|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).
“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.”
|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.