RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy

Ultragenyx Pharmaceutical (RARE) announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway.Earlier this year, the FDA aligned with Ultragenyx, after a thorough evaluation, on the use of cerebral spinal fluid (CSF) heparan sulfate (HS) as a well-characterized biomarker to support an accelerated approval pathway for mucopolysaccharidoses ...