April 9, 2026
CRISPR/Cas9: Genethon Unveils an Innovative Strategy to Increase Utrophin in Duchenne Muscular Dystrophy
In a study published in March 2026 in the journal Molecular Therapy, scientists from the “ Gene Editing ” team , led by Dr. Mario Amendola, unveil an innovative approach to genome editing aimed at sustainably increasing utrophin expression. This protein could compensate for the absence of dystrophin in people with… (read more)
March 27, 2026
Sickle cell disease: ANSM gives green light to the DREPAMIR clinical trial. Genethon, a key contributor to the development of the innovative lentiviral vector
The French National Agency for Medicines and Health Products Safety (ANSM) has authorized the start of the Phase I/II DREPAMIR clinical trial for gene therapy treatment of sickle cell disease, a genetic blood disorder that affects millions of patients worldwide.
(read more)February 3, 2026
Limb‑Girdle Muscular Dystrophy: First Three Children Treated with ATA‑200 Gene Therapy in on-going LGMD‑R5 Clinical Trial, and extension of Partnership with The Dion Foundation
Atamyo Therapeutics, a clinical-stage biotechnology company, spun of Genethon, focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, announced that the first three patients have been treated with its ATA‑200 gene therapy in a phase 1b/2 clinical… (read more)
Genethon studies cardiotoxicity associated with high doses of AAV vectors in gene therapy
In a study published on December 24, 2025 in the Journal Molecular Therapy, scientists from Isabelle Richard’s laboratory evaluated the cardiac effects associated with the administration of high doses of AAV vectors used in gene therapy.
(read more)October 1, 2025
Genethon to Present Long-Term Follow-Up of Duchenne Muscular Dystrophy Patients Treated in Its Phase 1/2/3 Trial at the European Society of Gene and Cell Therapy 32nd Annual Congress October 7 – 10, 2025
The oral presentation October 9 will focus on patients treated in the dose escalation portion of Genethon’s all-in-one Phase1/2/3 clinical trial. The Phase 3 trial is under way in Europe with the first patients included in France with Genethon’s low-dose micro-dystrophin gene therapy GNT0004.
ESGCT 2025: Genethon at the forefront of gene therapy!
From October 7 to 10, 2025, Seville will host the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), the unmissable gathering of the world’s leading gene therapy experts.
(read more)July 28, 2025
Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
Phase 3 trial clearance is based on the Phase 1/2 results demonstrating safety and efficacy of the 3×10¹³ vg/kg dose of microdystrophin, which is lower than doses used in other gene therapies for Duchenne muscular dystrophy (DMD).
The double blind trial will initiated in the UK and France beginning in August and… (read more)
May 17, 2025
Duchenne Muscular Dystrophy: Genethon presents Two-Year consolidated results of the GNT0004 Gene Therapy
Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne muscular dystrophy, at the American Society of Gene and Cell Therapy, in New Orleans, May 13 – 17, 2025.
(read more)May 16, 2025
New advances in the treatment of ALS: Genethon’s DNA bank involved in the trial
Promising results from the European clinical trial MIROCALS (Modifying Immune Responses and Outcomes in ALS) have been published in The Lancet, opening up new prospects for the treatment of Amyotrophic Lateral Sclerosis (ALS)
(read more)May 15, 2025
GenoTher Summit 2025: a key event to shape the future of gene therapy
Genethon, co-founder of GenoTher, is pleased to invite you to the first GenoTher Summit 2025 on June 11 in Evry, an international event at the heart of innovation in gene therapy. Organized as part of the official launch of GenoTher, the biocluster of excellence dedicated exclusively to gene therapy and… (read more)
May 13, 2025
Genethon arrives in force at ASGCT 2025 with 12 communications, including 5 oral presentations
Genethon will participate to the 28th annual meeting of the American Society of Gene and Cell Therapy (ASGCT) from Tuesday, May 13 to Saturday, May 17 in New Orleans. Scientists, researchers, engineers, and medical affairs experts will present their work and advancements through five oral presentations and seven posters,… (read more)
March 20, 2025
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.
(read more)March 7, 2025
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)
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… (read more)
December 17, 2024
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.
(read more)