Spinal muscular atrophy (SMA) ranks among orphan diseases as one of the most deadly and heartbreaking, as it disproportionately affects infants and young children, leading to a high mortality rate and progressively deteriorating quality of life. The disease, which is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons caused by a genetic defect in the SMN1 gene, is responsible for more infant death than any other genetic disease in the world today.
Dallas-based synthetic biology platform company AveXis, Inc. has made it its primary aim to take on this disease and seek to develop viable, effective NAV-based gene delivery treatments that can curtail its rapid progression and correct the gene coding issues that lead to the weakening of lower motor neurons. As part of their ongoing commercialization efforts, the company recently announced an exclusive agreement with Washington, D.C.-based REGENX Biosciences, LLC, which AveXis believes will advance further development and commercialization of SMA treatments using NAV rAAV9 vectors.
Read more about Spinal Muscular Atrophy.
The new agreement between the two companies will give AveXis an exclusive, worldwide license, with rights to sublicense, for the use of REGENX’s NAV rAAV9 vector for treatment of SMA disease in humans. REGENX’s NAV vector technology platform has established itself as a means of developing a new class of personalized therapies for a wide range of serious, unmet medical needs, such as SMA.
John A. Carbona, CEO of AveXis, explained in a recent press release how this new drug development technology agreement with REGENX will specifically enable AveXis to target type I SMA, the infantile form of the disease, which is most difficult to treat and leads to the most tragic outcomes: “AveXis is committed to the development of new treatments for patients with SMA using NAV-vector technology,” he said. “We feel rAAV9 is the most promising vector to achieve this goal. We call it our special snowflake, because, AAV9 has unique properties that allow us to develop novel targeted treatments for infants with SMA. We’ve named the product chariSM A® from Greek origin meaning ‘a gift of grace.'”
Mr. Carbona sees the new agreement as one that transcends a mere business transaction, and will lead to positive outcomes for patients and the companies alike: “Everyone associated with our SMA program is very pleased to establish this agreement with REGENX, which expands our leadership position in SMA gene therapy and supports a strong foundation for our team to continue to develop novel therapies for patients with all types of SMA,” he explained.
Read other news about AveXis:
[feed url=”http://bionews-tx.com/news/news-tags/avexis/feed” number=”5″ ]
In return for granting licensing rights to AveXis, REGENX will receive an up-front payment, certain milestone fees, and royalties on net sales of products incorporating NAV rAAV9, according to the press release. “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.”
While licensing agreements between biotech companies may not at face value play a direct role in treating or curing diseases, these types of business relationships and collaborations create foundations upon which companies such as AveXis and REGENX can advance and accelerate the valuable work they are performing to cure some of the most deadly diseases facing society in the 21st century.