Funding to support development of rare-disease treatments

NOVATO -- Ultragenyx Pharmaceutical Inc. today announced the closing of a $45 million series A financing to develop treatments for rare disease.

The company told the Business Journal in April that it was pursuing the funding and planning to start clinical trials this summer. According to today's announcement, this funding will advance multiple rare disease product programs in the pipeline as well as the development of new product candidates and partnerships. The lead product, UX-001, a treatment of hereditary inclusion body myopathy, or HIBM, is expected to enter clinical trials in 2011.

The co-lead investors are TPG Biotech and Fidelity Biosciences, joined by European investor HealthCap and Pappas Ventures. Additional funding came from John Klock, M.D., and William Aliski, both experienced rare-disease company executives. Joining the Ultragenyx board of directors are Eran Nadav, Ph.D., 0f TPG Biotech, Ben Auspitz of Fidelity Biosciences and Mårten Steen, M.D., Ph.D. of HealthCap.

“I am pleased and excited to have found a set of investors with the insight to support the development of a new rare disease company with multiple product candidates,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president. "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."

Dr. Kakkis formed and funded Ultragenyx in April 2010, based on his experience in academia and more than 11 years at Novato-based BioMarin Pharmaceutical, during which time three rare-disease products were approved. He was chief medical officer of BioMarin.

The company will be located in Novato.

For more information on Ultragenyx, please visit the company’s website at