GSK amplified on its plans to focus on rare diseases as part of its strategic mission. Their goal is to develop therapies for a list of around 200 rare diseases out of the estimated 6000-8000 known diseases. A GSK spokeman said "We will target those diseases where we realistically believe we can make a significant contribution; ideally we will be creating new medicines, but importantly by adding to the weight of scientific knowledge into rare diseases to the benefit of all working in this area." See also Bioreasearch Online.
As a first foray in the area, GSK announced a licensing agreement Monday with Fondazione Telethon and Fondazione San Raffaele for a novel gene therapy treatment for ADA Severe Combined Immune Deficiency (ADA-SCID) - a rare life-threatening immunodeficiency that affects 350 children worldwide. Working with the same two partners, GSK will also develop six more ex vivo stem cell treatments for a range of disorders including - metachromatic leukodystrophy (MLD) and Wiskott-Aldrich Syndrome (WAS), beta-thalasemia, mucopolysaccharoides type I (MPS), globoid leukodystrophy (GLD) and chronic granulomatous disorder (CGD). All of the disorders are caused by a single gene defect and have well understood molecular mechanisms. Clinical trials for MLD and WAS are currently underway.
"This collaboration is a breakthrough for gene therapy in rare diseases," said Professor Luigi Naldini, Director of San Raffaele Telethon Insititute for Gene Therapy, "We have spent years researching these promising new technologiess, but without GSK's experience in developing medicines, we would not be able to take all the steps towards making a potential therapy available to patients."
I'm actually both stunned and impressed that GSK can pursue a strategy that is such a divers set of niche diseases and presumably do so profitably. It would appear to be a Godsend to those patients with one of these very rare diseases that would certainly not otherwise be addressed in any large scale or systematic fashion.
Posted by Bruce Lehr October 22nd 2010