Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children's Hospital received Fast Track designation from the U.S. Food and Drug Administration for its scAAV9.CB.SMN gene therapy product for the treatment of spinal muscular atrophy (SMA). This new gene therapy product created by scientists at The Research Institute was granted Fast Track status after demonstrating preliminary effectiveness in mouse models of SMA, potentially addressing this unmet medical need.

"Fast track," an important hastened phase in the nation's drug review and approval process, signifies that the FDA can expedite the review and development of the scAAV9.CB.SMN gene therapy product which, in preclinical work, has shown to slow the progression of SMA symptoms.

SMA is the most common genetic cause of infant death. SMA is an autosomal recessive disease caused by a genetic defect in the SMN1 gene that encodes for the SMN protein. SMA manifests in various degrees of severity which all have in common general muscle wasting and mobility impairment.

The scAAV9.CB.SMN gene therapy product currently being developed by The Research Institute, received an IND approval in September 2013 to initiate Phase I clinical testing in SMA Type 1patients.The clinical trial is sponsored by The Sophia's Cure Foundation and AveXis/BioLife.

"Today's notice of Fast Track designation is welcomed news. This demonstrates to the families afflicted with SMA, the FDA's regulatory priority for addressing this orphan disease," said Chief Executive Officer John A. Carbona.