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For the love of a child
LEXINGTON, Mass.—Targeted toward developing new treatments for rare pediatric diseases, Shire PLC, an Irish drug company with its Human Genetics Therapies program located in Lexington, and Boston Children's Hospital have signed a three-year research collaboration agreement.
The goal of this joint endeavor—which draws on Shire's development and commercialization skills and Boston's research expertise—is to develop therapies deemed to have the most potential to deliver a development candidate in less than three years.
The collaboration calls for Shire to make an undisclosed initial upfront payment to Boston, with the exclusive option to fund select research programs, while Boston is eligible to receive milestone payments and royalties on sales of related products from Shire. After three years, Shire will have the primary responsibility for any further clinical development and commercialization of products arising from the collaboration.
"As a leader in rare diseases, Shire is pleased to enter into this innovative collaboration with Boston Children's, which joins together leaders in academia and industry to advance pioneering research from the lab to the clinic," Dr. Philip J. Vickers, global head of research and development at Shire, stated in a news release. "Shire has a deep commitment to patients with rare diseases, and our developmental expertise, combined with Boston Children's research capabilities, should enable an acceleration of our discovery and development efforts. Our collaboration with Boston Children's is an ideal example of how we advance these goals. "
Vickers tells ddn he cannot disclose any of the rare diseases targeted, nor the dollar amount of the upfront payment.
"Our overall goal is to bring novel rare disease treatments to patients around the world, fulfilling an unmet need in the community," Vickers says. "Through this particular partnership, the goal is to have Boston Children's conduct research programs to identify potential development candidates that will transition into Shire's development pipeline over the course of the research collaboration."
Even if successful, it would appear the joint venture will only scratch the surface of a tremendous global need for treating a number of rare diseases.
"There are 7,000 rare diseases, and only a small fraction of those have therapies to treat them," Vickers says. "So right now, access to therapies is a large unmet need in the rare disease community. Shire is working to bridge that gap with novel products and services that treat those suffering from rare diseases."
He also recognizes the effort from patients' families in drumming up support and raising money toward treating their children's rare diseases—some of which most people have ever heard of.
"We have strong relationships with rare disease patient advocacy groups around the world," Vickers says. "At Shire, we believe that by partnering with these groups, we can achieve more and develop a deeper understanding of each other's perspectives and needs. It is integral to who we are as a patient-centric company."
Dr. Alan Beggs, director of the Manton Center for Orphan Disease Research at Boston, stated in a news release, "We are excited about the potential that the collaboration with Shire represents. 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."