Press releases

Media contact: Stéphanie Bardon – –

First in vivo proof-of-concept in Steinert’s myotonic dystrophy, a neuromuscular disease

Ana Buj Bello’s team, a researcher at Genethon, has made the proof-of-concept of a CRISPR-Cas9 approach in a mouse model of Steinert’s myotonic dystrophy, the most common neuromuscular disease in adults. (read more)

Towards a clinical trial for gamma-sarcoglycanopathy, limb-girdle muscular dystrophy

Isabelle Richard’s team, a CNRS researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, in mouse models of the disease. Based on these encouraging results, published in Molecular Therapy -… (read more)

Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD

Orchard Therapeutics Presents Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD (read more)

Genethon announces dosing of the first patient with Crigler–Najjar Syndrome

Genethon announces dosing of the first patient with Crigler–Najjar Syndrome, a rare liver disorder, in the European phase I/II gene therapy clinical trial – CareCN.
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. (read more)

Co-administration of AAV Vectors with SVP-Rapamycin Enables Vector Re-administration in Pre-clinical Gene Therapy Study Published in Nature Communications by Généthon and Selecta Biosciences

Généthon, a non-profit R&D organization founded by the AFM-Téléthon, and Selecta Biosciences, Inc. (Nasdaq:SELB), a clinical-stage biopharmaceutical company, today announced that Nature Communications has published their jointly authored paper entitled… (read more)

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

Genethon, a laboratory created by AFM-Telethon, starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease. The trial, sponsored by Généthon, will include 17 patients in four centres in Europe, and will assess safety and therapeutic efficacy of the gene therapy product developed by Genethon. (read more)

First promising results towards the treatment of myotubular myopathy

Genethon welcomes interim data of a phase I / II clinical trial with a gene therapy product developed in its laboratories. (read more)

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)

Restore muscle strength in Duchenne muscular dystrophy through microdystrophin

Gene therapy:  Microdystrophin restores muscle strength in Duchenne muscular dystrophy (read more)

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

Evry (France), 5 April, 2017. A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of… (read more)

Creation of YposKesi

AFM-Téléthon and the SPI fund, managed by Bpifrance under the Programme d’Investissement d’Avenir, are creating YposKesi, the first French industrial pharmaceutical company dedicated to producing gene and cell therapy drugs for rare  diseases. (read more)

Genethon and CRISPR Therapeutics announce Research Collaboration

EVRY, France, BASEL, Switzerland and CAMBRIDGE, Mass. – December 16th, 2015 – Généthon, a leader in the field of gene therapy treatments for rare diseases, and CRISPR Therapeutics, a biopharmaceutical company focused on developing transformative genebased medicines for patients with serious diseases, have today announced an… (read more)

Genethon: a gene therapy drug being tested in the US

Phase I/II clinical trial to enroll 10 patients with an immunodeficiency disorder at
three different U.S. locations. (read more)