News

High-throughput screening identification of two molecules as potential approaches for treating a form of limb-girdle muscular dystrophy (LGMD-R2)

I-Stem, Genethon and the Institute of Myology, laboratories of the Institute of Biotherapies created by the AFM-Telethon have collaborated on work recently published in the British Journal of Pharmacology of March 19th. Their objective: to identify a pharmacotherapy to treat LGMD-R2, a form of limb-girdle myopathy.

Gene therapy for sickle cell anemia: Genethon has contributed its expertise to the design of an innovative vector

In a press release published on March 5, 2025, the collaboration between SK pharmteco Cell & Gene Europe, AP-HP, AFM-Téléthon and the Imagine Institute for the production of lentiviral vectors as part of an innovative clinical trial on sickle cell disease was announced. A breakthrough to which Genethon has… (read more)

Ana Buj Bello, winner of the Léon Baratz Prize, Docteur Darolles for her research on a gene therapy to cure myotubular myopathy

Congratulations to Ana Buj Bello, Director of Research at Inserm and Head of the Neuromuscular Diseases and Gene Therapy team at Genethon, winner of the Léon Baratz, Docteur Darolles prize awarded on Tuesday 17 December by the French Academy of Medicine for her work on the development of a gene therapy to cure myotubular… (read more)

Long-term success of gene therapy in patients with Fanconi anemia

The results of a clinical trial conducted by Rocket Pharmaceuticals and the Spanish CIEMAT teams, in collaboration with Genethon, which helped design and develop the drug vector used in the trial, have demonstrated, for the first time, the efficacy and safety of gene therapy in patients with Fanconi anemia, a rare blood disorder.

GNT0004, where are we with this drug-candidate?

Since the presentation of positive results of gene therapy trial for Duchenne muscular dystrophy at Breakthroughs in Muscular Dystrophy organized by ASGCT in Chicago, Genethon’s CEO, Frederic Revah discusses how the drug candidate GNT004 has the potential of becoming best-in-class gene therapy for DMD (Duchenne Muscular… (read more)

 Clinical results of the gene therapy trial in myotubular myopathy: efficacy on respiratory and motor functions in this extremely severe disease, challenges ahead

The journal The Lancet Neurology published on November 15 the clinical results of the gene therapy trial with a drug candidate designed at Généthon in 24 children suffering from myotubular myopathy, a rare and very severe muscle disease.

The American organization Team Titin awards its first research grant funding to Genethon

Team Titin, a non-profit organization, will fund $50,000 to support the project “Generation and characterization of skeletal muscle organoids from TTN patients’ iPSC for therapeutic screening”.

Genethon evaluates a new therapeutic approach for glycogen storage disease III

In a study published in the « The Journal of Clinical Investigation » on November 28, Jérémy Rouillon, a research engineer at Genethon and Antoine Gardin, a pediatrician and PhD student at Généthon, in the Immunology and Liver Diseases team led by Giuseppe Ronzitti, have designed an innovative strategy that provides a truncated version… (read more)

NEWSLETTER : “We have multiple examples of how these technologies can be used to develop products that more efficient and safer with improved production technologies”

CEO Frederic Revah discusses how Artificial Intelligence is creating next-generation gene therapy vectors and reducing bioproduction costs in the latest issue of Genethon’s Newsletter.

Myotubular myopathy: gene therapy trial results demonstrate major improvement of motor and respiratory functions

The Lancet Neurology published on November 15 the clinical results of a gene therapy trial using a drug candidate developed at Genethon in 24 children suffering from myotubular myopathy, a rare genetic muscle disease.

First clinical results of a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy presented at ESGCT Congress

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Genethon assesses a therapeutic potential of a Dual AAV vector approach for Duchenne muscular dystrophy

In a study published in the International Journal of Molecular Sciences on July 13th, Genethon’s researcher Sonia Albini, deployed a dual AAV approach to deliver quasidystrophin.

Genethon’s Optidys project rewarded by BpiFrance as part of France 2030

Genethon’s Optidys project is rewarded in response to the call for proposals “Innovation in Biotherapies and Bioproduction” led by BPIfrance as part of France 2030.

Genethon researchers involved in the MAGIC international consortium

Three Genethon experts are participating in the MAGIC project (Next-generation Models and Genetic therapIes for rare neuromusCular diseases), dedicated to the development of innovative therapeutic strategies in muscular dystrophies.

”A milestone in gene therapy: proof of long-term efficacy”

Frédéric Revah, CEO of Genethon discusses Genethon’s major contribution to therapeutic innovation