Ultragenyx snags another candidate for its rare disease drug pipeline

Fresh from adding $75 million in venture cash to its accounts, Novato, CA-based Ultragenyx has picked another rare-disease drug candidate, plucking a program from the Baylor Research Institute in Dallas.

The object of the biotech's desire is triheptanoin, a treatment for long-chain fatty acid oxidation disorders (FAOD). And they account for some ultrarare conditions. The developer says that the treatment is a specially designed synthetic compound that can "replace intermediate compounds of the tricarboxylic acid cycle, a key energy-generating process." A few thousand people are afflicted with such disorders, and about 100 are diagnosed with the four most common long-chain FAODs every year in the U.S.

"We believe that UX007 has the potential to benefit many patients with FAOD and other diseases of energy metabolism," said company CEO Emil D. Kakkis in a press release. "Recent published data suggest that as many as half of these FAOD patients die from this disease and are in need of better treatment options. This new program is another example of our focus on transformative treatments for rare diseases."

Kakkis, a veteran of rare-disease star BioMarin ($BMRN), has raised more than $100 million since its launch, gaining some eager backers who would like to capitalize on one of the hottest fields in biotech today. Several major operators like GlaxoSmithKline ($GSK) have jumped in over the last few years, drawn by tighter development cycles and the prospect of developing new drugs that can fetch blockbuster returns.

- here's the press release


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