Press Releases
RSS feedA gene therapy based clinical trial for Fanconi anemia patients offers its first successful results
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. This clinical trial has been carried out by members of a … [Read more]
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 the press release
Genethon welcomes the first FDA-approved gene therapy for a neuromuscular disease and emphasizes its decisive role in this historic step
On May 24, the Food and Drug Administration approved Zolgensma®, a gene therapy drug for the treatment of spinal muscular atrophy developed by AveXis (Novartis). Genethon played a decisive role in the design of both the product and the route of administration of this first gene therapy for a neuromuscular disease, thanks to the pioneering … [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 – Methods and Clinical Development, the … [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 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. Read the press release More Information
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 the press release
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 “Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration”. Read the press release
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).
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 the … [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. At the 12-week timepoint, the first 3 children treated show early signs of efficacy. Read the press release
