Ultragenyx Pharmaceuticals, a biotechnology company focused on developing novel therapies for rare and ultra-rare genetic diseases, plans to seek accelerated FDA approval for its gene therapy product candidate UX11, 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, the Rose H. Weiss Professor of Medicine at the University of Pennsylvania.
UX11 has been designed to address Sanfilippo syndrome type A, a rare, inherited disorder where 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.