Genethon Advances Groundbreaking Gene Therapies for Patients with Rare Genetic Diseases

Genethon’s Newsletter highlights the latest developments in gene therapies for diseases once considered incurable, including Duchenne muscular dystrophy

PARIS--(BUSINESS WIRE)--Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), began the new year with an R&D licensing deal for its gene therapy approach to Pompe disease and continues pivotal clinical trials of its best-in-class low dose micro-dystrophin gene therapy (GNT0004) for Duchenne muscular dystrophy (DMD). Read about these and other advances in Genethon’s latest Newsletter.

In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon’s 240 scientists and professional staff as they pursue new gene therapy treatments for rare genetic diseases, which disproportionately affect children worldwide.

Other Newsletter highlights:

• Genethon’s license agreement with AskBio, a gene therapy subsidiary of Bayer AG, for a proprietary technology developed by Genethon and integrated into AskBio’s gene therapy clinical trial candidate for Pompe disease.
• An update on Genethon’s pivotal clinical trial of its gene therapy for DMD.
• A research discovery that could lead to a combination treatment with gene therapy for DMD.
• Interviews with Dr. Revah in two global publications.

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 professionals, Genethon's goal is to develop innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen 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 in preparation for clinical trials over the next five years. More information at www.genethon.com.