GlaxoSmithKline in R&D pact with two Italian groups for rare genetic disorders; outlines rare disease strategy

UK pharmaceutical giant GlaxoSmithKline (LSE: GSK) yesterday revealed a new strategic alliance with two Italian groups to research and develop novel treatments to address rare genetic disorders, using gene therapy carried out on stem cells taken from the patient’s bone marrow (ex-vivo). The alliance capitalizes on research performed at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), a joint venture between Fondazione Telethon and Fondazione San Raffaele established since 1995.

Fondazione Telethon will receive an upfront 10 million euro ($14 million) from GSK and is eligible to receive further payments upon successful completion of a number of predetermined development milestones.

Under the terms of the deal, GSK will gain an exclusive licence to develop and commercialize an investigational gene therapy, for ADA Severe Combined Immune Deficiency (ADA-SCID) - a rare and life-threatening immune deficiency, which affects around 350 children worldwide. Phase I/II studies have demonstrated the potential of this treatment option to restore long-term immune function and protect against severe infections in children with ADA deficiency.