Boston Children’s Hospital and Shire, a specialty biopharmaceutical company based in Ireland, have signed a three-year, broad research collaboration in rare diseases.

The goal of the collaboration is to develop novel therapies to treat a number of rare pediatric diseases with high unmet medical need, thereby leveraging Boston Children's research expertise and Shire's development and commercialization capabilities. The partnership underscores Shire's long-term commitment to bring innovative therapies to patients with rare diseases worldwide.

Shire will make an initial upfront payment to Boston Children's and will have the opportunity to fund selected research programs. The emphasis will be on opportunities that have the potential to deliver a development candidate in less than three years from project initiation. Following the completion of these programs, Shire will have an exclusive option to enter into a licensing agreement for each program.

Upon entering into any subsequent licensing agreement, Boston Children's will be eligible to receive certain development and commercial milestone payments, as well as royalties on product sales from Shire. Shire will have the primary responsibility for any further clinical development and commercialization of products arising from the collaboration. The agreement represents a flexible structure that aligns the parties according to their strengths.

"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," said Dr. Philip J. Vickers, global head of R&D, Shire HGT. "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."

Alan Beggs, director of the Manton Center for Orphan Disease Research at Boston Children's, added, "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."