REGENXBIO Inc., a clinical-stage biotechnology exploring the curative potential of gene therapy based on its NAV Technology Platform, will receive an $80 million milestone payment from Novartis AG based on the achievement of $1.0 billion in cumulative net sales of Zolgensma.

Zolgensma uses REGENXBIO’s NAV AAV9 vector and was designed and developed by AveXis, Inc., known as Novartis Gene Therapies. In May 2019, Zolgensma was granted U.S. FDA approval for the treatment of spinal muscular atrophy (SMA) in pediatric patients who are less than two years of age, and has since been approved in Europe, Japan and other countries. REGENXBIO continues to receive tiered royalties on sales of Zolgensma. Since FDA approval in 2019, REGENXBIO has earned more than $140 million in royalties and commercial milestones related to Zolgensma.

“Zolgensma is a transformative, one-time gene therapy for the treatment of pediatric patients with SMA, a debilitating and potentially deadly disease, and the first approved gene therapy based on REGENXBIO’s NAV Technology. We believe that this achievement provides further validation of the broad potential of NAV Technology to improve the lives of patients in need,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO.  “We are pleased to have contributed to a therapy that has impacted the lives of over 700 patients with SMA and their families around the world.”