Company intelligence / Profile preview
Généthon
Évry, Île-de-France, Francegenethon.com ↗
- Ownership
- Private
- Employees
- ~150
- Development stage
- Other
01
Overview
Généthon has validated gene therapies in the clinic for multiple rare diseases, including initial commercial success with spinal muscular atrophy (contribution to Zolgensma). Clinical results in Duchenne muscular dystrophy and Crigler-Najjar syndrome show promising efficacy and safety, with advanced programs in Fanconi anemia, Pompe, and others. Over 20 candidates are in preclinical or clinical development[1][6][7].
- Therapeutic areas
- HematologyNeurologyRare Diseases
- Modalities
- Gene therapy
- Industry
- Biotech
02
Drug pipeline
21 assets20 more assets in the full pipeline.
03
Partnerships
Spark Therapeutics (Pompe disease)Sarepta Therapeutics (Duchenne muscular dystrophy)Hansa Biopharma (imlifidase + gene therapy for Crigler-Najjar)Samabriva (plant-based AAV manufacturing)Eukarÿs (biomanufacturing cost reduction)
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