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Shire, Boston Children's join forces against rare pediatric diseases

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Shire ($SHPG) has tapped the labs of Boston Children's Hospital in an effort to discover new therapies for rare pediatric diseases, in the latest announced collaboration for the Ireland-based drugmaker's fast-growing Human Genetic Therapies (HGT) unit based in Lexington, MA.

Shire HGT, which makes the company's rare disease therapies such as Elaprase and Replagal, revealed this morning that the company has made an upfront payment to the Harvard teaching hospital and made a three-year commitment to fund select research programs there. Shire wants any program it funds at the hospital to generate a therapy candidate in under three years from its start date. And the deal gives Shire an exclusive option to license therapy candidates from those programs it funds at Children's Hospital, which would then be eligible for additional payments based on development goals and royalties on potential sales from Shire.

Shire, Sanofi ($SNY) and GlaxoSmithKline ($GSK) are among a herd of drugmakers with major investments in the rare diseases field, which offers an array of unmet medical needs as thousands of orphan diseases are without approved therapies. And Shire's alliance with Children's Hospital follows a spate of such collaborations between academics and pharma companies that put industry dollars behind university labs with the goal of advancing science into the commercial realm, and for companies, to provide an ample source of new products.

"We are excited about the potential that the collaboration with Shire represents," said Dr. Alan Beggs, director of the Manton Center for Orphan Disease Research at Boston Children's, in Shire's release. "Boston Children's brings the research infrastructure to make the foundational discoveries about these diseases and our clinicians know the patients and their needs. Partnering with Shire's drug development capabilities represents a powerful combination that we hope will facilitate the development of new classes of therapy to ultimately benefit patients."

Shire HGT has actively sought external tie-ups to bolster its internal R&D efforts. In addition to the company's $521 million buyout of Germany's Jerini several years ago, Shire recently formed a $22 million discovery alliance with Telethon Institute of Genetics and Medicine in Naples to find new therapies for 13 rare diseases. And late last year Shire and Atlas Venture agreed to combine resources to support young developers of rare-disease drugs. 

- here's the release
- see GEN's coverage

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