Ultragenyx Pharmaceuticals, a biotechnology company focused on developing novel therapies for rare and ultra-rare genetic diseases, announced that the FDA accepted for review the Biologics License Application (BLA) seeking accelerated approval for its gene therapy product candidate UX111, the world-wide rights to which it acquired through a $30–million deal with Abeona Therapeutics in 2022. The drug relies on licensed foundational gene therapy technology created in the laboratory of Dr. James Wilson, MD, PhD, Emeritus Professor of Medicine at Penn Medicine.
UX111 has been designed to address Sanfilippo syndrome type A, a rare, inherited disorder in which affected individuals lack a specific enzyme needed to break down certain sugars, ultimately leading to severe health problems which often includes severe brain damage. Read more here.