellness where individuals increasingly in the future will be able to access through their mobiles programmes and support services which will help them track their mood and maintain their wellness. We know that many diseases, for example, of the gastrointestinal system like irritable bowel syndrome, are markedly impacted by mental health and wellbeing. Interventions such as digital therapeutics, such as apps that are regulated and provided as therapeutics, are set to transform the way these diseases are managed. I am consistently looking for technologies that can be placed into the hands of individuals for self-directed and personalised prevention and care management.

Secondly, the ability of technology to improve the way we plan and conduct research and development on a global basis truly again going to be disrupted in the future by our ability to get large datasets in an ethical way to translate that into pre-clinical discoveries of new therapeutics. And then in the clinical setting to conduct novel clinical trials and to really change the way we bring new therapies to market, like new therapeutics, medical devices, diagnostics, biochemical markers across the board, so I am very interested in companies that are pursuing that.

Thirdly, the area of how do we optimise healthcare utilisation, the efficiency and the effectiveness, whether that be here in the UK in the NHS [National Health Service] or globally. Its absolutely clear that if we want to ensure affordable access to the top quality healthcare and outcomes for populations, were going to need embrace digital innovations, so companies that operate in that space are very attractive.

Those are the cross-cutting themes. If I look at the verticals, I would say to you, of course, there are very good investments to be made in the area of cancer and oncology. However, there is a marked amount of money in that space, its a little bit overpopulated. So for family offices and alternate sources of capital I would not be placing my money there at this point. I would be looking at counter cyclical trends, so areas that are perhaps now emerging. Areas such as womens health, otherwise known as Femtech, clearly now emerging as an area for investment and has been systematically underinvested in over the last 10-15 years. Cardiovascular disease continues to be the biggest killer and cause of illness on a global basis and yet it really hasnt received a lot of attention in investment. Were now seeing a wave of innovations and were starting to see more about the biology of heart disease, stroke and diabetes so again I would see that as an area of interest [as is] stem cells in therapeutics across a whole spectrum of diseases. Im particularly interested in Type 1 diabetes and the ability to generate beta cells for the production of insulin in patients with Type 1 diabetes. We are starting to see companies emerging and exploring that space so really stem cells and the cell therapy space, along with gene therapy is very interesting.

For family offices, the whole area of mental health and wellness is enormous and growing. The reason is its now becoming apparent people are talking about mental health and wellness in a way thats reducing stigma and then allowing money to flow into this space. How we deal with the human brain and preserving the health and wellness of the brain, both from an emotional and physical point of view, whether that be the prevention of degenerative brain disorders like dementia or the management of anxiety and all the related diseases known as depression, is a very interesting space for investment and its where I am investing in a lot.

How can family offices enter and succeed in the healthcare and biotech investment market?

Its extremely difficult as a family office to make an initial move into the sector, largely because its extremely complicated and it really rather depends on what the investment goals are and the returns that are desired. The sector is highly fragmented and family offices have traditionally and largely invested in the more sustainable, long-term world of pharma as part of a general equity portfolio. Were talking about a very different sector which is largely based on entrepreneurship, a lot of risk and deep science, so I think the first thing to say is that family offices, before moving into or when in this space, should ensure they have the access to the right expertise around the table. They can gain that either through their own networks of advisers or by participating through investments in a fund.

Often the best way to get started is to find a fund that meets the investment criteria of a family office and to start to invest through that. Not through a traditional VC fund, there are number of funds coming together with the sole purpose of accessing family offices, sovereign wealth funds, alternate sources of capital and then investing through that because its really important in the risky healthcare and life science sector to have a portfolio view that is very strategically put together. You have to be prepared to fail and to be prepared to be a long-term patient investor thats willing to along five-to-seven years for the ride.

Read more:
Dr Annalisa Jenkins on healthcare and biotech investments for family offices - Campden FB

A team at the Case Western Reserve University has been awarded up to $3.7 million from the National Heart, Lung, and Blood Institute (NHLBI) to conduct early studies of emerging gene therapies for sickle cell disease (SCD).

Led by Umut Gurkan,PhD, the team will examine blood samples collected from SCD patients before and after they receive gene therapies, and test them for improvements in red blood cells.

These potential gene therapies work by modifying a patients own hematopoietic stem cells, which generate red and other blood cells. Then, the modified stem cells are given back to the patient via a bone marrow transplant, which overcomes the difficulty in finding matched donors in those with SCD.

Patients will receive the therapies over the next two years as part of clinical trials conducted at leading U.S research universities and hospitals, includingStanford University, the University of California San Francisco, Emory University, the University of North Carolina, and Albert Einstein College of Medicine.

The overall goal is to make genetic therapies for SCD available within five to 10 years.

The big-picture potential here is to test whether this is dream or reality when it comes to gene therapy curing sickle cell, Gurkan said in a press release. We dont know the answers yet, but we have to ask whether these gene therapies are safe and effective in alleviating the symptoms and curing the disease and if we have the right tools.

Due to a mutation in the HBBgene, red blood cells of SCD patients acquire an abnormal and more rigid shape, while also becoming stickier than normal. This contributes to the formation of clogs that prevent or slow blood flow in small vessels, depriving tissues of oxygen.

Using a kind of lab-on-a-chip approach, researchers essentially mirror the tiny capillaries of the human body, which allows them to investigate how red blood cells move in these engineered capillaries.

The Case team will investigate if the gene therapies improve blood flow and test for improvements in red blood cell stickiness, as well as density, and shape.

If a curative therapy is successful and effective, we should see a significant improvement in these vital properties of blood, Gurkan said. Essentially we would like to objectively and quantitatively assess how well the blood cells flow in tiny capillaries after a gene-based therapy.

According to Gurkan, Case University has been playing an important role in the development of these new blood tests, which could help identify patients who respond to a given therapy early on.

As the genetic cure for sickle cell becomes a clinical reality, longitudinal, simple and accurate assessment and control through the tests that Dr. Gurkan has developed becomes ideal and opens up this new treatment to patients across the world, said Stanton Gerson, MD, director of the Case Comprehensive Cancer Center.

Our team is committed to making these new blood tests available for translation on global scale in both high- and low-resource settings, Gurkan said.

The work at Case will be conducted in collaboration with researchers at multiple other institutions, which, in addition to the centers conducting the clinical studies, include University Hospitals Rainbow Babies and Childrens Hospital, Childrens Healthcare of Atlanta, and Childrens Hospital of Montefiore.

Success in SCD could pave the way for similar benefits in other genetic diseases, which is the goal of the National Institutes of Health (NIH).

The reason the NIH is so focused on curing SCD is that it is the poster child for gene-editing efforts, said Gurkan. If we can prove that we can cure an inherited mutation like SCD effectively and safely, then you convince the funders and the public that it is worth the expense and the effort to go after more complex inherited diseases which are less understood.

View original post here:
Case Researchers Awarded $3.7 Million to Test Emerging SCD Gene... - Sickle Cell Anemia News

Hackers leak data related to WHO, Wuhan lab and Gates Foundation

A common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients, say researchers.

Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight.

Current treatments include either eye drops, laser or surgery, all of which have limitations and disadvantages. "At present, there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early," said study researcher Dr Colin Chu from the University of Bristol in the UK.

For the findings, published in the journal Molecular Therapy, the research team tested a new approach that could provide additional treatment options and benefits. The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.

The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye. Using the latest gene-editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure.

"We hope to advance towards clinical trials for this new treatment in the near future. If it's successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money," Chu said. The researchers are currently in discussion with industry partners to support further laboratory work and rapidly progress this new treatment option towards clinical trials.

Continued here:
Glaucoma can be successfully treated with gene therapy - International Business Times, Singapore Edition

Ever since their son Michael was diagnosed with a rare genetic disorder, Terry and Georgia Pirovolakis have been racing to find a cure. They were getting close, and then everything came grinding to a halt.

"We had a whole bunch of researchers around the world doing different things. All of our research has basically stopped," says Terry Pirovolakis.

Michael is two-and-a-half years old and has spastic paraplegia type 50 (SPG-50), a rare neurodegenerative disease caused by a missing gene that is progressively robbing him of his ability to walk.

Over the past year, his parents have raised more than $1 million to help finance a clinical trial for custom gene therapy that promises to halt the disease. But the COVID-19 pandemic has paralyzed research around the world, and their hope along with it.

"It's hard to accept the fact that we were making so many gains," says Georgia Pirovolakis. "You know, we were hoping he would be walking by September."

Michael is just one of the many casualties of the global health crisis. From new gene therapies to help children like Michael, to the latest in cancer treatments, thousands of clinical trials deemed non-essential are on hold.

Brad Wouters is executive vice-president of Science and Research at Toronto's University Health Network, Canada's largest academic hospital. He says an example of the impact is a patient currently being treated for pancreatic cancer.

"He was part of a medical research study here that looked at the underlying genetics of that tumour, and that information revealed a potential new therapy that could be very effective for that patient in immunotherapy. But we only have access to that drug through a clinical trial. And so he won't get it."

Wouters says the only new research happening at the moment is around COVID-19. About 200 of UHN's researchers have pivoted to that, but thousands more risk losing their jobs, Wouters says, because in the past month alone UHN has lost $6 million in industry funding. It's why UHN is asking the federal government for help.

"We've been doing everything we can to try and support these jobs and support this research sector," says Wouters. "But it's over a month now, and we're really at a tipping point where we're going to see significant job losses if something can't be done."

Canada's $3-billion medical research industry relies on that brain trust, much of it made up of international students, says Martha Crago, vice-principal of Research and Innovation at McGill University.

"We wouldn't want to lose this wonderful set of brains coming into our country and helping bring solutions to the public domain," Crago says. "We need to do what we can to keep them."

The pandemic will be over at some point, Crago says, and those researchers will be needed to help regain lost scientific momentum.

Meanwhile, the Pirovolakis family fear they're running out of time.

All the therapies Michael has been doing to maintain the movement he has now, like physical and occupational therapy, have been cancelled. They're trying to improvise at home to keep him moving, and to keep their hope alive too.

The stalled research efforts means a cure for Michael may be delayed for up to a year. That could mean the difference between him ever walking or not.

"It means he's going to degrade," says Terry Pirovolakis. "The progression of the disease is going to kick in, he'll slowly become more and more paralyzed."

It's why the couple are trying to keep research into Michael's gene therapy going.

They're considering using money the family has raised so far to finance early toxicology tests on a potential treatment researchers in the U.S have already developed. They are also lobbying Canada's National Research Council to help develop a treatment or help with funding.

Georgia Pirovolakis says there are so many unknowns, and she can't bear to dwell on them.

"I just look at one day at a time. I'm not thinking about, you know, tomorrow. I'm not thinking about one month from now. I'm not thinking about the research stopping, him potentially not getting cured."

The rest is here:
'We're really at a tipping point': COVID-19 brings research into other medical conditions to a grinding halt - CBC.ca

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Single-Use Technologies for the Biologic Market Research Report 2020-2026 (Based on 2020 COVID-19 Worldwide Spread) - Cole of Duty

Insulin injections cancontrol diabetes, but patients still experience serious complications such as kidney disease and skin infections. Transplanting pancreatic tissues containing functional insulin-producing beta cells is of limited use, because donors are scarce and patients must take immunosuppressant drugs afterward.

Now, scientists atWashington University in St. Louis havedeveloped a way to use gene editing system CRISPR-Cas9 to edit a mutation in human-induced pluripotent stem cells (iPSCs) and then turnthem into beta cells. When transplanted into mice, the cells reversed preexisting diabetes in a lasting way, according to results published in the journal Science Translational Medicine.

While the researchers used cells from patients with Wolfram syndromea rare childhood diabetes caused by mutations in the WFS1 genethey argue that the combination of a gene therapy with stem cells could potentially treat other forms of diabetes as well.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

One of the biggest challenges we faced was differentiating our patient cells into beta cells. Previous approaches do not allow for this robust differentiation. We use our new differentiation protocol targeting different development and signaling pathways to generate our cells, the studys lead author, Kristina Maxwell, explained in a video statement.

Making pancreatic beta cells from patient-derived stem cells requires precise activation and repression of specific pathways, and atthe right times, to guide the development process. In a recent Nature Biotechnology study, the team described a successful method that leverages the link between a complex known as actin cytoskeleton and the expression of transcription factors that drive pancreatic cell differentiation.

This time, the researchers applied the technology to iPSCs from two patients with Wolfram syndrome. They used CRISPR to correct the mutated WFS1 gene in the cells and differentiated the edited iPSCs into fully functional beta cells.

After transplanting the corrected beta cells into diabetic mice, the animals saw their blood glucose drop quickly, suggesting the disease had been reversed. The effect lasted for the entire six-month observation period, the scientists reported. By comparison, those receiving unedited cells from patients were unable to achieve glycemic control.

RELATED:CRISPR Therapeutics, ViaCyte team up on gene-edited diabetes treatment

The idea of editing stem cells with CRISPR has already attracted interest in the biopharma industry. Back in 2018, CRISPR Therapeutics penned a deal with ViaCyte to develop off-the-shelf, gene-editing stem cell therapies for diabetes. Rather than editing iPSCs from particular patients themselves to correct a faulty gene, the pairs lead project used CRISPR to edit healthy cells so that they lackedthe B2M gene and expressed PD-L1 to protect against immune attack. The two companies unveiled positive preclinical data inSeptember.

Other research groups working on gene therapy or stem cells for diabetes include a Harvard University scientist and his startup Semma Therapeutics, whichdeveloped a method for selecting beta cells out of a mixture of cells developed from PSCs. Scientists at the University of Wisconsin-Madison recently proposed that removing the IRE1-alpha gene in beta cells could prevent immune T cells from attacking them in mice with Type 1 diabetes.

The Washington University team hopes its technology may help Type 1 diabetes patients whose disease is caused by multiple genetic and environmental factors as well as the Type 2 form linked to obesity and insulin resistance.

We can generate a virtually unlimited number of beta cells from patients with diabetes to test and discover new drugs to hopefully stop or even reverse this disease, Jeffrey Millman, the studys co-senior author, said in the video statement. Perhaps most importantly, this technology now allows for the potential use of gene therapy in combination with the patients own cells to treat their own diabetes by transplantation of lab-grown beta cells.

Go here to see the original:
Reversing diabetes with CRISPR and patient-derived stem cells - FierceBiotech

Glaucoma can be successfully treated with gene therapy

London, April 22 (IANS) A common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients, say researchers.

Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight.

Current treatments include either eye drops, laser or surgery, all of which have limitations and disadvantages.

"At present, there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early," said study researcher Dr Colin Chu from the University of Bristol in the UK.

For the findings, published in the journal Molecular Therapy, the research team tested a new approach that could provide additional treatment options and benefits.

The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.

The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye.

Using the latest gene-editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure.

"We hope to advance towards clinical trials for this new treatment in the near future. If it''s successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money," Chu said

The researchers are currently in discussion with industry partners to support further laboratory work and rapidly progress this new treatment option towards clinical trials.

--IANS

bu/na

Disclaimer :- This story has not been edited by Outlook staff and is auto-generated from news agency feeds. Source: IANS

Original post:
Glaucoma can be successfully treated with gene therapy - Outlook India

HOUSTON, April 22, 2020 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates, today announced that it has welcomed Dr. Richard Whitley to its Science Advisory Board to guide its development strategy for WP1122 for the potential treatment of COVID-19 and other viral diseases.

"Our development effort to get WP1122 into a clinical trial with COVID-19 patients is now getting into high gear," commented Walter Klemp, Chairman and CEO of Moleculin. "Among his many accomplishments, Dr. Whitley leads the Drug Discovery and Development Center for the National Institute of Allergy and Infectious Diseases, making him one of the Nation's leading authorities on the clinical development of antiviral therapies."

Richard Whitley, M.D., is a Distinguished Professor of Pediatrics, Professor of Microbiology, Medicine and Neurosurgery; Loeb Eminent Scholar Chair in Pediatrics; Co-Director, Division of Pediatric Infectious Diseases; Vice-Chair, Department of Pediatrics; Senior Scientist, Department of Gene Therapy; Scientist, Cancer Research and Training Center; Faculty, Gene Therapy Center; Associate Director for Drug Discovery and Development and Senior Leader, Pediatric Oncology Program, O'Neal Comprehensive Cancer Center at the University of Alabama at Birmingham (UAB); and Co-Founder and Co-Director, Alabama Drug Discovery Alliance.

Dr. Whitley is responsible for the National Institute of Allergy and Infectious Diseases Collaborative Antiviral Study Group whose role is to perform clinical trials of antiviral therapies directed against medically important viral diseases of children and adults including viruses considered as threats to human health. He has published more than 347 articles. He participates in numerous Data Safety and Monitoring Boards for ongoing clinical studies. He is a past President of the Infectious Diseases Society of America and received the UAB President's Medal in 2007. In 2013, he was named as the inaugural recipient of the Distinguished Clinical Research Scholar and Educator in Residence at the NIH Clinical Center.

About Moleculin Biotech, Inc.

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors and viruses. The Company's clinical stage drugs are: Annamycin, a Next Generation Anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity, being studied for the treatment of relapsed or refractory acute myeloid leukemia, more commonly referred to as AML; WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, under investigation for brain tumors, pancreatic cancer and hematologic malignancies; and WP1220, an analog to WP1066, being developed for the topical treatment of cutaneous T-cell lymphoma. Moleculin is also engaged in preclinical development of additional drug candidates, including additional immune/transcription modulators, as well as WP1122, a compound capable of metabolism/glycosylation disruption.

For more information about the Company, please visithttp://www.moleculin.com.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the ability of WP1122 to demonstrate safety and efficacy in humans. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin Biotech has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission ("SEC") and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. The Company cautions investors not to place undue reliance on the interim results announced today.

ContactsJames Salierno / Carol RuthThe Ruth Group646-536-7028 / 7000jsalierno@theruthgroup.comcruth@theruthgroup.com

View original content:http://www.prnewswire.com/news-releases/moleculin-announces-head-of-niaid-antiviral-drug-discovery-and-development-center-joins-covid-19-drug-development-team-301044999.html

SOURCE Moleculin Biotech, Inc.

Original post:
Moleculin Announces Head of NIAID Antiviral Drug Discovery and Development Center Joins COVID-19 Drug Development Team - BioSpace

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy.

This press release was orginally distributed by SBWire

San Francisco, CA -- (SBWIRE) -- 04/22/2020 -- TMR Research states that global gene therapy for inherited genetic disorders market will see a witness major developments in the coming years.Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

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Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

Some of the most prominent competitors operating in the competitive landscape of global gene therapy for inherited genetic disorders market include

Spark Therapeutics Inc.Orchard TherapeuticsNovartis AGBluebird bio Inc.BioMarin PharmaceuticalGlobal Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

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Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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For more information on this press release visit: http://www.sbwire.com/press-releases/gene-therapy-for-inherited-gen/release-1288387.htm

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Gene Therapy for Inherited Genetic Disorders Market Analysis Key Players, Share Dynamic Demand and Consumption by 2018 to 2028 - Press Release -...

ROCKVILLE, Md., April 22, 2020 /PRNewswire/ --

REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAVTechnology Platform, today provided additional long-term data from the ongoing Phase I/IIa trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD).

"I am impressed by the overall outcomes in patients after a one-time administration of RGX-314. I believe that RGX-314 is the leading gene therapy program for a major retinal disease such as wet AMD and could be an important potential one-time treatment option for AMD patients who require frequent and burdensome anti-VEGF injections. Real-world evidence demonstrates that patients lose vision over time with our current standard of care and incur significant treatment burden with frequent clinic visits and injections," said Allen C. Ho, M.D., Director of Retina Research at Wills Eye Hospital and Mid Atlantic Retina and investigator surgeon in the RGX-314 trial.

"The clinical profile of RGX-314 appears very promising as a one-time treatment strategy for wet AMD as we continue to learn about the consistent and durable effects of our anti-VEGF gene therapy," added Steve Pakola, M.D., Chief Medical Officer ofREGENXBIO. "We are encouraged that all patients in Cohort 5 who were anti-VEGF injection-free at six months remained anti-VEGF injection-free at nine months. We also have further evidence of dose-dependent intraocular protein levels in this Phase I/IIa study. We are on track to have one-year data from our later cohorts in mid-2020 and look forward to initiating a pivotal program for the subretinal delivery of RGX-314 in the second half of 2020. We are also continuing our preparations to initiate a Phase II trial for the in-office suprachoroidal delivery of RGX-314 in wet AMD patients in the first half of this year."

Study Design and Safety

In the Phase I/IIa trial of RGX-314, 42 patients with severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3x109 GC/eye to 2.5x1011 GC/eye. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314.

Patients in the study are being assessed each month for 24 months and will receive safety follow-up for five years after RGX-314 administration. Efficacy assessments for the study include reduction in anti-VEGF intravitreal injections, change in vision as measured by Best Corrected Visual Acuity (BCVA), change in central retinal thickness (CRT) as measured by spectral domain optical coherence tomography (SD-OCT), and protein expression levels as measured from aqueous samples by electrochemiluminescence immunoassay (ECL).

As of April 6, 2020, RGX-314 continued to be well-tolerated across all cohorts, with no drug-related serious adverse events (SAEs) reported. Sixteen SAEs that were not related to RGX-314, including two ocular procedure-related SAEs, were reported in ten patients. There have been no reports of clinically-determined immune responses, drug-related ocular inflammation, or post-surgical inflammation beyond what is expected following routine vitrectomy.

Across all 42 patients in the study, the most common nonserious adverse events in the study eye were generally assessed as mild (90%). These included post-operative conjunctival hemorrhage (69% of patients), post-operative inflammation (36% of patients), eye irritation (17% of patients), eye pain (17% of patients), and post-operative visual acuity reduction (17% of patients). In 67% of patients across all cohorts, and in 83% of patients in Cohorts 3-5, mild to moderate retinal pigmentary changes were observed on imaging, the majority of which were in the peripheral inferior retina. There was no evidence of clinical symptoms or changes to visual acuity related to retinal pigmentary changes. Retinal hemorrhage was observed in 17% of patients and is an anticipated event in patients with severe wet AMD.

Summary of Long-term Data for Cohort 3 Over Two Years

Positive long-term potential efficacy signals were sustained over two years in Cohort 3.The mean change in visual acuity across all six patients in Cohort 3 was markedly improved over two years, with a mean BCVA improvement of +14 letters, and the mean change in CRT was stable, with an increase of +2 m.

Patients in Cohort 3 also demonstrated long-term reductions in anti-VEGF treatment burden over two years with a mean annualized rate of 2.8 anti-VEGF injections after administration of RGX-314, which is a reduction of over 60% from the mean annualized injection rate during the twelve months prior to administration of RGX-314. Three out of six (50%) patients received no anti-VEGF injections over two years following one-time administration of RGX-314. One patient received four anti-VEGF injections after RGX-314 administration and then did not receive anti-VEGF injections from nine months through two years.

The four patients who did not receive anti-VEGF injections after nine months demonstrated a mean BCVA improvement of +14 letters, with a range of +6 letters to +25 letters. In addition, these patients had stable retinal thickness with a mean change of +9 m.

Additionally, long-term intraocular RGX-314 protein expression was stable in patients in Cohort 3 over two years. The mean RGX-314 protein expression level in Cohort 3 was 227.2 ng/ml at two years, compared to 217.8 ng/ml at six months. The mean RGX-314 protein expression level in the four patients who did not receive anti-VEGF injections after nine months was 291.7 ng/ml at two years, compared to 273.6 ng/ml at six months.

Summary of Data for Cohorts 4 and 5

Consistent with previous results, intraocular RGX-314 protein expression levels increased in a dose-dependent manner across cohorts when measured at six months after administration of RGX-314; the mean protein expression level in Cohort 4 and Cohort 5 was 653.6 ng/ml and 848.7 ng/ml, respectively.

Patients in Cohort 5 continued to demonstrate a meaningful reduction in anti-VEGF treatment burden over nine months following administration of RGX-314, with 8/11 (73%) patients remaining anti-VEGF injection-free, and a reduction across the cohort of over 80% from the mean annualized injection rate during the 12 months prior to RGX-314 administration.

Conference Call

In connection with this announcement, REGENXBIO will host a webcast and conference call with accompanying slides today at 8:30 a.m. ET. This event will feature study investigators Allen C. Ho, M.D., Wills Eye Hospital and Mid Atlantic Retina, Robert Avery, M.D., Founder of California Retina Consultants and Research Foundation, and Peter Campochiaro, M.D., Johns Hopkins Wilmer Eye Institute.

To access a live or recorded webcast of the call and accompanying slides, please visit the "Investors" section of the REGENXBIO website at http://www.regenxbio.com. To access the live webcast by phone, dial (855) 422-8964 (domestic) or (210) 229-8819 (international) and enter the passcode 6668158. The recorded webcast will be available for approximately 30 days following the call.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other additional chronic retinal conditions treated with anti-VEGF. RGX-314 consists of the NAV AAV8 vector encoding an antibody fragment which is designed to inhibit VEGF, modifying the pathway for formation of new leaky blood vessels which lead to retinal fluid accumulation and vision loss.

About the Phase I/IIa Clinical Trial of RGX-314

RGX314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, doseescalation study in adult patients with wet AMD in the United States. The study includes patients previously treated for wet AMD who are responsive to anti-VEGF therapy. The study is designed to evaluate five escalating doses of RGX-314, with six patients in the first three dose cohorts and 12 patients in the fourth and fifth dose cohorts. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the study is safety at 6 months following administration of RGX-314. Secondary endpoints include visual acuity, retinal thickness on SDOCT, ocular RGX-314 protein expression, and the need for additional anti-VEGF therapy. Following completion of the primary study period, patients enter a follow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

AboutREGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at http://www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements,whether as a result of new information, future events or otherwise.

Contacts:

Tricia TruehartInvestor Relations and Corporate Communications347-926-7709ttruehart@regenxbio.com

Investors:Heather Savelle, 212-600-1902heather@argotpartners.com

Media:David Rosen, 212-600-1902david.rosen@argotpartners.com

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REGENXBIO Announces Additional Positive Long-term and Interim Phase I/IIa Trial Update for RGX-314 for the Treatment of Wet AMD - Herald-Mail Media

MILAN--(BUSINESS WIRE)--Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan, announced today an investment in Genespire, a Milan-based gene-therapy company developing best-in-class therapies for patients affected by genetic diseases. This is the third investment for the Sofinnova Telethon Fund, bringing the total financing of Italian startups by Sofinnova Partners to 25 million over the past six months. The Sofinnova Telethon Fund is the largest fund in Italy dedicated to biotechnologies, making early-stage investments in Italian companies targeting cures for rare and genetic diseases.

Genespire was spearheaded by the renowned gene therapy pioneer Professor Luigi Naldini. This investment by the Sofinnova Telethon Fund follows on the heels of its leading-edge investments in Epsilen Bio, which is developing a technology to turn off genes linked to specific pathologies without irreversibly modifying the DNA (a major potential safety advantage), and PinCell, a pre-clinical stage biotech company focused on the development of new therapies for rare dermatological diseases.

These investments are the result of the excellent collaboration between Sofinnova Partners and The Telethon Foundation, said Graziano Seghezzi, Managing Partner of Sofinnova Partners. These exceptional start-up companies validate our conviction in the strength of the Italian market for supporting biotechnological advances.

The Sofinnova Telethon Fund, managed by Sofinnova Partners, received commitments totaling 108 million, exceeding its target and establishing it as the largest fund to emerge from ITATech, a joint venture between the European Investment Fund (EIF) and Italys Cassa Depositi e Prestiti (CdP). We have gained unprecedented levels of support from Italian investors for our Italian biotech fund, added Mr. Seghezzi. We are proud and humbled by the trust we received from some of the countrys leading institutional, corporate and private investors who more than doubled the initial support provided by ITATech.

Francesca Pasinelli, CEO of The Telethon Foundation, said the biggest winners of this partnership would be the patients who suffer from rare and genetic diseases. This Fund has always been about them and additionally, is dedicated to elevating Italian science, supported by Italian investors and focused on Italys best entrepreneurs and companies, she said. We are seeing deep synergies between what we are doing with Sofinnova Partners and our ongoing, fruitful collaboration with the San Raffaele Hospital.

The Sofinnova Telethon Fund team is led by technology transfer specialists Lucia Faccio and Paola Pozzi, Partners at Sofinnova Partners, who are based in the Milan office. The office also houses BiovelocITA, Italys first biotechnology accelerator co-founded in 2015 by Sofinnova Partners in collaboration with Silvano Spinelli and Gabriella Camboni, two serial entrepreneurs with close ties to Sofinnova Partners. Ms. Faccio has over 20 years of experience in the domain as the Director of Research and Business Development of The Telethon Foundation and as the Director of the Technology Transfer Office at the San Raffaele Hospital. Ms. Pozzi, who succeeded Ms. Faccio at the San Raffaele Hospital, has 16 years of experience in patent portfolio market-oriented management and development leading to incorporation of new companies with strong IP assets.

The Sofinnova Telethon Fund will continue to invest exclusively in Italy, seeking to identify, create and develop some of the best companies in the field of rare and genetic diseases.

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management. For more information, please visit: http://www.sofinnovapartners.com

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Prof. Alessio Cantore, The Telethon Foundation and San Raffaele Hospital. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and backed by Sofinnova Partners.

About Epsilen Bio

Epsilen Bio is a biotechnology company developing transformative therapies for patients affected by underserved medical conditions, through stable and long-lasting epigenetic silencing of genes involved in pathological processes. Based in Milan, Italy, Epsilen Bio was founded in December 2019 by the renowned gene therapy experts Prof. Angelo Lombardo and Prof. Luigi Naldini, The Telethon Foundation and San Raffaele Hospital. Epsilen Bio is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute, and received seed funding from Sofinnova Partners. For more information, please visit: http://www.epsilenbio.com

About PinCell

PinCell is a biotechnology company targeting a novel pathological pathway to develop first-in-class anti-inflammatory therapy for the treatment of rare, severe skin diseases with high unmet medical need. Based in Milan, Italy, PinCell was founded in October 2008 as an academic spin-off of the University of Modena and Reggio Emilia by the world-class dermatology experts Prof. Carlo Pincelli and Dr. Alessandra Marconi. PinCell received seed funding from Sofinnova Partners. For more information, please visit: http://www.pincell.it

About The Telethon Foundation (Fondazione Telethon)

The Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a group of stakeholders, who saw scientific research as the only real opportunity to fight genetic diseases and win. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 28 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of research with a view to developing treatments. For more information, please visit: http://www.telethon.it

About San Raffaele Hospital (Ospedale San Raffaele)

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctors to grow professionally. For more information, please visit: http://www.hsr.it

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Sofinnova Partners Announces Third Investment From Its Italian Fund - Business Wire

How do we learn when to cooperate, and when to cheat? It likely depends in part on the social interactions we observe growing up. Turns out that the same is true for cleaner fish (Labroides dimidiatus), according to a recent study.

Cleaner fish, also known as bluestreak cleaner wrasse, feed on ectoparasites found on other fishes their clients. What cleaner fish really like to eat, however, is the protective mucus that covers their clients bodies. While eating ectoparasites is great for client fishes, cheating by eating mucus is bad. Client fishes will punish cheaters by fleeing, or by chasing the cheaters around.

Photo by David Clode on Unsplash

Whats the best option, to cheat and eat a brief but delicious snack (and risk being chased around), or to cooperate and eat a larger, peaceful, but not as tasty meal? A group of researchers led by Noa Truskanov, from the University of Neuchtel, set out to understand how juvenile cleaner fish learn to answer that question.

In a series of experiments recently published in Nature Communications, Truskanov and her collaborators allowed juvenile cleaner fish to observe adults interacting with model clients. They then placed these juveniles in the same situations they had just observed, and tested if they copied the adult fish, learned from the adult fish, or behaved independently of what they had just seen.

Their results show that juvenile fish are actively learning to cooperate by observing the interactions of adult fish with their clients. Juvenile fish are more cooperative when client fishes are intolerant of cheating. When given the choice, however, they prefer clients who allow them to cheat a little. Juvenile fish also didnt just imitate random adult preferences, showing that there are not copying, but rather learning.

Although both social learning and cooperation are widespread in nature, examples of animals that learn socially how to cooperate are extremely scarce. This leads us, humans, to erroneously assume that we are the only ones who learn to cooperate by observing others. Cleaner fish are putting us right back in our place: they can do it, too.

Link:
A real world version of Pokmon Go lets you track orangutans in the jungle - Massive Science

Add a new, 100 million California facility to the growing gene therapy infrastructure.

Merck KGaA announced they are opening a second gene therapy and viral vector factory in Carlsbad, California. The new center will be just one point in the global supply chain Big Pharma is rapidly erecting to keep manufacturing capacity for the new technology at pace with clinical development. Over the past year, Novartis, PTC Therapeutics, Pfizer and Vertex each announced or opened new facilities in Switzerland, North Carolina and New Jersey that will help build gene therapy.

In November,Reutersreported that 11 drugmakers had set aside $2 billion for the manufacturing effort. They were led by Novartis, the giant behind the second FDA-approved gene therapy in Zolgensma which planned to spend $500 million, and Pfizer, which has yet to get a gene therapy approved but will spend $600 million. Catalent and Thermo Fisher, meanwhile, each spent over a billion dollars acquiring companies involved in gene therapy or viral vector manufacturing.

Merck KGaA last shored up its gene therapy manufacturing in 2016 the year before the first gene therapy was approved in the US when they expanded their original Carlsbad facility from 44,000 to 65,000 feet. At 140,000 feet, the new facility will be more than double the size of its older neighbor. Gene therapies and the viral vectors used to deliver them will be cooked up in 1,000 liter bioreactors metal vats roughly the size of 260 gallons of milk. Merck has not said when it is scheduled for completion.

Although Merck KGaA lists no gene therapies in its most recent pipeline, the German drugmaker uses its facilities to help biotechs focused on the emerging modality to manufacture their products. The list of approved gene therapies can still be counted on one hand, but hundreds are now in clinical trials.

Manufacturing emerged as a pressing bottleneck almost as soon as Spark Therapeutics got Luxturna, a treatment for a form of inherited blindness, approved as the US first gene therapy. In an op-ed in STAT last year, Sparks head of technical operations, Diane Blumenthal, described the process of building in Philadelphia one of the first in-house gene therapy manufacturing facilities. With regulators increasingly open to approving the treatments quickly, she encouraged other companies to invest in manufacturing in advance of even knowing if the therapy works, particularly because gene therapy requires customization in ways few other modalities do.

And she warned the problem will only get more pressing.

There isnt a gene therapy manufacturing playbook yet to guide the development of gene therapies, Blumenthal wrote. Manufacturing a gene therapy is only half the battle. The other half is making enough of it, doing that as efficiently as possible, and getting it to the patients who need it. These challenges become even more urgent to tackle as the industry shifts to the next chapter in gene therapy development, from treatments made in small batches for small patient populations to bigger volumes for larger rare-disease populations and commercial scale.

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Merck KGaA adds to gene therapy manufacturing boom with 100M facility in California - Endpoints News

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Viral vectorNon-viral vector

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Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

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Global Gene Therapy Market Analysis, Technologies, Innovations, Future Guidelines and Forecasts Report 2026 - Cole of Duty

Nancy J. Hutson, Ph.D.

Nancy J. Hutson, Ph.D. has been appointed to Clearside Biomedical's Board of Directors, effective April 21, 2020.

ALPHARETTA, Ga., April 22, 2020 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc.(NASDAQ:CLSD), a biopharmaceutical company dedicated to developing and delivering treatments that restore and preserve vision for people with serious back of the eye diseases, announced today that Nancy J. Hutson, Ph.D. has been appointed to the Company's Board of Directors, effective April 21, 2020.

The addition of Nancy Hutson to our Board of Directors comes at a key time as we are expanding our research and development pipeline and preparing to initiate a clinical program targeting the treatment of wet age-related macular degeneration with CLS-AX, said William Humphries, Chairman of the Clearside Board of Directors. Nancy brings a deep understanding of the business coupled with an in-depth knowledge of research, drug development and dynamic business situations from her years of R&D leadership at Pfizer and extensive board service in a number of biopharmaceutical companies. We welcome Nancy and look forward to making further progress in achieving our strategic goals.

I look forward to working with the Clearside team to leverage the Companys proprietary suprachoroidal space injection platform, said Dr. Hutson. With a flexible treatment approach, unique R&D pipeline, multiple global partnerships, and a robust patent portfolio, Clearside has a compelling opportunity to make a difference for patients with back of the eye diseases and deliver value for shareholders.

Nancy J. Hutson, Ph.D., is a highly accomplished healthcare executive with more than 30 years of experience as a seasoned research and development expert and leader within the biopharmaceutical industry. Dr. Hutson retired from Pfizer, Inc. in 2006 after spending 25 years in various research and leadership positions, serving as Senior Vice President, Pfizer Global Research and Development and Director of Pfizer's pharmaceutical R&D site, known as Groton/New London Laboratories. At Pfizer, she led 4,500 colleagues and managed a budget in excess of $1 billion. Dr. Hutson is currently a board member of Endo International, plc (ENDP), BioCryst Pharmaceuticals, Inc. (BCRX) and PhaseBio Pharmaceuticals, Inc (PHAS). She serves as Vice-Chair of the Board of Trustees at Illinois Wesleyan University. Dr. Hutson holds a Bachelor of Arts degree from Illinois Wesleyan University and a Ph.D. degree from Vanderbilt University. She completed a Postdoctoral fellowship in Biochemistry at University of Oxford.

About Clearside Biomedical

Clearside Biomedical, Inc. is a biopharmaceutical company dedicated to developing and delivering treatments that restore and preserve vision for people with serious back of the eye diseases. Clearsides proprietary SCS Microinjector targeting the suprachoroidal space (SCS) offers unique access to the macula, retina and choroid where sight-threatening disease often occurs. The Companys SCS injection platform is an inherently flexible, in-office, non-surgical procedure, intended to provide targeted delivery to the site of disease and to work with both established and new formulations of medications, as well as future therapeutic innovations such as gene therapy. For more information, please visit http://www.clearsidebio.com.

Investor and Media Contacts:

Jenny Kobin Remy Bernarda ir@clearsidebio.com(678) 430-8206

Source: Clearside Biomedical, Inc.

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/27843a49-a1c9-4e2f-a571-ff96a1a56ae3

The rest is here:
Clearside Biomedical Appoints Nancy J. Hutson, Ph.D. to its Board of Directors - GlobeNewswire

Bluebird Bio

Global Gene Therapy For CNS Disorders Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

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The report provides an in-depth assessment of growth and other aspects of the market in key countries such as the United States, Canada, Mexico, Germany, France, the United Kingdom, Russia and the United States Italy, China, Japan, South Korea, India, Australia, Brazil and Saudi Arabia. The chapter on the competitive landscape of the global market report contains important information on market participants such as business overview, total sales (financial data), market potential, global presence, Gene Therapy For CNS Disorders sales and earnings, market share, prices, production locations and facilities, products offered and applied strategies. This study provides Gene Therapy For CNS Disorders sales, revenue, and market share for each player covered in this report for a period between 2016 and 2020.

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Table of Contents:

Study Coverage: It includes study objectives, years considered for the research study, growth rate and Gene Therapy For CNS Disorders market size of type and application segments, key manufacturers covered, product scope, and highlights of segmental analysis.

Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Gene Therapy For CNS Disorders market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Gene Therapy For CNS Disorders Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

Production by Region: It shows how the revenue and production in the global market are distributed among different regions. Each regional market is extensively studied here on the basis of import and export, key players, revenue, and production.

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Tags: Gene Therapy For CNS Disorders Market Size, Gene Therapy For CNS Disorders Market Growth, Gene Therapy For CNS Disorders Market Forecast, Gene Therapy For CNS Disorders Market Analysis

Excerpt from:
Gene Therapy For CNS Disorders Market Analysis by Size, Share, Top Key Manufacturers, Demand Overview, Regional Outlook And Growth Forecast to 2026 -...

Grand View Research, Inc. Market Research And Consulting

According to report published by Grand View Research, The global advanced therapy medicinal products market size was valued at USD 3.14 billion in 2019 and is projected to expand at a CAGR of 17.4% during the forecast period

The globaladvanced therapy medicinal products marketsize is expected to reach USD 9.6 billion by 2026, expanding at a CAGR of 17.4% over the forecast period, according to a new report published by Grand View Research, Inc. The use of advanced therapy medicinal products (ATMPs) is well-proven for the improvement in the quality of life and health status in the long run for patients suffering from life-threatening conditions. This is particularly for diseases with few or no alternative treatment options. These novel treatment modes deliver transformative advantages not offered by conventional forms of disease treatment, thereby driving the ATMP market.

In the current scenario, ATMPs are expensive for patients as well as health insurance schemes. Growing competition to gain revenue share could help reduce prices and improve the affordability, along with the acceptance of these products as mainstream medicine. Furthermore, manufacturers are embracing efforts to streamline technology and logistics for the provision of therapies at accessible prices.

Request a sample Copy of the Global Advanced Therapy Medicinal Products Market Research Report @ http://www.grandviewresearch.com/industry-analysis/advanced-therapy-medicinal-products-market/request/rs1

Moreover, lack of manufacturing capabilities to meet growing consumer demand coupled with the resource and budget constraints faced by advanced therapy medicinal product developers has created lucrative avenues for contract services providers and has intensified the market competition. This has led to a rise in market activities by contract service providers. For instance, in November 2019, Fujifilm Diosynth established a 60,000-square-feet Gene Therapy Innovation Center to offer downstream, upstream, and analytical services.

Further key findings from the report suggest:

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Grand View Research has segmented the global advanced therapy medicinal products market on the basis of therapy type and region:

Advanced Therapy Medicinal ProductsTherapy Type Outlook (Revenue, USD Million, 2015 2026)

Advanced Therapy Medicinal ProductsRegional Outlook (Revenue, USD Million, 2015 2026)

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About Grand View Research

Grand View Research provides syndicated as well as customized research reports and consulting services on 46 industries across 25 major countries worldwide. This U.S.-based market research and consulting company is registered in California and headquartered in San Francisco. Comprising over 425 analysts and consultants, the company adds 1200+ market research reports to its extensive database each year. Supported by an interactive market intelligence platform, the team at Grand View Research Guides Fortune 500 companies and prominent academic institutes in comprehending the global and regional business environment and carefully identifying future opportunities.

Media ContactCompany Name: Grand View Research, Inc.Contact Person: Sherry James, Corporate Sales Specialist U.S.A.Email: Send EmailPhone: 1-415-349-0058, Toll Free: 1-888-202-9519Address: 201, Spear Street, 1100 City: San FranciscoState: CaliforniaCountry: United StatesWebsite: https://www.grandviewresearch.com/industry-analysis/advanced-therapy-medicinal-products-market

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Advanced Therapy Medicinal Products Market to Reach $9.6 Billion by 2026 | Key Players Spark Therapeutics, Inc.; Bluebird Bio, Inc.; Novartis AG;...

Novel promoters based on those in the herpes viruses, enabled delivery of larger genes and increased the period they were active in the nervous system.

Novel promoter sequences have been developed to improve gene therapy treatments for neurological diseases. The researchers suggest these engineered promoters should allow for the delivery of both larger genes and also enable them to remain active for longer.

Esteban Engel, a researcher in viral neuroengineering in the Princeton Neuroscience Institute, US, and his team have developed new gene promoters for use in gene therapy. The practice of replacing faulty genes or adding additional functional ones to augment a deficit or damage is thought to be a promising strategy for treating many diseases, including neurodegenerative diseases like Parkinsons and Alzheimers.

To deliver these genes to the required cells, scientists often use viral vectors engineered viruses that cannot cause disease but have all the necessary viral machinery to enter human host cells. One of the most commonly used examples is adeno-associated virus (AAV), because it is relatively harmless.

Gene promoters are added into the viral genome alongside the desired gene in order to turn on their expression. To boost the efficacy of these promoters in the nervous system, Engel and his team adopted several attributes of herpes virus promoters, because the herpes viruses can persist for years in the body through establishing a chronic infection in the nervous system.

The team reported their promoters occupied much less space than existing ones, which could enable larger genes or multiple genes to be transported in the vectors. They also observed the new promoters are long-lasting, being less prone to repression or inactivation than most, so the therapeutic genes are active for long periods of time.

The team established that these novel promoters could work with AAVs as well as other viral and non-viral gene-delivery systems.

The research was published in the journalMolecular Therapy: Methods & Clinical Development.

Originally posted here:
Novel gene promoters could improve gene therapies - Drug Target Review

NEW YORK, April 20, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the European Medicines Agency (EMA) has granted Advanced Therapy Medicinal Product (ATMP) classification to MB-107, Mustangs lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. The U.S. Food and Drug Administration (FDA) previously granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

EMA grants ATMP classifications to new therapeutics that are based on genes or cells and intended as long-term or permanent therapeutic solutions to acute or chronic human diseases at a genetic, cellular or tissue level. The ATMP program provides specific regulatory guidelines for preclinical development, manufacturing and product quality testing of ATMPs and offers incentives, including fee reductions for regulatory advice, recommendations and evaluation and certification of quality and non-clinical data.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are extremely encouraged that the EMA has granted MB-107 with ATMP classification, an important step in establishing our path to market approval and commercialization in Europe. This classification complements the RMAT designation we received last year from the FDA and brings us closer to realizing our goal of commercializing MB-107 for XSCID patients, as these patients are in desperate need of innovative and potentially curative treatment options.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital (St. Jude), UCSF Benioff Childrens Hospital in San Francisco and Seattle Childrens Hospital and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Under a licensing partnership with St. Jude, Mustang intends to develop the lentiviral gene therapy for commercial use as MB-107.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

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

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

Original post:
Mustang Bio Receives Advanced Therapy Medicinal Product Classification from European Medicines Agency for MB-107 Lentiviral Gene Therapy for X-Linked...

Evotec is building a gene therapy R&D center manned by ex-Takeda scientists and has already landed a multi-year drug discovery pact for it with their former Japanese employer.

Evotecis expanding into the field of gene therapy by building an R&D center in Austria using scientists previously employed by Takeda, whose first project will be a long-term research and discovery project with their former Japanese employer, exploring oncology, rare diseases, neuroscience and gastroenterology.

The entry by the German discovery alliance and development partnership group into gene therapy further expands its capabilities beyond small molecules, cellular therapies and biologics while the research alliance withTakeda Pharmaceutical, announced on 6 April, builds upon an existing collaboration begun last September around drug discovery programs in which Evotec will deliver clinical candidates for Takeda to pursue into clinical development.

Image: iStock/sittithat tangwitthayaphum

This addition of gene therapy is the latest step in a very long strategy at Evotec to build a truly comprehensive organization which can deliver medicines discovery and development right across the different modalities, or scientific tools, that we see in modern medicine today, Evotec chief operating officer Craig Johnstone toldScrip.

He said the move was consistent with Evotecs two-pronged drug discovery strategy, which uses the Hamburg-based groups so-called Execute segment allying with external pharma partners which in turn supports reinvestment into Evotecs internal pipeline within its so-called Innovate segment.

This allows us to bring gene therapy project concepts into the Evotec Innovate pipeline at our choice and discretion. That was not possible before, so we can now use gene therapy to support our partners and also use it to support ourselves.

Johnstone said Evotec already makes broad use of CRISPR in its discovery and development activities. This will only amplify that aspect of Evotecs activities, he added.

Evotec Gene Therapy (Evotec GT) will start operations with a team of gene therapy experts at an R&D site in Orth an der Donau, Austria. Its scientists have deep expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases.

We are recruiting the team, which will be composed of ex-Takeda employees. Its leadership has a long history in gene therapy stretching back years and to legacy organizationsBaxaltaandShire which were acquired by Takeda, but who were made redundant. Well be bringing them all on board over the next eight weeks, Johnstone said. Friedrich Scheiflinger, previously head of drug discovery for Takeda in Austria, will head up the new gene therapy unit.

No financial details were disclosed about Evotecs latest collaboration with Takeda.

Its a fairly straight-forwardcontractual framework that has been agreed between Evotec and Takeda, Johnstone said, without elaborating.

Sten Stovall is a London-based editor and writer with 40 years of experience in the field of journalism, including more than 20 years with Reuters and eight years with The Wall Street Journal/Dow Jones Newswires. He can be reached at sten.stovall@informa.com

Originally posted here:
Evotec extends Takeda pact to gene therapy - Bioprocess Insider - BioProcess Insider

REDWOOD CITY, Calif., April 20, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the appointment of Scott Whitcup, M.D. to the Companys Board of Directors.

We are delighted to welcomeScott to our board, said Patrick Machado, J.D., Board Chair of Adverum Biotechnologies. Scott's expertise and accomplished track record as an ophthalmologist and successful drug developer fit perfectly with Adverum's mission to substantially elevate the standard of clinical care for patients suffering from neovascular AMD and other vision-threatening indications. My colleagues and I all look forward to working with Scott to realize fully the significant potential benefits our technology offers patients at risk of losing their sight.

This is an exciting time to join Adverums Board as the company strategically executes on expanding its pipeline through its novel vector discovery and drug development expertise to commercialize gene therapies to treat patients with serious ocular and rare diseases, said Dr. Whitcup. The development progress of ADVM-022, including the promising clinical data demonstrated to date in the OPTIC trial, has been impressive. I look forward to partnering with the Board, and the Adverum management team, on further developing the pipeline of drug candidates and advancing ADVM-022 towards commercialization for patients with wet AMD and diabetic retinopathy.

Scott Whitcup, M.D. is the founder and chief executive officer of Akrivista and Whitecap Biosciences, two companies focused on developing new therapies in ophthalmology and dermatology. In addition, he is on the clinical faculty at the UCLA Stein Eye Institute. Previously, Dr. Whitcup was the executive vice president of research and development and chief scientific officer at Allergan, where he led the discovery, clinical development, and medical affairs organizations focused on therapeutic areas including ophthalmology, CNS, urology, dermatology, and medical aesthetics. Earlier at Allergan, he served as vice president and head, ophthalmology therapeutic area, where he secured regulatory approvals for Alphagan P, Lumigan, Restasis, and Ozurdex. Earlier in his career, Dr. Whitcup was the clinical director at the National Eye Institute at the National Institutes of Health (NIH). Dr. Whitcup earned a B.A. from Cornell University and an M.D. from Cornell University Medical College. He completed an internal medicine residency at UCLA and an ophthalmology residency at Harvard University at the Massachusetts Eye and Ear Infirmary.

Dr. Whitcup serves on the board of directors of Scilex Pharmaceuticals and Anivive Lifesciences.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration. For more information, please visit http://www.adverum.com.

Investor and Media Inquiries:Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com 1-646-876-5196

Excerpt from:
Adverum Biotechnologies Appoints Ophthalmology Industry Veteran Scott Whitcup, M.D. to Board of Directors - GlobeNewswire

LOS ANGELES--(BUSINESS WIRE)-- T-Cure Bioscience, Inc., a privately held company focused on developing autologous T cell receptor (TCR) therapy products for the treatment of solid tumors, today announced the appointment of seasoned biopharmaceutical industry executive James G. McArthur, Ph.D. to the Companys Board of Directors. Dr. McArthur brings more than 25 years of experience in drug development, financial, strategic and operational experience. Dr. McArthur has successfully founded five biotech companies and brings extensive knowledge of the healthcare space, having led companies through all phases of development from technology assessment through lead identification and proof of concept human studies.

James extensive knowledge of and experience with all aspects of drug development as well as his broad healthcare industry connections will be vital as we advance the clinical development of our TCR product candidates for the treatment of tumors expressing KK-LC-1 in collaboration with the National Cancer Institute, stated Gang Zeng, Ph.D., Chief Executive Officer of T-Cure. Dr. McArthur has previously established multiple successful biotechnology companies, led the R&D efforts to build out portfolios from the ground up, and found validating partnership opportunities with big pharma. This experience will be essential as T-Cure progresses its pipeline and increases its focus on product development efforts moving forward.

Dr. McArthur is a founder, a member of the Board of Directors and formerly served as CEO of Imara, Inc., a public clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin. He was also a founder of Vtesse, a company developing drugs for patients suffering from Niemann-Pick Type C1 Disease, which was subsequently acquired by Sucampo Pharmaceuticals, and most recently founded Tiburio, a clinical stage company focused on rare neuroendocrine disorders. Prior to co-founding Imara, Vtesse and Cydan, Dr. McArthur was an entrepreneur-in-residence at HealthCare Ventures. There, he helped to create and then served as Chief Scientific Officer of Synovex Co., a rheumatoid arthritis therapeutic company which was subsequently acquired by Roche. He has extensive research and development expertise in a number of areas including rare diseases, oncology, hematologic diseases, vascular diseases as well as gene therapy. Dr. McArthur is a listed investor on 15 issued patents and multiple patent applications. He previously served as a member of the Board of Directors of Nightstar Therapeutics, a clinical stage gene therapy company which was acquired by Biogen. Dr. McArthur obtained a PhD in biochemistry and oncology at McGill University and was a post-doctoral fellow studying Immunology at Massachusetts Institute of Technology and the University of California at Berkeley with Noble laureate James Allison.

About T-Cure Bioscience, Inc.

T-Cure Bioscience, Inc. is an innovative immuno-oncology company committed to delivering effective and durable cell therapies for the treatment of cancer. The Company focuses on isolating high avidity TCR that can be used to engineer a patients T cells to effectively target and destroy solid tumors. The Company maintains a pipeline of TCR under clinical and pre-clinical development targeting HERV-E, KK-LC-1, NY-ESO-1 and other antigens associated with solid tumors with significant unmet medical needs, including kidney cancer, breast cancer, lung cancer, gastric cancer, ovarian cancer, liver cancer, pancreatic cancer, sarcoma, and glioblastoma.

More information is available at http://www.T-Cure.com.

Forward Looking Statements

T-Cure cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industrys actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as may, might, will, should, could, expect, plan, anticipate, believe, estimate, project, intend, future, potential or continue, and other similar expressions are intended to identify forward looking statements. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that we expected. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200420005106/en/

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T-Cure Bioscience Announces Appointment of Seasoned Industry Executive James G. McArthur, Ph.D. to Board of Directors - BioSpace

Global Blood Therapeutics reports Q4 earnings, summarizes upcoming catalysts

Global Blood Therapeutics' (GBT) novel sickle cell disease drug, Oxbryta, has officially hit the market, netting a mere $2M in revenue. Meanwhile, R&D costs have nearly doubled in the last year. SG&A costs have nearly tripled! Such is expected when launching a drug, but the question is always, "to what extent is it tolerable?"

Global Blood hopes, following a non-dilutive loan, to be cash flow positive before having to resort back to dilution again:

Entered into a $150 million loan agreement with funds managed by Pharmakon Advisors LP, and drew down on the first $75 million of the non-dilutive loan. The proceeds will be used to advance the discovery and development of potential novel treatments for SCD and other grievous blood-based conditions without diverting financial resources from the launch of Oxbryta. GBT believes that the proceeds from this loan, in conjunction with existing cash and investments, have the potential to provide the necessary runway for the company to achieve positive cash flow while enabling the continued advancement of clinical development programs and other earlier-stage product candidates.

Source: GBT

The company has nearly $700M in cash and investments. This can be expected to get them through mid-2021, even with an additional $75M pending from the loan. I highly doubt the company will be cash flow positive before 2022 and suspect Global Blood will have to dilute once more. This will hopefully occur at a more advantageous pricing and following good news (e.g. successful confirmatory trial, strong sales ramp).

On the clinical front, GBT continues to enroll patients into the confirmatory TCD study.

Source: GBT March '20 corporate slides

Success would likely expand the label to children under the age of 12 as well. The confirmatory trial would also serve to prove its clinical benefit, which Oxbryta has yet to do. Accelerated approval was granted because Oxbryta is theoretically likely to procure a clinical benefit. If the confirmatory trial is to fail, Oxbryta would be pulled from the market and one would have to ask if the drug is a viable one at all. However, I propose success in the TCD trial is likely:

Source: GBT Nov. '19 corporate slides

Remaining catalysts are summarized below:

Source: GBT March '20 corporate slides

Global Blood is also involved with a few non-pivotal trials that aim to shed light on their therapeutic:

Despite achieving accelerated approval and grabbing nearly $90/share in a bull market, major catalysts and upside remain for Global Blood.

While I am not super positive about Oxbryta's revenue prospects prior to full approval, I do expect major clinical developments over the next several months. Such clinical developments could help shed light on the clinical effects of Oxbryta beyond hemoglobin improvement. Global Blood Therapeutics remains a conviction idea as I believe Oxbryta is a game-changer in sickle cell disease due to its ability to improve hemoglobin levels without a worsening of blood viscosity. A ~$3.5B enterprise valuation for Global Blood does not yet fully consider its potential in sickle cell.

Risks include, but are not limited to: Oxbryta confirmatory trial failure, dilution, Oxbryta safety and/or efficacy issues, poor Oxbryta market performance, competition (e.g. gene therapy), and US recession.

To expand on competitive risks, I do not believe gene therapy will serve as a major competitor to Oxbryta within the drug's relevant market time (patent expires in several years). As I've touched upon in previous articles, sickle cell patients are weary of curative therapies and gene therapy would be very costly and for only a select few. Furthermore, I am not aware of any additional drugs in development that share the potentially disease-modifying mechanism of action that Oxbryta bears.

I present and update my best ideas & biotech research only to subscribers of my exclusive marketplace, The Formula. I also maintain a model portfolio of my top biotech ideas whichoutperformed the market (XBI, IBB, SPY) by over 16% in 2019!

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Disclosure: I am/we are long GBT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: The intention of this article is to provide insight, not investment advice. While the information provided in this article is intended to be factual, there is no guarantee and prospect investors are encouraged to do their own fact-checking and research before investing in a company. One must also consider one's own financial standings, risk tolerance, portfolio diversification, etc. before making a decision to buy shares in a company. Many of my articles detail biotechnology companies with little or no revenue. These stocks are, therefore, speculative and volatile. Even when prospects seem promising, there is no predicting the future. Losses incurred may be significant.

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Global Blood Therapeutics: Key Catalysts Remain As The Search For Clinical Benefit Continues - Seeking Alpha

Competitive landscape section of this Gene Therapy Products Mreport covers strategic profiling of key players in the market, comprehensively analyzing their core competencies, and strategies. The global market document compiles exhaustive information acquired through proven research methodologies and from dedicated sources across several industries. Gene Therapy Products market report puts forth an absolute overview of the market that contains various aspects of market analysis, product definition, market segmentation, key developments, and existing vendor landscape. This Gene Therapy Products market document also includes geographical markets and key players that have adopted significant strategies for business developments.

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Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

Few of the major competitors currently working in the globalgene therapy products marketareAdaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Complete report on Global Gene Therapy Product Market Research Report 2019-2026 spread across 350 Pages, profiling Top companies and supports with tables and figures

Segmentation: Global Gene Therapy Products Market

Gene Therapy Products Market : By Product

Gene Therapy Products Market : By Application

Gene Therapy Products Market : ByGeography

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Key Developments in the Gene Therapy Products Market:

Gene Therapy Products Market Drivers

Gene Therapy Products Market Restraints

Competitive Analysis: Gene Therapy Products Market

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Key questions answered in the report :-

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Gene Therapy Products Market Shows Evolving Opportunities in 2020. Major Players are Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis...

At the front lineof advances in personalized medicine are cell and gene therapies. These are two overlapping fields of medical research whereby the overall aim is to treat the underlying causes of both genetic and acquired diseases.

Is the promise of personalized medicine on the edge of being delivered? Decades of research and advances in genomics, cell biology, cancer and analytical technologies have permitted exciting progress in the cell and gene therapy space recently. Technology Networks recently spoke with three of the leaders in this space, Allogene Therapeutics, bluebird bioInc. andMogrify to gain their insights on the next developments in cell and gene therapy.

Joe Foster, COO, Mogrify.

"We are on the cusp of new breakthroughs in this field and that evolution of engineered cell therapy has the potential to expand beyond cancer. Engineering, synthetic biology and gene editing has opened the door beyond allogeneic cell therapy. There is a bright future with transformative technologies that may hold the key to addressing solid tumors."

David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder, Allogene Therapeutics.

"Although gene therapies are still a nascent technology, they present a new paradigm for healthcare, offering a one-time treatment that can address the underlying genetic cause of certain severe genetic diseases and cancer. We anticipate that the three techniques being studied for gene therapy gene editing, gene addition and gene-based immunotherapy will become more distinct and that the entire treatment journey will become more efficient."

Martin Butzal, Head of Medical Europe, bluebird bio.

Read more here:
Looking to the Future of Cell and Gene Therapies - Technology Networks