Ultragenyx Press Release – Ultragenyx Announces In-Licensing of Clinical-Stage Product Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorders

News | 01. 10. 2013

NOVATO, CA –January 10, 2013— Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, announced that it has in-licensed rights for triheptanoin,  a promising treatment for long-chain fatty acid oxidation disorders (FAOD), from Baylor Research Institute in Dallas, TX. Triheptanoin is intended as a substrate replacement therapy to restore deficient intermediates in the mitochondria and to enable energy metabolism in patients with FAOD and potentially other genetic metabolic disorders.