GlaxoSmithKline (GSK) has agreed to transfer its portfolio of approved and investigational rare-disease gene therapies to Orchard Therapeutics, a Foster City, California-based clinical-stage biotechnology company.

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.

The acquisition of GSK’s programs adds to Orchard’s pipeline of clinical and preclinical gene therapies for primary immune deficiencies and inherited metabolic disorders. The portfolio of gene-therapy programs Orchard has acquired includes: Strimvelis, an autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency, approved by the European Medicines Agency in 2016; two late-stage clinical programs in ongoing registrational studies for metachromatic leukodystrophy, a lysosomal storage disease, and Wiskott Aldrich syndrome, an immunodeficiency disease; and one clinical program for beta thalassaemia, a blood disorder. Orchard will also acquire rights to exclusively license three additional preclinical programs from Telethon/Ospedale San Raffaele, a joint research institute, upon completion of clinical proof-of-concept studies for mucopolysaccharidosis Type I, a lysosomal storage disease; chronic granulomatous disease, an immunodeficiency disease; and globoid cell leukodystrophy, an autosomal recessive disorder.

GSK will continue to invest in the development of its platform capabilities in cell and gene therapies with a focus on oncology.

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 programs in two current therapy areas, respiratory and HIV/infectious diseases, and two potential areas, oncology, and immuno-inflammation.

Source: GlaxoSmithKline