AveXis in gene therapy patent deal with Genethon

AveXis, Inc. and Genethon announced they 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).

Under terms of the agreement, Genethon granted AveXis a license to patents in the US, 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.

“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 AveXis CEO Sean Nolan.

“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.”

Genethon CEO Frédéric Revah said: “Genethon is pleased to enter into this agreement with AveXis and to contribute to the efforts for the development of treatments for SMA patients who have urgent medical needs.

“It demonstrates Genethon’s capability to develop effective first-in-class technologies and the excellence of our translational research driven by the commitment to treat patients living with rare diseases.”