REGENX, AveXis to develop new treatment for SMA using NAV rAAV9 vectors

SMA is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons.

It is caused by a genetic defect in the SMN1 gene, which codes SMN, a protein necessary for the survival of motor neurons.

As part of the deal, REGENX granted AveXis an exclusive, worldwide license, with rights to sublicense its NAV rAAV9 vector for treatment of SMA disease in humans.

In return, REGENX will receive an up-front payment, certain milestone fees and royalties on net sales of products incorporating NAV rAAV9.

REGENX president and CEO Ken Mills said the company believes this exclusive license agreement is important to the successful development of NAV-based gene delivery treatments for patients with SMA.

"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," Mills said.

AveXis CEO John Carbona said the company is committed to the development of new treatments for patients with SMA using NAV-vector technology.

"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," Carbona said.

"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."