GlaxoSmithKline offloads rare disease gene therapies to Orchard Therapeutics

GlaxoSmithKline is bidding adieu to its stable of gene therapies—it is transferring the assets to Orchard Therapeutics for a 19.9% stake in Orchard and an undisclosed amount in potential milestones and royalties.The portfolio includes the EMA-approved Strimvelis, which treats ADA-SCID, also known as “bubble boy syndrome,” a rare genetic disorder that causes immunodeficiency. Orchard will also pick up late-stage candidates for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical program for beta thalassemia. It will gain the rights to exclusively license three additional preclinical programmes from Telethon/Ospedale San Raffaele for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD) and globoid cell leukodystrophy (GLD).