Orchard Therapeutics Acquires GSK’s Gene Therapy Portfolio

Rare Daily Staff

GSK will said it will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard Therapeutics in exchange for a 19.9 percent stake in the biotech.

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

For GSK, the agreement with Orchard allows is in line with efforts to narrow its focus and improve profitability in its core businesses. The move follows a strategic review of GSK’s rare disease unit, announced in July 2017, as part of its decision to prioritize existing programs in respiratory and HIV/infectious diseases therapies, and two potential areas, oncology and immuno-inflammation.

For Orchard, the agreement deal complements its existing pipeline of gene therapies for primary immune deficiencies and inherited metabolic disorders.

“Acquiring this portfolio further advances Orchard’s vision to be a global, fully integrated company leading the field of gene therapy for rare diseases,” said Mark Rothera, CEO, Orchard. “The acquisition immediately expands our primary immune deficiency and inherited metabolic disorder franchises and adds the potential for other franchises in the future.”

In addition to its equity stake, GSK will also receive financial considerations in the form of royalties and commercial milestone payments related to the acquired portfolio. GSK and Orchard will exchange manufacturing, technical, commercial and other information relating to the development of gene therapy medicines to ensure the success of the assets.

Orchard Therapeutics also agreed to assume all obligations arising from GSK’s 2010 collaboration agreement with the Ospedale San Raffaele and Fondazione Telethon and from GSK’s collaboration agreement with MolMed.

To ensure a smooth transition of the programs, the companies said they have agreed to a transition period during which GSK will continue to conduct certain activities through to the end of 2018.

The portfolio of gene therapy programs involved in the transaction includes: Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved by the EMA in 2016, two late-stage clinical programs in ongoing registrational studies for metachromatic leukodystrophy  and Wiskott Aldrich syndrome, and one clinical program for beta thalassemia.

Orchard will also acquire rights to exclusively license three additional preclinical programs from 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.

“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,” said John Lepore, senior vice president, R&D pipeline, for GSK. “Orchard [is] 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 programs to enable them to benefit patients.”

April 12, 2018
Photo: Mark Rothera, CEO, Orchard Therapeutics