If you have an ACS member number, please enter it here so we can link this account to your membership. (optional)

ACS values your privacy. By submitting your information, you are gaining access to C&EN and subscribing to our weekly newsletter. We use the information you provide to make your reading experience better, and we will never sell your data to third party members.


Biological Chemistry

GSK Outlines Rare-Diseases Strategy

by Lisa M. Jarvis
October 25, 2010 | A version of this story appeared in Volume 88, Issue 43

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." These Italian organizations have developed gene-transfer technology that enables the development of gene therapy based on stem cells from a patient's own bone marrow. The ADA-SCID treatment has shown promise in a small Phase I/II trial.


This article has been sent to the following recipient:

Chemistry matters. Join us to get the news you need.