RARE Rises on Completion of Rolling Submission for AAV Gene Therapy

Key Takeaways RARE rises 15.5% after finishing its rolling BLA submission to the FDA for DTX401 in GSDIa.DTX401 aims to treat the underlying cause of GSDIa, a rare disorder with no approved drug therapies.RARE's BLA is backed by phase III data showing reduced cornstarch use and improved glucose control.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 th ...