Remove Clinical Development Remove Gene Therapy Remove Genetics Remove Life Science
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Forge Biologics joins BGTC for new AAV gene therapies development

Pharmaceutical Technology

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Topic sponsors are not involved in the creation of editorial content.

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GSK and Wave Life Sciences partner to develop oligonucleotide therapeutics

Pharmaceutical Technology

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics. By Cytiva Thematic.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. How Can Study Protocols Be More Effective?

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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD

XTalks

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. According to the FDA, life expectancy for those with DMD has increased over the years, with some patients surviving beyond 30 years.

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Transcending expectations for cell & gene therapy development

Drug Discovery World

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall

XTalks

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

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2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

XTalks

The pandemic propelled the life science and healthcare sectors onto center stage, and they rose to the occasion against the most unprecedented health challenge in recent times. While COVID-19 has undoubtedly been the biggest story in the life science industry in 2020, it was a busy and positive year in many other areas.