Spinal muscular atrophy : AveXis enters into licensing agreement with Genethon

AveXis, Inc. and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).

In 2009, Genethon and the Institute of Myology published the first results of gene therapy in spinal muscular atrophy mice. The French researchers, led by Martine Barkats (CNS Gene Transfer & Biotherapy of Motor Neuron Diseases at the Myology Centre for Research, Institute of Myology), had the effectiveness of the vector AAV9-SMN and its administration Intravenous administration of auto-complementary AAV-9 allows transgenic transmission to molecular therapy of adult motor neurons (Intravenous Administration of Self-Complementary AAV-9 Enables Transgene Delivery to Adult Motor Neurons Molecular Therapy. 2009 Jul;17).

“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.” 

Under the terms of the agreement, Genethon granted AveXis a license to patents in the U.S., Europe and Japan, for the AAV9 SMN product and in vivo gene therapy delivery of AAV9 vector into the CNS using intrathecal or intravenous routes of administration for the treatment of SMA.

This new agreement reinforces the fact that Genethon is today a key player in the development of gene therapy for rare diseases worldwide.