The Texas-based company AveXis has closed a new financing round that will help to develop the company’s gene therapy program related to Spinal Muscular Atrophy (SMA). The recent funding round came from PBM Capital Group, a private investment firm focused on healthcare and life science investments, which will also join the clinic-ready synthetic biology platform company’s Board of Directors.
“Securing this capital not only provides AveXis with additional resources to advance its SMA gene therapy program both commercially and clinically but also aligns well with the company’s strategy of integrating entrepreneurship and philanthropy,” said the Director of Scientific and Corporate Affairs at AveXis, Mindy Du.
SMA is a genetic disease, characterized by progressive weakness of the lower motor neurons, and it kills more children than any other disease in the world today. With this funding, AveXis will be able to lead the Phase 1 clinical trial for chariSMA, its gene therapy product, in the beginning of the second quarter of the year.
“AveXis and Nationwide Children’s are continually striving to aggressively advance chariSMA to market for patients afflicted with SMA. Our focused efforts have resulted in the signing of an exclusive license to ReGenX’s NAV rAAV9 vector for SMA and the initiation of a clinical trial within the coming months,” said Minna Du.
AveXis’ Chief Scientific Officer Allan Kaspar has explained that this trial represents “the best of ‘bench-to-bedside’ translational research.” It will test the safety of the company’s lead therapy and will able to show early measures of efficacy. Dr. Jerry Mendell, a professor at The Research Institute at Nationwide Children’s Hospital, will lead the clinical trial.
AveXis aims to create innovative treatments for people with unmet medical needs, but it is specially focused on SMA, a disease caused by a genetic defect in the SMN1 gene.