Industrial partnerships


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


“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


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


« 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


“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


“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