—GSK Outlines Rare-Diseases Strategy “” Months after creating a unit dedicated to rare diseases, GlaxoSmithKline has unveiled its strategy for developing drugs for some of the more than 6,000 diseases that afflict a relatively small number of people. Established in February, the rare-diseases unit comprises 30 people who will work with drug discovery scientists at GSK while also looking outside the company's labs for partnership opportunities. In a conference call with journalists last week, Marc Dunoyer, head of the rare-diseases unit, said research will be focused on 200 diseases within four therapeutic areas: metabolism and inherited disorders, central nervous system and muscle disorders, immunoinflammation, and rare malignancies and hematology. While GSK outlined the strategy for the unit, it also announced its fourth partnership in rare diseases. The British firm will pay Fondazione Telethon and Fondazione San Raffaele $14 million for access to a gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID), also known as "bubble boy disease."
by Lisa M. Jarvis |
October 25, 2010