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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

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

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”). Généthon is a non-profit research and development organization whose mission is to develop gene therapies for orphan … [Read more]

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 Audentes Therapeutics, abiotechnology company focused on developing and commercializing gene therapy products for patients  living with serious, life-threatening  rare diseases, today announced it has commenced dosing of patients in ASPIRO, a Phase 1/2 clinical trial of AT132 for the treatment of … [Read more]