Ultragenyx gene therapy Orphan Drug in U.S. for rare metabolic disorder

 

• The FDA designates Orphan Drug status to Ultragenyx Pharmaceutical's (NASDAQ:RARE) UX701 for the treatment of Wilson disease, a rare metabolic disease in which the body is unable to transport copper, thus accumulation of copper in the liver and other tissues. It is caused by mutations in the ATP7B gene, which results in deficient production of ATP7B, a protein that transports copper.
• UX701 is an investigational AAV type 9 gene therapy designed to deliver stable expression of the ATP7B gene. Currently in late preclinical development, and an investigational new drug application is expected to be filed with the FDA by the end of this year.
• https://seekingalpha.com/news/3643098-ultragenyxs-gene-therapy-orphan-drug-in-u-s-for-rare-metabolic-disorder