Ultragenyx Completes Rolling Submission for AAV Gene Therapy

 Shares of Ultragenyx Pharmaceutical RARE were up 15.5% yesterday following the completion of the rolling submission of a biologics license application (“BLA”) to the FDA seeking approval for its AAV gene therapy, DTX401 (pariglasgene brecaparvovec), for the treatment of glycogen storage disease type Ia (GSDIa).

GSDIa is a rare, life-threatening metabolic disorder that leads to severe hypoglycemia (excess glycogen storage) and other complications. Managing the disease relies on frequent cornstarch intake, but current approaches are burdensome, and no approved pharmacologic treatments exist. GSDIa affects around 6,000 people in commercially accessible regions.

https://www.nasdaq.com/articles/rare-rises-completion-rolling-submission-aav-gene-therapy