Ultragenyx Pharmaceutical submits BLA to FDA for Sanfilippo Syndrome Gene Therapy, positive.

From Zacks Investment Research: 2024-12-20 12:38:19

Ultragenyx Pharmaceutical (RARE) has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A under the accelerated approval pathway. The BLA is supported by data from the ongoing Transpher A study, showing reduced HS levels in CSF, leading to improved cognitive development. RARE has also initiated a phase III study for GTX-102 in Angelman syndrome patients. Other key programs in RARE’s pipeline include UX143 for OI and UX701 for Wilson disease. RARE currently holds a Zacks Rank #3, with Castle Biosciences, CytomX Therapeutics, and Spero Therapeutics being top-ranked stocks in the sector.



Read more at Zacks Investment Research: RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy – December 20, 2024