GSK pass orphan gene therapy assets to Orchard Therapeutics

Under the strategic agreement GSK transfers its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for patients. GSK will receive royalties and commercial milestone payments related to the acquired portfolio. GSK and Orchard will exchange manufacturing, technical and commercial know how on the development of gene therapy medicines to ensure the success of the assets. GSK will continue to invest in the development of its platform capabilities in cell and gene therapies, with a focus on oncology. The acquisition of GSK’s programmes complements Orchard’s pipeline of clinical and preclinical gene therapies for primary immune deficiencies and inherited metabolic disorders.

The portfolio of gene therapy programmes Orchard has acquired includes: Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase deficiency (ADA-SCID), which leads to a lack of lymphocytes, approved by the EMA in 2016, two late-stage clinical programmes in ongoing registrational studies for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical programme for beta thalassaemia. Orchard will also acquire rights to exclusively license three additional preclinical programmes from Telethon/Ospedale San Raffaele upon completion of clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD) and globoid cell leukodystrophy (GLD). 

The agreement follows GSK’s strategic review of its rare disease unit and the Group’s ongoing prioritisation on programmes in respiratory and HIV/infectious diseases, as well as oncology and immuno-inflammation. 

Orchard Therapeutics will assume all obligations arising from GSK’s 2010 collaboration agreement with the Ospedale San Raffaele and Fondazione Telethon and from GSK’s collaboration agreement with MolMed, the exclusive manufacturer of Strimvelis.