Our therapies

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, 12 products derived from research conducted at Genethon are in clinical trials and 7 others should be in clinical trial within the next five years.

Neuromuscular diseases

Duchenne muscular dystrophy

GNT 0004

Design of the gene therapy product, proof of concept in a DMD canine model. Genethon sponsor of the ongoing multicenter clinical trial. Collaboration and license agreement with Sarepta.

Infant Spinal muscular atrophy

Zolgensma®

Proof of concept of the efficacy of a gene therapy strategy. In 2019, a treatment based on technologies from Genethon obtained marketing authorization in the US and then in France in 2020 for type 1 SMA and other types of SMA.
License Agreement to Novartis

Myotubular myopathy

Myotubular myopathy

Development of the gene therapy product, proof of concept in mouse and canine models. Product licensed to Astellas Gene Therapies.

Limb girdle muscular dystrophies

FKRP LGMD – GNT 0006

Gene therapy product developed at Genethon, proof of concept in animals.
Licensed to Atamyo for clinical development

Gamma sarco-glycanopathy – GNT 0007

Design of the gene therapy product, proof of concept in animals and pre-clinical development.
Will be transferred to Atamyo for clinical development.

Calpaïnopathy – GNT 0008

Gene therapy product design, proof of concept in animals, preclinical development.
Will be transferred to Atamyo for clinical development.

Immune system/blood diseases

Wiskott-Aldrich syndrome

Wiskott-Aldrich – N°EU/3/13/1196

Design of a gene therapy product, preclinical development then clinical development. Genethon promoter of the trial.
Orphan drug status from the European Medicines Agency.

Fanconi’s anemia

Fanconi anemia type A

Genethon contributed  to the design of the gene therapy product, proof of concept in animals and preclinical development.
Ciemat sponsor of the trial

Chronic septic granulomatosis

X-linked chronic granulomatosis

Contribution of Genethon to the design of the gene therapy product, proof of concept in animals, clinical design and Généthon sponsor of the French trial.
R&D collaboration with AP-HP and license agreement with Orchard.

Cori Forbes disease

Crigler-Najjar – GNT 0003

Gene therapy product design, proof of concept in animals, preclinical development. Genethon promoter of the trial.

Metabolic diseases

Pompe disease

Pompe disease

Vector design, proof of concept in animals, contribution to preclinical development.
Spark sponsor of the current trial.

Other diseases

Leber Hereditary Optic Neuropathy

Lumevoq®

Preclinical development. Licensed to Gensight, sponsor of the trial