Our pipeline

The teams at Genethon develop gene therapy products for rare diseases, in collaboration with academic and industrial teams in France and abroad. Several products derived from R&D at Genethon or to which Genethon contributed as part of a collaboration, are currently in the clinical trial stage. These trials are sponsored by Genethon or by partners in Europe or the United States. These therapies involve neuromuscular pathologies, immune system diseases, ocular and metabolic diseases. To date, 1 product derived from research conducted at Genethon is on the market, 12 products are in clinical trials and 5 others should be in clinical trial within the next 3 years. Find out more about our pipeline.

Neuromuscular diseases

Duchenne muscular dystrophy

Duchenne Muscular Dystrophy – GNT 0004

Genethon developed a gene therapy product in partnership with the Institute of Myology, the University of London and Nantes Gene Therapy Laboratory, and established a proof of concept in a canine DMD model.
Genethon is the sponsor of an ongoing international clinical trial.
A collaboration and licensing agreement was signed with Sarepta

Infant Spinal muscular atrophy

Spinal muscular atrophy – Zolgensma®

Proof of concept of a gene therapy strategy obtained and patented by Martine Barkats’ team at Genethon.
In 2019, a treatment based on these technologies gained marketing authorization in the US and then in France in 2020 for type 1 SMA and other types of SMA.
License agreement to Novartis

SMA-PME and Farber’s disease

SMA-PME and Farber's disease

A Genethon team is working to develop gene therapy for patients with SMA-PME and Farber’s disease.

Myotubular myopathy

Myotubular myopathy

Genethon developed a gene therapy product and initiated pre-clinical development which was then carried out, like clinical development, by Astellas Gene Therapies, to whom the product was licensed and who is the sponsor of the ongoing clinical trial.

Limb girdle muscular dystrophies


Genethon developed a gene therapy product, demonstrated its efficacy in animals and carried out the pre-clinical development of the product.
It was licensed to Atamyo for clinical development.

Gamma sarco-glycanopathy – GNT 0007

Genethon developed a gene therapy product and performed much of the pre-clinical development.
This product was licensed to Atamyo for clinical development and a clinical trial application is expected to be filed shortly.

Calpaïnopathy – GNT 0008

Genethon developed a gene therapy product that was licensed to Atamyo, which is responsible for the pre-clinical development initiated by Genethon.

Immune system/blood diseases

Wiskott-Aldrich syndrome

Wiskott-Aldrich syndrome

Genethon developed the gene therapy product, carried out preclinical development and then launched clinical development. As such, Genethon was the sponsor of two clinical trials, one in France and the other in the UK. Genethon is committed to the long-term monitoring of these trials, which will end in 2032.

Fanconi’s anemia

Fanconi anemia type A

Genethon developed a lentiviral vector, in collaboration with teams from Ciemat, sponsor of the trial, which is continuing today with a long-term follow-up study.

Chronic septic granulomatosis

X-linked chronic granulomatosis

Genethon developed a lentiviral vector and carried out clinical development in collaboration with APHP within the framework of two clinical trials of which it is the promoter and which are now closed for inclusion, one in France and the other in the UK.

Genethon also licensed the vector to Orchard Therapeutics, which is continuing its clinical development, with the aim of a trial in the United States.

Metabolic diseases

Crigler-Najjar syndrome

Crigler-Najjar syndrome

Genethon developed the gene therapy product.
An international clinical trial sponsored by Genethon is currently underway, as part of the European CureCN consortium

Pompe disease

Pompe disease

The product was licensed to Spark, which finalized the pre-clinical development, carried out the clinical development and is now the sponsor of the clinical trial started in 2021.

Cori Forbes disease

Cori-Forbes Disease

A team at Genethon is working on the development of a gene therapy product to restore the expression of the deficient enzyme in the disease.

Other diseases

Leber Hereditary Optic Neuropathy


This gene therapy product stems from the original work of Dr. Marisol Corral-Debrinski and Pr. José-Alain Sahel, carried out at the preclinical stage as part of a collaboration with Genethon, which also contributed to early clinical development.
This product was licensed to Gensight, sponsor of the clinical trial. Gensight is seeking marketing authorization for Europe.