For the love of a child

Shire, Boston Children’s Hospital partner on treatments for rare pediatric diseases

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LEXINGTON, Mass.—Targeted toward developing new treatmentsfor rare pediatric diseases, Shire PLC, an Irish drug company with its HumanGenetics Therapies program located in Lexington, and Boston Children's Hospitalhave signed a three-year research collaboration agreement.
The goal of this joint endeavor—which draws on Shire'sdevelopment and commercialization skills and Boston's research expertise—is todevelop therapies deemed to have the most potential to deliver a developmentcandidate in less than three years.
The collaboration calls for Shire to make an undisclosedinitial upfront payment to Boston, with the exclusive option to fund selectresearch programs, while Boston is eligible to receive milestone payments androyalties on sales of related products from Shire. After three years, Shirewill have the primary responsibility for any further clinical development andcommercialization of products arising from the collaboration.
"As a leader in rare diseases, Shire is pleased to enterinto this innovative collaboration with Boston Children's, which joins togetherleaders in academia and industry to advance pioneering research from the lab tothe clinic," Dr. Philip J. Vickers, global head of research and development atShire, stated in a news release. "Shire has a deep commitment to patients withrare diseases, and our developmental expertise, combined with Boston Children'sresearch capabilities, should enable an acceleration of our discovery anddevelopment efforts. Our collaboration with Boston Children's is an idealexample of how we advance these goals."
Vickers tells ddn hecannot disclose any of the rare diseases targeted, nor the dollar amount of theupfront payment.
"Our overall goal is to bring novel rare disease treatmentsto patients around the world, fulfilling an unmet need in the community,"Vickers says. "Through this particular partnership, the goal is to have BostonChildren's conduct research programs to identify potential developmentcandidates that will transition into Shire's development pipeline over thecourse of the research collaboration."
Even if successful, it would appear the joint venture willonly scratch the surface of a tremendous global need for treating a number ofrare diseases.
"There are 7,000 rare diseases, and only a small fraction ofthose have therapies to treat them," Vickers says. "So right now, access totherapies is a large unmet need in the rare disease community. Shire is workingto bridge that gap with novel products and services that treat those sufferingfrom rare diseases."
He also recognizes the effort from patients' families indrumming up support and raising money toward treating their children's rarediseases—some of which most people have ever heard of.
"We have strong relationships with rare disease patientadvocacy groups around the world," Vickers says. "At Shire, we believe that bypartnering with these groups, we can achieve more and develop a deeperunderstanding of each other's perspectives and needs. It is integral to who weare as a patient-centric company."
Dr. Alan Beggs, director of the Manton Center for OrphanDisease Research at Boston, stated in a news release, "We are excited about thepotential that the collaboration with Shire represents. Boston Children'sbrings the research infrastructure to make the foundational discoveries aboutthese diseases, and our clinicians know the patients and their needs.Partnering with Shire's drug development capabilities represents a powerfulcombination that we hope will facilitate the development of new classes oftherapy to ultimately benefit patients."

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