An upstart biotech based in Novato, CA and focused on developing new drugs for rare diseases has hauled in an impressive $45 million A round from some high-profile venture backers. TPG Biotech and Fidelity Biosciences co-led the round for Ultragenyx Therapeutics. They were joined by European investor HealthCap and Pappas Ventures.
"They believe in the concept that a tightly focused company of committed rare disease development specialists will be able to create value as well as bring a series of novel, life-changing therapeutics to patients affected by rare and ultra-rare disorders," said Emil D. Kakkis, M.D., Ph.D., the CEO of Ultragenyx. Kakkis is a veteran developer who completed an 11-year stint as chief medical officer at BioMarin. He founded the company a little more than a year ago.
Ultragenyx's lead product, UX-001, is described as a first-in-class therapy for treatment of Hereditary Inclusion Body Myopathy and is expected to enter the clinic later this year. John Klock, M.D., and William Aliski have also invested in the company.
- here's the Ultragenyx release
Special Report: Top 15 Biotech Venture Capital Deals of 2010