Press releases

Media contact: Stéphanie Bardon – communication@genethon.fr – 01.69.47.12.78

AveXis Enters into Licensing Agreement with Genethon

AveXis, Inc. (NASDAQ: AVXS) and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA). (read more)

Genethon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

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First promising results towards the treatment of myotubular myopathy

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A gene therapy alliance in X-linked chronic granulomatous disease with Orchard Therapeutics

Orchard Therapeutics (“Orchard”), a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies announces today a strategic alliance with Généthon, to develop gene therapy for X-linked chronic granulomatous disease (“X-CGD”)…. (read more)

Audentes Therapeutics announces dosing of first patient in ASPIRO for the treatment of X-Linked Myotubular Myopathy

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Spark Therapeutics Enters into Licensing Agreement with Genethon

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Restore muscle strength in Duchenne muscular dystrophy through microdystrophin

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New partnership for the treatment of Duchenne muscular disease (DMD)

Sarepta Therapeutics, a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, have signed a gene therapy research collaboration to jointly develop treatments for Duchenne… (read more)

New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

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Creation of YposKesi

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Genethon and CRISPR Therapeutics announce Research Collaboration

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Genethon: a gene therapy drug being tested in the US

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ASGCT 18th Annual Meeting 2015: Ana Buj Bello receives the Outstanding New Investigator Award

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Selecta and Genethon Collaborate to Create Next Generation Gene Therapies Using Selecta’s Synthetic Vaccine Particle Platform

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Evidence of efficacy of gene therapy in rodents affected by a rare genetic liver disease, Crigler-Najjar syndrome

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