Originally published on April 1, 2014.
REGENX Biosciences, LLC and AveXis, Inc. announce that they have entered into an exclusive 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 REGENX’s 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 Children’s Hospital and The Ohio State University, who have demonstrated tremendous dedication to the development of innovative gene therapy treatments for patients with SMA.”
Cure SMA and NINDS are providing collaborative funding for CNS delivered AAV9-SMN gene therapy for SMA.
“My research team at Nationwide Children’s Hospital is excited to advance this promising cerebrospinal fluid delivery approach of AAV9-SMN to the clinic for SMA patients and we are extremely grateful to Cure SMA and NINDS for the support of this important work,” said Dr. Kaspar from Nationwide Children’s Hospital and The Ohio State University. “We stand committed to bring SMA experimental therapeutics to the clinic in the most rapid and safe manner.” “Development of therapies requires collaboration of academics, advocacy, industry, and government—no single party has the resources to do this alone. The collaboration between Dr. Brian Kaspar, Cure SMA, and the NIH is an exciting model in leveraging resources and expertise in the hope of accelerating therapy development for SMA,” said Dr. John Porter, PhD, Program Director at the National Institute of Neurological Disorders and Stroke.
Cure SMA would like to thank all those who have contributed funding to Cure SMA for this particular program, including special gifts from The Michael and Chandra Rudd Foundation, The Miller McNeil Woodruff Foundation, and The Jacob Isaac Rappoport Foundation.