Date: 2018-04-12

Type of information: Pipeline acquisition

Compound: approved and investigational rare disease gene therapies including Strimvelis®

Company: GSK (UK) Orchard Therapeutics (UK) Molmed (Italy)

Therapeutic area: Rare diseases - Genetic diseases

Type agreement: pipeline acquisition

Action mechanism: gene therapy

Disease: adenosine deaminase severe combined immunodeficiency (ADA-SCID), metachromatic leukodystrophy (MLD), Wiskott Aldrich syndrome (WAS) , beta thalassaemia, mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD), globoid cell leukodystrophy (GLD)


  • • On April 12, 2018, GSK and Orchard Therapeutics announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for patients. This acquisition strengthens Orchard’s position as a global leader in gene therapy for rare diseases. GSK will continue to invest in the development of its platform capabilities in cell and gene therapies, with a focus on oncology.
  • GSK and Orchard will exchange manufacturing, technical and commercial insights and learnings on the development of gene therapy medicines to ensure the success of the assets.
  • The acquisition of GSK’s programmes complements Orchard’s pipeline of clinical and preclinical gene therapies for primary immune deficiencies and inherited metabolic disorders.
  • The portfolio of gene therapy programmes Orchard has acquired 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 programmes in ongoing registrational studies for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical programme for beta thalassaemia.
  • 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). Orchard Therapeutics will 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. In order to support a smooth transition of these programmes with minimal disruption to the projects, both companies have agreed to a transition period during which GSK will continue to conduct certain activities through to the end of 2018.
  • The agreement follows GSK’s strategic review of its rare disease unit, announced in July 2017, as part of the Group’s ongoing prioritisation and strengthening of its pharmaceuticals pipeline with a focus on priority programmes in two current therapy areas, respiratory and HIV/infectious diseases, and two potential areas, oncology and immuno-inflammation.
  • Following this strategic agreement, MolMed and Orchard Therapeutics announced the beginning of their collaboration in the field of gene therapy for rare diseases.

Financial terms:

  • Under the agreement, GSK will become an investor in Orchard Therapeutics, receiving a 19.9% equity stake along with a seat on the company’s board. GSK will also receive financial considerations in the form of royalties and commercial milestone payments related to the acquired portfolio.

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