Orchard Takes GSK’s Rare Disease Gene Therapies

UK biotech Orchard Therapeutics has acquired GlaxoSmithKline’s (GSK) portfolio of approved and investigational rare disease gene therapies. The deal will see GSK take a 19.9% equity stake in Orchard Therapeutics along with a seat on the biotech’s board.  The UK drugs giant will also receive royalties and commercial milestone payments related to the acquired portfolio.

The programs acquired by Orchard include Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved by the European Medicines Agency in 2016 as well as two late-stage clinical programs in ongoing registration studies for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS); and one clinical program for beta thalassemia.

Orchard will also acquire rights to exclusively license three additional preclinical programs from Italian research institutes Ospedale San Raffaele and Fondazione Telethon 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).

In addition, Orchard will assume all obligations arising from GSK’s 2010 collaboration agreement with Ospedale San Raffaele and Fondazione Telethon and from GSK’s collaboration agreement with Italian biotech MolMed. 

“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,” said Orchard’s CEO, Mark Rothera.

GSK announced last July that it was undertaking a strategic review of its rare disease unit.  The drugmaker is prioritizing and strengthening its pharmaceuticals pipeline with a focus on priority programs in two current therapy areas: respiratory and HIV/infectious diseases; along with two potential areas – oncology and immuno-inflammation. GSK said it will continue to invest in developing its platform capabilities in cell and gene therapies, but with a focus on oncology.