GSK and Orchard Therapeutics have entered a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programs and access for patients. 

Under the agreement, GSK will become an investor in Orchard Therapeutics, receiving a 19.9% equity stake along with a seat on the company’s board. GSK will also receive financial considerations in the form of royalties and commercial milestone payments related to the acquired portfolio. GSK and Orchard will exchange manufacturing, technical and commercial insights and learnings on the development of gene therapy medicines to ensure the success of the assets.

Orchard Therapeutics is a clinical-stage gene therapy company based in the United Kingdom and United States, dedicated to transforming the lives of patients with rare diseases through innovative gene therapies. 

The portfolio of gene therapy programs Orchard has acquired includes: Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved by the EMA in 2016, two late-stage clinical programs in ongoing registrational studies for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical program for beta thalassaemia. Orchard will also acquire rights to exclusively license three additional preclinical programs 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, announced in July 2017, as part of the Group’s ongoing prioritization and strengthening of its pharmaceuticals pipeline with a focus on priority programmes in two current therapy areas, respiratory and HIV/infectious diseases, and two potential areas, oncology and immuno-inflammation.

Mark Rothera, chief executive officer, Orchard, said, “Acquiring this portfolio further advances Orchard’s vision to be a global, fully integrated company leading the field of gene therapy for rare diseases. The acquisition immediately expands our primary immune deficiency and inherited metabolic disorder franchises and adds the potential for other franchises in the future. At Orchard, we are committed to transforming the lives of patients with rare diseases through innovative gene therapies. We look forward to building upon the great achievements of GSK and its collaborators.  This acquisition and the planned transfer of the agreement with MolMed secure the continued development of GSK’s programs and leverages Orchard’s deep expertise and capabilities. In the two late stage programs MLD and WAS for example, the clinical data* are very encouraging and we look forward to continuing to progress development.”