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GlaxoSmithKline has offloaded its rare disease gene therapy portfolio to the biotechnology company Orchard Therapeutics, in exchange for a near-20 per cent stake in the two-year-old business.

A sale of the rare disease unit had been mooted since last July, when GSK said it would focus its pharmaceuticals research and development resources on “priority assets” such as treatments for respiratory conditions, HIV and infectious diseases.

The portfolio of gene therapy programmes acquired by Orchard includes a treatment for the ultra-rare “bubble boy” disease, which has won the backing of the UK’s National Institute for Health and Care Excellence, the agency tasked with determining whether new medicines are sufficiently cost-effective, despite its €549,000 price tag.

As part of the deal, Orchard committed to maintaining access to the one-off treatment, known as Strimvelis, which uses a patient’s own stem cells to treat the inherited genetic disorder that causes such extreme vulnerability to infection that sufferers are often forced to live in isolation.

GSK will get a 19.9 per cent stake in Orchard and a seat on its board, as well as royalties and milestone payments from the treatments. The FTSE 100 pharmaceutical company will carry on some of its work on the programmes for a transition period through to the end of 2018 to avoid disruption, it said.

Orchard, which raised $110m in a private funding round last year to help develop its treatment pipeline, said the deal strengthened its position “as a global leader in gene therapy for rare diseases”.

“The acquisition of GSK’s programmes complements Orchard’s pipeline of clinical and preclinical gene therapies for primary immune deficiencies and inherited metabolic disorders,” it added.

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.

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