GlaxoSmithKline (GSK) has signed a strategic agreement to transfer its rare disease gene therapy portfolio to Orchard Therapeutics, following a review of the rare disease unit in July 2017.

GSK will take a 19.9% equity stake and board membership at Orchard, and will be eligible for royalties and milestone payments based on drug sales.

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The deal, which includes both approved and investigational products, will bolster Orchard’s clinical and preclinical pipeline for immune deficiencies and inherited metabolic disorders.

Orchard Therapeutics CEO Mark Rothera 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.”

“The acquisition immediately expands our primary immune deficiency and inherited metabolic disorder franchises and adds the potential for other franchises in the future.”

GSK plans to continue developing its platform capabilities in cell and gene therapies, and will primarily focus on oncology.

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The company will trade manufacturing, technical and commercial insights with Orchard, along with information on developing gene therapy drugs.

GSK R&D pipeline senior vice-president John Lepore said: “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.”

The divested portfolio includes gene therapy Strimvelis for adenosine deaminase severe combined immunodeficiency (ADA-SCID) in children and one beta thalassaemia programme.

In addition, Orchard gets two late-stage programmes for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS).

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).

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