Triangle Business Journal - by James Gallagher -- Pharmaceutical giant GlaxoSmithKline has signed a new alliance with two Italian organizations to research and develop new treatments for rare genetic disorders.
U.K.-based GSK (NYSE: GSK) will work with researchers at the San Raffaele Telethon Institute for Gene Therapy, a joint venture between Fondazione Telethon and Fondazione San Raffaele, to work to develop new treatments for ADA Sever Combined Immune Deficiency, a very rare immune deficiency, commonly referred to as “bubble boy” disease. The disease affects about 350 children worldwide.
Under the terms of the deal, U.K.-based GSK (NYSE: GSK) will gain exclusive licenses to develop and commercialize an investigational gene therapy for ADA-SCID, and GSK will co-develop six additional applications for ex vivo stem cell therapy using gene transfer technology developed by the San Raffaele Institute.
“This alliance is an important addition to GSK’s growing portfolio in rare diseases and advances the work our stem cell researchers have been doing to deliver transformative regenerative therapies,” said Dr Philippe Monteyne, head of development and chief medical officer for GSK Rare Diseases, in a news release. “Not only does the lead programme for ADA-SCID represent an opportunity to provide a treatment option for an under-served patient population, by combining our experience in manufacturing complex biological products with the pioneering research of world-leaders in gene and stem cell therapies, we have the chance to dramatically advance this field.”
GSK, which employs about 5,000 across the Triangle, has its North American headquarters in Research Triangle Park.
