Our pipeline

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.

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

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

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.

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.

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.

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

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.

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.

A phase I/II trial is being carried out at Necker Enfant, with the aim of verifying the tolerability of GLOBE1 and the entire protocol (mobilization, conditioning & transplantation), as well as the efficacy criteria for blood count and % corrected red blood cells. 6 patients were included.

Study Details | Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE) | ClinicalTrials.gov

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.

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

Genethon developed the gene therapy product and contributed to its preclinical development.

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.