December 19, 2024
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 myopathy. (read more)
November 26, 2024
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)
March 4, 2024
Clinical results of the gene therapy trial in myotubular myopathy: efficacy on respiratory and motor functions in this extremely severe disease, challenges ahead
February 7, 2024
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”. (read more)
November 30, 2023
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 of… (read more)
November 21, 2023
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. (read more)
November 17, 2023
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. (read more)
October 27, 2023
First clinical results of a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy presented at ESGCT Congress
September 15, 2023
Genethon assesses a therapeutic potential of a Dual AAV vector approach for Duchenne muscular dystrophy
September 11, 2023
Genethon’s Optidys project rewarded by BpiFrance as part of France 2030
May 30, 2023
Research work carried out at Genethon rewarded at the 26th edition of the ASGCT
February 24, 2023
