News

Genethon gets the green light from the ANSM to start an innovative gene therapy trial for Duchenne muscular dystrophy

Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30^th of November the authorisation from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular… (read more)

Genethon welcomes the submission to EMA of Marketing Authorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON)

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A Genethon team succeeds in inhibiting the immune response linked to AAV, opening up the possibility of treating more patients by gene therapy

A Genethon team, working as part of an international collaboration, published the results of their work in Nature Medicine on June 1, 2020, opening up new perspectives for gene therapy. Using the IdeS enzyme, these researchers succeeded in inhibiting the immune response to AAV, which is caused by antibodies present as a result of… (read more)

Genethon welcomes the conclusive results of a gene therapy trial in Chronic Septic Granulomatosis, a rare disease of the immune system

Genethon, which developed the lentiviral vector used and sponsored initial clinical studies, is pleased with the conclusive results of the gene therapy trial underway in the United States and England in X-linked Chronic Septic Granulomatosis (X-CGD), a rare and severe immune dysfunction. Six of the nine patients are free of treatments related… (read more)

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)

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. 

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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.

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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. 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.

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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.

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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).

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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)

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.

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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)