News

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

(read more)

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

Research work carried out at Genethon rewarded at the 26th edition of the ASGCT

Ai vu Hong, Research Fellow, and Laura Palmieri, PhD student, both members of Généthon’s progressive muscular dystrophies team, led by Isabelle Richard, received awards at the ASGCT 2023 congress held in Los Angeles from May 16 to 20.

Chronic septic granulomatosis: biomarkers predict the efficacy of gene therapy

In a gene therapy trial promoted by Genethon, teams from Necker children’s hospital working with teams from Inserm and Université Paris Cité, at Institut Imagine, have identified 51 biomarkers that could predict the success of this treatment.

“We made important achievements in 2022 but we still face key challenges in the year ahead”

Frédéric Revah, CEO of Genethon looks back on the year 2022 marked by major advances for gene therapy and presents the challenges ahead in 2023

Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

The gene therapy would represent a first-of-its kind treatment for this life threatening genetic disease.