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Orchard Therapeutics to buy GSK’s rare disease gene therapy portfolio

EBR Staff Writer Published 13 April 2018

Privately held clinical-stage biotechnology firm Orchard Therapeutics has agreed to acquire the rare disease gene therapy portfolio of GlaxoSmithKline (GSK).

Under the agreement, GSK will receive a 19.9% stake in Orchard and get a seat on its board. 

The acquisition of the portfolio, which includes both approved and investigational therapies like Strimvelis and a therapy for beta thalassaemia, is expected to consolidate Orchard’s position in gene therapy for rare diseases.

Orchard also expects the GSK rare disease candidates to complement its own pipeline of clinical and preclinical gene therapies developed for primary immune deficiencies and inherited metabolic disorders.

Orchard 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.

Strimvelis is an autologous ex vivo gene therapy, approved in the European Union in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID) in children.

The GSK rare disease portfolio also has two gene therapy candidates in late-stage development, which have been developed for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS).

Through the deal, Orchard will also gain rights to exclusively license three more preclinical programs from Telethon/Ospedale San Raffaele post completion of clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD) and globoid cell leukodystrophy (GLD).

For GSK, the sale of the rare disease unit follows its strategic review announced in last July. The company has been looking to prioritize and boost its pharmaceuticals pipeline, while focusing on developing therapies for respiratory and HIV/infectious diseases, and also oncology and immuno-inflammation.

GSK R&D pipeline senior vice president John Lepore said: “GSK is proud of the advances we have achieved in collaboration with the cell and gene therapy pioneers at Ospedale San Raffaele, Fondazione Telethon and MolMed in Milan. 

“Since we announced our intent to review these medicines, our goal has been to identify the right owner who can build on what we’ve already achieved, and can advance these important medicines for patients, allowing GSK to focus on building its broader cell and gene therapy platform capabilities.”

GSK will also be entitled to get financial considerations through royalties and commercial milestone payments associated with the acquired portfolio.

The sale of its rare disease gene therapy unit for GSK follows its deal in late March to acquire Novartis’ stake in their consumer healthcare joint venture for $13bn.


Image: GSK House in Brentford, London. Photo: courtesy of GlaxoSmithKline plc.