Ultragenyx’s Gene Therapy for Wilson Disease Wins Orphan Drug Status

Ultragenyx’s gene therapy UX701 has been given Orphan Drug designation by the FDA for treating Wilson disease, a rare genetic disorder that causes excessive copper to accumulate in the organs.

Unlike current treatments, which patients must take for their entire lives, the gene therapy being developed by Ultragenyx is a one-time treatment that aims to restore normal copper metabolism in the liver, the company said.

UX701 is currently in late preclinical development, and the company expects to file an Investigational New Drug application before the year ends.