GSK, foundations in gene therapy deal
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) received exclusive rights from two foundations in Italy to develop and commercialize a gene therapy for adenosine deaminase severe combined immune deficiency (ADA-SCID). The therapy, which uses an ex vivo approach to insert the correct gene into a patient's own bone marrow stem cells, has completed a Phase I/II trial for the indication. The deal covers research from the San Raffaele Telethon Institute for Gene Therapy, a joint venture formed by the two foundations, Fondazione Telethon and Fondazione San Raffaele, in 1995.
GSK and the foundations will also co-develop the ex vivo stem cell gene therapy for six other indications: metachromatic leukodystrophy (MLD); Wiskott-Aldrich syndrome (WAS); beta-thalassemia; mucopolysaccharoidosis type I (MPS-I); globoid leukodystrophy (GLD); and chronic granulomatous disorder (CGD). The MLD and WAS programs are in Phase I/II testing, while the others are in preclinical development. Foundazione Telethon will receive EUR 10 million ($13.9 million) up front and is eligible for undisclosed milestones. Further financial terms were not disclosed. ...