Read Genethon's Newsletter for advances in novel AAV vectors and clinical progress in Duchenne muscular dystrophy, limb-girdle muscular dystrophy and Crigler-Najjar syndrome

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), reported clinical trial updates for its best-in-class Duchenne muscular dystrophy (DMD) gene therapy and announced a unique collaboration with a US-based company to improve precision targeting of gene therapies. Read about these and other milestones in Genethon’s latest Newsletter.

In his CEO Commentary, Frederic Revah, Ph.D., observes genetic medicine has proved its potential to treat rare as well as more common diseases, but to achieve the goal of ensuring broad patient access requires overcoming formidable challenges.

Other Newsletter highlights include:

• A collaboration with Ampersand Biosciences of Boston, MA, to engineer a new generation of AAV capsids with superior tissue specificity, unlocking the full therapeutic potential of AAV-based gene therapy.
• Long-term efficacy and safety confirmation of GNT0004, a low dose microdystrophin gene therapy in Phase 3 trials for DMD.
• Promising efficacy and safety data from the first patients treated with ATA-200, a gene therapy for limb-girdle muscular dystrophy R5.
• A scientific publication detailing the use of basket clinical trials to accelerate development of treatments for rare diseases.
• A report on the second summit of GenoTher, France’s government-backed biocluster dedicated to accelerating development of genetic medicine, featuring international leaders from academia, clinical research, industry, investment, and regulatory agencies.

Read the full Newsletter and learn more about Genethon on its website (www.genethon.com).

About Genethon

A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory created by the AFM-Telethon. The first gene therapy drug, to which Genethon contributed, has been approved for marketing for spinal muscular atrophy. With more than 240 scientists and experts, Genethon's goal is to develop innovative therapies that change the lives of patients suffering from rare genetic diseases. Fifteen gene therapy products resulting from Genethon's research, or to which Genethon has contributed, are currently undergoing clinical trials for diseases of the liver, blood, immune system, muscles, and eyes. Others are preparing for clinical trials over the next five years. Discover Genethon’s pipeline: https://www.genethon.com/our-pipeline

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