Type of information: Licensing agreement
- AVXS-101 (adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene) - AAV9-SMN product and route of administration
Company: Avexis (USA - IL) Genethon (France)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: licensing
Disease: spinal muscular atrophy
- • On March 13, 2018, AveXis and Genethon 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).
- 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.