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Investors gamble $75M on Ultragenyx's plan to jump-start rare-disease player

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Ultragenyx CEO Emil Kakkis

Little Novato, CA-based Ultragenyx has gone to a long lineup of backers to add a whopping $75 million in Series B cash only two years after launching. The money brings its total raised so far to more than $100 million from a group of investors betting on Emil Kakkis' ambitious game plan for developing a pipeline of experimental therapies for rare and "ultra"-rare genetic diseases--one of the hottest fields in biotech.

Taking the lead, Adage Capital Partners joined an unusual mix of life sciences venture funds and largely public company investors in putting the bankroll together. T. Rowe Price Associates jumped in, while Jennison Associates brought in clients. Funds and accounts under management by subsidiaries of BlackRock, Sanofi-Genzyme BioVentures 1, Shire and additional public market funds also chipped in. And existing investors TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures all came in on the new round.

Kakkis--a veteran of BioMarin ($BMRN), where he worked on their genetic disorder drugs as chief medical officer--says he already has two drugs in midstage development, with a third, undisclosed, program right behind them. UX001 is a potential substrate replacement therapy--replacing a missing molecule--for hereditary inclusion body myopathy. UX003 is an enzyme replacement therapy for extraordinarily rare cases of mucopolysaccharidosis type 7 (MPS 7), which will enter a Phase I/II clinical study in MPS.

The new money, Kakkis tells FierceBiotech, will fund the company for three more years, enough time to complete late-stage testing and start jumping into the marketplace as he delivers on a plan to quick-march Ultragenyx from a startup into a fully fledged biopharma company. And he says he could take the company public as early as 2014.

The public markets have been an unwelcoming place for biotechs for several years now, but Kakkis says a company like his, following a path into rare diseases blazed by the likes of Genzyme and BioMarin, can succeed where others have failed.

"We haven't set any plans on partnering," says Kakkis. It's best to retain ownership of the products as much as possible, he adds, but he'll consider partnering if it can help expand the company and speed up its development plans.

Adds Kakkis: "We want to be a top rare disease company, an extremely efficient drug developer, smart and aggressive, which takes on products that seem too small but where the biology is clear."

Ultragenyx has 27 staffers right now, which Kakkis says will grow to about 35 to 40 next year.

- here's the press release

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