January 9, 2020
Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy
Evry (France) – January 9th, 2020 – Genethon, a French laboratory dedicated to the design and development of gene therapy products for rare diseases, and Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, today announced an expansion of their collaboration to develop a gene therapy approach for Duchenne muscular… (read more)
September 9, 2019
A gene therapy based clinical trial for Fanconi anemia patients offers its first successful results
Madrid, 9 September 2019 – The prestigious journal Nature Medicine publishes the first results of a gene therapy based clinical trial in patients with Fanconi anemia. In this study the authors demonstrate for the first time the production of blood cells derived from the patients’ genetically corrected stem cells.
(read more)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. Six Patients Continue to Show Sustained Levels of Functioning Neutrophils After 12 Months and No Longer Receive Treatment with CGD-related Prophylactic Antibiotics.
Regulatory Discussions on Registrational Trial Design Planned for 2019.
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.
(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… (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)
September 21, 2017
Audentes Therapeutics announces dosing of first patient in ASPIRO for the treatment of X-Linked Myotubular Myopathy
Preliminary data from ASPIRO expected to be available in the fourth quarter of 2017
(read more)September 20, 2017
Spark Therapeutics Enters into Licensing Agreement with Genethon
Spark Therapeutics, a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced it has entered into a licensing agreement with… (read more)
July 25, 2017
Restore muscle strength in Duchenne muscular dystrophy through microdystrophin
Gene therapy: Microdystrophin restores muscle strength in Duchenne muscular dystrophy. Researchers from Genethon, the AFM-Téléthon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, after… (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… (read more)
