DRAFT SUBJECT TO FINAL REVIEW AND APPROVAL
REGENX BIOSCIENCES AND AVEXIS ENTER INTO LICENSE AGREEMENT FOR DEVELOPMENT OF TREATMENTS FOR SPINAL MUSCULAR ATROPHY USING NAV® rAAV9 VECTORS
Washington, DC and Dallas, TX REGENX Biosciences, LLC (REGENX) and AveXis, Inc. (AveXis) announce that they have entered into an agreement for the development and commercialization of products to treat Spinal Muscular Atrophy (SMA) using NAV rAAV9 vectors.
Under the terms of the agreement, REGENX granted AveXis an exclusive worldwide license, with rights to sublicense, to REG ENXs NAV rAAV9 vector for treatment of SMA disease in humans. In return for these rights, REGENX receives an up-front payment, certain milestone fees and royalties on net sales of products incorporating NAV rAAV9.
We believe this exclusive license agreement is important to the successful development of NAV-based gene delivery treatments for patients with SMA, said Ken Mills, President and CEO of REGENX. As a leader in gene therapy, we are pleased to be formally collaborating with AveXis which has assembled a world class team of scientific and clinical experts in SMA, led by Brian Kaspar, Ph.D. and his colleagues at Nationwide Childrens Hospital and The Ohio State University, who have demonstrated tremendous dedication to the development of innovative gene therapy treatments for patients with SMA.
AveXis is committed to the development of new treatments for patients with SMA using NAV-vector technology and we feel rAAV9 is the most promising vector to achieve this goal, something we like to call our special snowflake. We believe the unique properties of rAAV9 will allow us to effectively develop novel treatments, and is at the center of research being done at the Kaspar Laboratory in Columbus, Ohio, said John A. Carbona, CEO of AveXis. Everyone associated with our SMA program is very pleased to establish this agreement with REGENX, which provides an important foundation for our team to continue to develop novel therapies for patients with all types of SMA.
About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons. SMA is caused by a genetic defect in the SMN1 gene which codes SMN, a protein necessary for survival of motor neurons. SMA kills more infants than any other genetic disease in todays world.