Media contact: Stéphanie Bardon – communication@genethon.fr – 01.69.47.12.78
June 26, 2019
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)
May 24, 2019
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)
February 25, 2019
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)
December 10, 2018
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)
October 5, 2018
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)
March 13, 2018
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)
February 12, 2018
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)
January 12, 2018
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)
December 15, 2017
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)
July 25, 2017
Restore muscle strength in Duchenne muscular dystrophy through microdystrophin
Gene therapy: Microdystrophin restores muscle strength in Duchenne muscular dystrophy (read more)
June 21, 2017
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)
April 5, 2017
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)
November 3, 2016
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)
December 18, 2015
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)
July 28, 2015
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)
