Ultragenyx Announces License of Intellectual Property Related to the Treatment of Huntington’s Disease With Triheptanoin

News | 01. 07. 2015

Ultragenyx Pharmaceutical

NOVATO, Calif., Jan. 7, 2015 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced a license agreement with Inserm Transfert SA and Institut du Cerveau et de la Moelle Epiniere (ICM) for intellectual property related to the treatment of Huntington’s disease with triheptanoin (UX007).

“Huntington’s is a severe and lethal rare genetic disease,” commented Emil D. Kakkis, Ph.D., M.D., Chief Executive Officer and President of Ultragenyx. “Energy deficiency is believed to play a role in the pathophysiology of Huntington’s disease, and we are encouraged by data from a small pilot study suggesting improvement in brain energy metabolism after treatment with triheptanoin.”