AveXis Enters into Licensing Agreement with Genethon

World News: . []

CHICAGO and ÉVRY, France , March 13, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) and Genethon today announced they have entered into an exclusive, worldwide license agreement for 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.”

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 gene therapy delivery of AAV9 vector into the CNS using intrathecal or intravenous routes of administration for the treatment of SMA.  

“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,” said Frédéric Revah, Chief Executive Officer of Genethon. “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.”

The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 2 typically presents between six and 18 months of age, and those affected will never walk without support and most will never stand without support. SMA Type 2 results in mortality in more than 30 percent of patients by the age of 25.

Media Inquiries:Lauren BarbieroW2O Group646-564-2156 Investor Inquiries:Jim GoffAveXis, Inc.650-862-4134

Genethon Press Contact:Stéphanie Bardon+330169471278

More news and information about AveXis, Inc.; Genethon

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Globe Newswire: 20:01 GMT Tuesday 13th March 2018

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