GSK hands rare disease gene therapies over to Orchard

The deal sees GSK become an investor in Orchard, with a 19.9 percent equity stake along with a seat on the firm’s board. The UK drugs giant also stands to receive royalties and commercial milestone payments related to the acquired portfolio.

The parties also noted that they intend to share manufacturing, technical and commercial insights and learnings on the development of gene therapy medicines to ensure the success of the assets.

The programmes acquired by Orchard includes Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved in Europe in 2016, and two late-stage clinical programmes in ongoing registrational studies for metachromatic leukodystrophy and Wiskott Aldrich syndrome, and one clinical programme for beta thalassaemia.

Orchard will also acquire rights to exclusively license three additional preclinical programmes from GSK partners Telethon/Ospedale San Raffaele upon completion of clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease and globoid cell leukodystrophy.

Explaining the rationale behind the move, Orchard said the programmes complement its own pipeline of clinical and preclinical gene therapies.

"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," noted Mark Rothera, the group’s chief executive.

For GSK, the agreement follows a strategic review of its rare disease unit, under wider prioritisation 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.

GSK said it would continue to invest in the development of its platform capabilities in cell and gene therapies, but with a focus on oncology.

"Orchard are committed to patient access, and we’re confident that this agreement combined with the ongoing relationship between the two companies will support the progression of these valuable programmes to enable them to benefit patients," said John Lepore, senior vice president, R&D pipeline, GSK.

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