It turns out a regulatory delay isnt the only bump on Sareptas road to gene therapy glory.

The biotech is being sued by Regenxbio, which is alleging that its slate of gene therapy programs, including two for Duchenne muscular dystrophy and limb-girdle muscular dystrophy infringes on a patent originally owned by the University of Pennsylvania.

The lawsuit, which was first reported by Bloomberg Law, adds another wrinkle as Sarepta strives to stay in the lead in a race to deliver the first genetic fix for Duchenne muscular dystrophy. After making its name with two antisense drugs neither of which has been proven to have an effect against the crippling disease the Cambridge, MA-based biotech has been viewed as the frontrunner versus Pfizer and trouble-prone Solid Bio. Roche was impressed enough to pay $1.15 billion to acquire ex-US rights to the program.

But Regenxbio wants Sarepta to stop stepping on their patents and pay up. The biotech, which is now based in Rockville, MD, is seeking damages for past, present and/or future infringement equaling no less than a reasonable royalty.

At the center of the dispute is US Patent No. 10,526,617. Jim Wilson, the gene therapy luminary who co-founded Regenxbio, was cited as an inventor on that patent, granted this January.

As it covers a cultured host cell containing a recombinant nucleic acid molecule encoding the capsid protein, the technology can be used to create adeno-associated vectors both in animal studies and for delivering a transgene into humans.

The vectors made using the claimed subject matter of the 617 Patent have unique properties, e.g., an ability to target certain types of cells in the body, the lawsuit states.

Specifically, Regenxbio alleges, Sareptas Duchenne program SRP-9001 is manufactured by a process that includes making and using a cultured host cell a recombinant nucleic acid molecule encoding an AAVrh74 vp1 capsid protein. The same capsid protein is also integral to SRP-9003, the limb girdle candidate, and a host of other follow-on gene therapies.

Regenxbio is suing as Sarepta is sorting out a new request from regulators that can push back its pivotal study for Duchenne. While CEO Doug Ingram assured investors that quality control issues such as this are not uncommon and pinned the delay on an overburdened FDA, he stopped short of promising concrete timelines.

Focused on retinal, metabolic and neurodegenerative diseases for its internal pipeline, Regenxbio is perhaps more accomplished as a gene therapy tech provider. Novartis and Abeona have both licensed its NAV tech platform.

It also doesnt shy away from legal actions. In its most recent quarterly report, Regenxbio disclosed that Abeona failed to make a $8 million payment due in April, effectively terminating their licensing agreement. In response to an arbitration claim Abeona filed in May alleging we breached certain responsibilities to communicate with Abeona regarding our prosecution of licensed patents, Regenxbio filed a counterclaim to ask for $28 million including $20 million that would have been owed under the pact.

Last November Regenxbio challenged the FDAs arbitrary and capricious decisions to issue a full clinical hold on its diabetic retinopathy trial and a partial hold on wet age-related macular degeneration. The company had withdrawn the IND for diabetic retinopathy, and the FDA lifted the partial hold for wet AMD two months later.

Original post:
Sarepta faces another gene therapy hiccup as Regenxbio sues over Jim Wilson's patent - Endpoints News

China is predicted to continue its rapid bio growth rates, with more than 10 new mAbs

CPhI Annual Report has launched ahead of the firstCPhI Festival of Pharma(5-16 October 2020), the worlds largest digital pharma Expo that predicts dramatic growth of new mAb production in China, capacity shortages for cell and gene therapies in the USA, and the widespread global adoption of single-use technologies, but only limited continuous bioprocessing.

Three CPhI experts from BioPlan Associates Vicky Qing XIA, Leo Cai Yang and Eric Langer explore the rapidly changing global biologics markets, with special reference to the implications for contract outsourcing and Chinas continued emergence as a hub for both bio innovation and contract services.

Remarkably, China is predicted to continue its rapid bio growth rates, with more than 10 new mAbs predicted to be launched per year in the country by 2025. In fact, the total market size will quadruple by 2025, reaching 120bn RMB, and rising further to 190bn RMB by 2030.

According to the CPhI report, bioprocessing outsourcing in China is currently highly stratified with four tiers and just one domestic company intierone WuXi Biologics and a number of international CDMOs including BI, Lonza and Merck. However, by 2025 it is anticipated that as many as five more domestic CDMOs may have reached tier one status, with FDA and EU facility approvals.

Single Use Systems (SUS) are now far and away the leader at both pre-clinical and clinical stages, with nearly 85% now involving a substantial SUS component. Yet whilst its usage continues to grow, continuous bioprocessing is not anticipated to be in mainstream usage by 2025.

The report also suggested that in the US and Europe there is likely to be a cell and gene therapy capacity crunch by 2025, with CDMOs investing in this area already expanding to try and meet the pipelines demand.

More here:
2025 will see cell & gene therapy capacity shortages in US: CPhI report - BSA bureau

David Dismuke, Ph.D., joins as Chief Technical Officer, Christopher Shilling as Vice President of Regulatory Affairs and Quality Assurance, and Christina Perry as Vice President of Finance and Operations.All three leaders will be in place in September and join Forge's executive leadership team.

"The addition of David, Chris, and Christina to the Forge leadership team brings immeasurable value to the Forge team and expands our team of gene therapy experts," said Timothy J. Miller, Ph.D., Forge's Co-Founder, President & CEO. "We believe that our accomplished and passionate team, with demonstrated experience in manufacturing and developing gene therapy products, is the foundation of fulfilling our mission to accelerate transformative medicines to reach those who need them most."

David Dismuke joins Forge with more than 15 years of experience in large-scale manufacturing. He is an authority in the bioprocessing and design of gene therapy vectors and has led CMC operations in the large-scale manufacturing of pre-clinical and clinical-grade AAV vectors for more than 10 years. Prior to joining Forge, David was the Vice President of Manufacturing at StrideBio where he directed the development of manufacturing and analytical processes for AAV vectors that utilize novel capsids. In addition, he led the design of therapeutic and reporter transgenes and innovative molecular enhancements to improve AAV production and therapeutic function.He was also previously the Head of Vector Production at Voyager Therapeutics where he led teams in the manufacturing and analytical testing of AAV using the baculovirus/Sf9 production system.Prior to Voyager, David was the Director of the UNC Vector Core, where he oversaw GMP operations as well as the production of research-grade vectors.He earned his PhD from Vanderbilt University, focusing on the molecular biology and lifecycle of HIV-1, and then performed his postdoctoral research at UNC Chapel Hill.

Christopher Shilling joins Forge as Vice President of Regulatory Affairs and Quality. He has over 15 years of experience in development of novel gene therapies for rare and severe disorders. Christopher is an experienced leader in gene therapy regulatory affairs, pharmacology, toxicology, and project management focused on developing strategies for early phase clinical trials in support of a variety of transformative therapeutics for pediatric and rare diseases. Prior to joining Forge, Christopher started the Drug and Device Development program at Nationwide Children's Hospital which was instrumental in gaining acceptance from regulators for over twenty first-in-human gene therapy clinical trials of novel biologic products, a dozen orphan drug designations, and two fast track designations. He received his Bachelor of Science degree in biology and a Master of Science degree in Pathology both from the Ohio State University.

Christina Perry joins Forge Biologics as the Vice President of Finance and Operations. She spent the last several years as the CFO of Drive Capital where she built out all accounting and finance operations, back-office needs, and investor relations and reporting. Christina managed complex entity legal structures and has had extensive exposure to equity and debt financings.Over the last 15+ years Christina has worked with public and private companies and startups across varying industries, developing operational processes to scale with high growth. She began her career at Deloitte, is a licensed CPA in the state of Ohio, earned her Bachelor of Business Administration at the University of Notre Dame and a Master of Accountancy from Miami University.

These additions to the Forge Biologics leadership team continue Forge's momentum within the biotechnology industry in Columbus Ohio, bringing positive impact to both Ohio and the global rare disease community.

About Forge BiologicsThe mission of Forge Biologics is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, referred to as "The Hearth", to serve as their headquarters. The Hearth will be home to a custom-designed cGMP facility, dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit https://www.forgebiologics.com.

Media Inquiries:Dan SalvoForge Biologics Director of Communications and Community Development[emailprotected]

Business Inquiries:Erandi De Silva, Ph.D.Forge BiologicsCo-Founder and Vice President of Product Development[emailprotected]

SOURCE Forge Biologics

https://forgebiologics.com

See the original post here:
Forge Biologics Strengthens Gene Therapy Leadership with Key Hires in AAV Manufacturing, Regulatory Affairs and Finance to Support Strategic Growth -...

Text size

Pfizer has spent $800 million to build the leading production platform for gene therapies, the company said on Tuesday in its second day of talks to show how it plans to become a fast-growing innovator. Within three years, the drug giant hopes to launch three potential cures for hemophilia and muscular dystrophyworth as much as $4 billion in annual revenue.

We are in an unrivaled position to go to market, said the head of Pfizers rare-disease business, Suneet Varma, in his Tuesday morning presentation. In another Tuesday talk, the companys immunology leaders highlighted coming breakthroughs for the tens of millions who suffer miserable autoimmune skin disorders.

Pfizer (ticker: PFE) is eager to recast itself as a high-multiple growth stock. By year-end, its slower-selling products will be spun off into a business run by Mylan (MYL). From that point, Pfizer thinks it can boost revenue by 6% a year, despite the patent expirations of nearly $20 billion worth of its products in the latter part of this decade. The expected growth will be fired up by 25 product launches, which Pfizer is explaining in two days of online talks.

After rising Monday, Pfizer stock was down 0.3%, at $36.91, in recent trading. The S&P 500 was up 0.4%.

A rare disease may affect only a few hundred thousand people, but there are a lot of these diseases. Cumulatively, they add up to 400 million people worldwide, said Varma, with fewer than 5% having approved treatments. Pfizer believes the rare-disease market will grow at a double-digit annual rate.

Clinical trials are under way for treatments for the bleeding disorders known as hemophilia A and hemophilia B. After treatment, none of the enrolled patients have had bleeding problems, including some in which more than a year has passed since dosing.

Pfizer hopes for approval and launch of its hemophilia B treatment in 2022. Peak annual sales could hit $500 million to $1 billion, says the company. The hemophilia A launch could happen in 2023 and eventually exceed $1 billion in annual sales.

The gene-therapy treatments can require a short hospital stay. But the possibility of a cure will inspire 30% to 40% of hemophilia patients to seek treatment, Pfizer predicts.

A rival in hemophilia A gene therapy, BioMarin Pharmaceutical (BMRN), had a head start over Pfizer in clinical trials. But last month, the U.S. Food and Drug Administration rebuffed BioMarins application for approval and demanded additional follow-up of its patients for a period that will extend through the end of 2021.

The FDA hasnt asked Pfizer to change its design for the hemophilia A trial that the company will begin in a few weeks, said the chief medical officer of the rare-disease unit, Brenda Cooperstone,

There were some worrisome immune reactions among boys in the continuing clinical trial for Pfizers gene therapy for Duchenne muscular dystrophy. But after adjustments in the trials preparatory regime, Pfizer says there have been no more of those events. The treatment showed promising efficacy in early phase trials.

The pivotal Phase 3 muscular dystrophy trial will begin within weeks, with the first data expected in 2022. Pfizer hopes for a launch in 2023 of a muscular dystrophy therapy that would be used by 30,000 people a year in the U.S. and Europe, generating over $2 billion in annual revenue. to reach that goal, Pfizer is racing with Sarepta Therapeutics (SRPT).

In addition to its rare-disease treatments, Pfizer plans to launch four products for autoimmune disorders by 2025. Two of the products would treat atopic dermatitis, which causes painful itching in up to 30 million Americans. Only a portion of those patients get any treatment today.

Based on successful clinical trials, sufferers of the skin disease can look forward to breakthrough treatments, similar to those launched in recent years for psoriasis, says Pfizers inflammation and immunology business head, Richard Blackburn.

Corrections & Amplifications

Pfizers gene-therapy treatments can require short hospital stays. An earlier version of this article incorrectly said the treatments are like bone-marrow transplants, which require months of grueling hospital care.

Write to Bill Alpert at william.alpert@barrons.com

Read the original:
How Pfizer Plans to Lead the Industry in Gene Therapies - Barron's

BOSTON, Sept. 15, 2020 /PRNewswire/ --GreenLight Bioscienceshas received a $3.3 million grant from the Bill & Melinda Gates Foundation to develop new mRNA-based gene therapies for Sickle Cell Disease and other global health challenges.

The funding will support GreenLight's research and testing of affordable therapies using the company's novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for Sickle Cell Disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle Cell Disease is a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. More than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens including blood transfusions and bone marrow transplants are costly, invasive, and impractical for treating large segments of affected patient populations.

"Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally," said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. "Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access. We are grateful for the Gates Foundation's support to advance novel gene editing approaches for populations in which those therapies are currently out of reach."

Gene editing therapies hold significant promise in the treatment of Sickle Cell Disease since it is a disorder caused by gene mutation. Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to the disease.

Once the therapy candidate is validated and moves into clinical use, GreenLight Biosciences' biomanufacturing platform will accelerate production of affordable treatments at scale. "Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population. That capability does not yet exist in the market, but GreenLight's end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines," Ortega-Valle added.

About GreenLight Biosciences, Inc.GreenLight is a bio-performance company with a unique, cell-free production platform that delivers high-performing RNA solutions to human, plant and animal challenges. GreenLight develops RNA products for plant and life science applications, and collaborates with industry leaders to advance vaccine development, pandemic preparation, crop management, and plant protection. The cutting-edge, natural platform delivers higher-quality RNA at a lower cost and higher speed than was ever before possible. The GreenLight team values diversity, inclusion, and equality and promises to use collaboration to remain scientifically imaginative and passionately focused on making a difference in the world. For more information, visithttps://www.greenlightbiosciences.com/.

SOURCE GreenLight Biosciences

Go here to read the rest:
GreenLight Biosciences Receives $3.3 Million Grant to Develop Sickle Cell Disease Cure Using mRNA Gene Therapy - PRNewswire

This is paid-for content from our commercial partners.Find out more

Gene therapies are set to revolutionise healthcare by treating diseases at the genetic level. They address the underlying cause of disease and can restore a patient to normal or near normal health. As one-time, personalised treatments, gene therapies have the potential to transform current care pathways by offering eligible patients durable outcomes when successful.

Sponsored by

They offer a one-time intervention, when often the alternative is decades of chronic treatment and monitoring, benefitting both patients and carers. Health and social care systems could also benefit, when complex chronic care regimes can be eliminated or greatly reduced, with significant resources re-deployed across the health and social care system; pertinent in a post-covid-19 world. Of course, gene therapy, as well as the procedure needed to prepare patients to receive it, can have serious side effects and there must be a rigorous assessment of potential risks and benefits to identify the right patients for the treatment.

Additionally, the cost-effectiveness of gene therapies, depends very much on their capacity of delivery health and social savings over a patients lifetime. As many patients treated with gene therapies will be children and young adults, the treatment may deliver additional societal gains over decades.

There are currently over 950 companies worldwide developing Advanced Therapy Medicinal Products, with therapies being tested in 1052 clinical trials, as of Q3 2019.1 Many of these are gene therapies that may become available in the UK over the next five years.

Provision needs to be made urgently for aligned regulatory assessment, health technology appraisal and NHS managed introduction, both in terms of infrastructure as well as reallocated budget. Life science companies also have a responsibility to set value-based prices and should consider alternative payment models and risk share agreements in collaboration with the NHS and government to further ensure value and affordability.

Gene therapies are positive and disruptive technologies that require whole system change to ensure that a post-Brexit NHS is at the forefront of provision rather than lagging behind its European neighbours. There is a window of opportunity for the MHRA with renewed responsibilities in 2021, as well as the National Institute for Health and Care Excellence currently conducting its Methods Review to ensure both regulatory and appraisal systems are aligned for optimal assessment of modern medicines, including gene therapies.

NICEs review of the methods is highly significant as it will set the framework for how England and Wales will provide access to new and breakthrough medicines. Areas of focus should include wider recognition of gene therapies and their benefits, including the one-off treatment offer to patients, gains to the health and social care system, plus pragmatic ways to address inherent lifetime uncertainty.

Crucially, for paediatric and young adult patients, there is an additional challenge the NICE methods review needs to resolve. High economic discount rates used in the health economic assessment process by NICE has a prejudicial impact on the cost-effectiveness of treatments that are intended to offer benefits over decades, such as gene therapies. This issue can be easily addressed if Treasury guidance for utilising lower discount rates is adopted.

A successful NICE Methods Review would ensure that the UK has a fit-for-purpose medicines assessment process. This will help to achieve world-leading status for bringing new medicines such as gene therapy to patients and will sustain UK-based research and development investment. This should be prioritised as the end of the EU exit transition period approaches, to ensure the governments vision of a vibrant post-Brexit economy, fuelled by science and technology, allows the UK to lead the world in healthcare innovation.

1. Alliance for Regenerative Medicine. Quarterly regenerative medicine sector report Q3. 2019. Available at: https://alliancerm.org/?smd_process_download=1&download_id=5556 [Accessed 11 February 2020].

The rest is here:
Gene Therapy will the NHS lead or follow? - Health Service Journal

Helixmith said Wednesday that it has submitted a phase 3-3 extension study protocol of its gene therapy Engensis (VM202) for diabetic peripheral neuropathy (DPN), to the U.S. Food and Drug Administration.

The company set a one-year follow-up period to confirm the pain reduction and safety of VM202 in treating DPN. DPN is one of the most common complications of diabetic diseases. About 30 million Americans have diabetes, and 28.5 percent of them develop DPN. Among the DPN patients, 40 to 50 percent experience painful symptoms.

The study's primary endpoint is the average pain reduction effect measured and recorded in the pain diary over the last week of the sixth month from the first injection.

The study will be carried out with patients who had not taken Gabapentinoids, such as Pregabalin and Gabapentine, in 15 research laboratories across the U.S., including Northwestern University in Chicago.

"Existing painkillers for DPN patients are not a fundamental treatment for the disease as they only relieved pain while often accompanying serious side effects and high addiction," Helixmith CEO Kim Sun-young said. "We will try our best for the success of phase 3-3 clinical trials as well as the ongoing phase 3-2 study."

FDA recognized the clinical results of Engennsis and designated it as an advanced regenerative medicine advanced therapy (RMAT) in 2018, the company said. RMAT is a new system designed to speed up the development and approval of innovative regenerative therapies. It gives special privileges of the U.S. fast track and priority or accelerated screening.

Read more here:
Helixmith eyeing P3-3 clinical trials of gene therapy Engensis - Korea Biomedical Review