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Ultragenyx生物公司获7500万美元支持其罕见病研究

来源:生物谷 2012-12-23 21:07

2012年12月23日讯 /生物谷BIOON/ --位于加州诺瓦托市的Ultragenyx生物公司成立仅两年便获得了7500万美元的投资,以支持其在罕见遗传病领域的研究。

Ultragenyx成立后在罕见病领域取得了一系列出色的成果,例如公司现有的UX001有希望成为遗传性包涵体肌病的替代疗法;UX003则是针对于7型粘多糖病(MPS 7),目前正进行临床阶段I/II研究。同时,还有一种尚未公开的药物研究计划。(生物谷Bioon.com)

详细英文报道:

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.

 

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