Industrial partnerships


New licencing agreement for the development of a gene therapy in Spinal Muscular Atrophy – March 2018

Genethon entered into licencing agreement with AveXis, a clinical-stage gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).

“Adding to our robust intellectual property estate, this agreement further strengthens our position by providing freedom to operate when using intravenous or intrathecal routes of administration to deliver the AAV9 vector into the CNS for the treatment of SMA,” said Sean Nolan, President and Chief Executive Officer of AveXis. “With our proprietary gene therapy, AVXS-101, currently being evaluated in patients with SMA in ongoing clinical trials in the U.S., and soon in Europe, we are pleased to have this exclusive worldwide agreement in place.”


Partnership signed for the development of an orphan drug for Chronic Granulomatous Disease – Dec. 2017

Genethon announces a strategic alliance with Orchard Therapeutics, a clinical-stage biotechnology company dedicated to developing gene therapies for treating immune and blood disorders, to develop a gene therapy for X-linked chronic granulomatous disease (“X-CGD”).

Mark Rothera, president and CEO of Orchard said: “This alliance with Généthon broadens our footprint in primary immune deficiencies. It adds an important second clinical stage program to our lead program OTL-101 in ADA-SCID (adenosine deaminase severe combined immunodeficiency) which is currently at the pre-registration stage. We look forward to working with Généthon and YposKesi and are excited about the much-needed patient benefits this alliance will deliver.”


Licence agreement for the development of a gene therapy in Pompe disease – Sept. 2017

Genethon entered into a licensing agreement with Spark Therapeutics, a US biotechnology company developing gene therapies for the treatment of rare eye, liver and neurodegenerative diseases, for the development and commercialization of an adeno-associated viral (AAV) gene therapy targeting the liver in Pompe disease.

« Capitalizing on our expertise and success to date in liver-directed AAV gene therapy, we look forward to collaborating with Généthon to develop a potential new gene therapy for a debilitating disease,” said Katherine A. High, M.D., president and chief scientific officer of Spark Therapeutics.


Collaboration for the development of a gene therapy in Duchenne Muscular Dystrophy – June 2017

Genethon and Sarepta Therapeutics, a biotechnology company specialising in the research and development of gene therapies in neuromuscular diseases, have started collaborating to develop a gene therapy for Duchenne muscular dystrophy (DMD).
They are working together to develop a treatment based on the use of a shortened version of the dystrophin gene with an AAV vector. This therapy developed by Genethon could be used for the majority of patients with Duchenne muscular dystrophy.

“Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industry’s most comprehensive franchise in DMD” stated Edward Kaye, Sarepta’s chief executive officer.


Collaboration to develop gene therapy treatment in Myotubular Myopathy disease – Feb. 2014

Genethon announces a strategic alliance with Audentes Therapeutics, Inc., a biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, to develop a gene therapy treatment as part of its projects for myotubular myopathy.

“Our agreement with Généthon is a significant step forward towards the development of a treatment for patients with this serious, rare disease,” said Matthew R. Patterson, President and CEO of Audentes. “The combination of Généthon’s expertise in the manufacturing and development of gene therapy products and Audentes’ world‐class orphan drug development team will allow us to rapidly advance this program.”

Press releases:

16 may 2018 : Audentes Announces Continuing Positive Data from First Dose Cohort of ASPIRO, a Phase 1/2 Clinical Trial of AT132 in Patients With X-Linked Myotubular Myopathy

21 september 2017 : Audentes Therapeutics announces dosing of first patient in ASPIRO for the treatment of X-Linked Myotubular Myopathy

5 april 2017 :  Nouvelle étape dans le traitement de la myopathie myotubulaire par thérapie génique

5 februar 2014 :  Audentes Therapeutics et Généthon s’associent pour développer un traitement de thérapie génique pour la myopathie myotubulaire


Partnership to develop a gene therapy in Leber’s Hereditary Optic Neuropathy – Feb. 2013

Genethon and GenSight Biologics, an innovative gene-therapy company working on neurodegenerative optical and central nervous system diseases, are in collaboration for the treatment by gene therapy of Leber’s hereditary optic neuropathy.
The therapeutic approach consists of reactivating the defective mitochondrial gene’s activity in the retina cells by administration of an AAV (Adeno-Associated Virus) carrying a copy of the mutated normal gene in the patient eye.
Phase III clinical trials are ongoing in collaboration with the Institut de la Vision.