GlaxoSmithKline transfers rare disease gene therapy portfolio to Orchard Therapeutics in strategic deal

GlaxoSmithKline (GSK), a leading British pharmaceutical company, has entered into a significant agreement with Orchard Therapeutics, transferring its rare disease gene therapy drugs in exchange for a 19.9% equity stake and a seat on Orchard’s board. This move is part of GSK’s strategic initiative to refine its focus on core areas such as respiratory, HIV/infectious diseases, oncology, and immuno-inflammation, which began with a strategic review in July 2017.

Strategic Shift to Enhance Pharma Pipeline

The deal involves the transfer of several key gene therapies, including Strimvelis—a European Medicines Agency (EMA) approved treatment for ADA-SCID, also known as bubble baby syndrome—and other investigational therapies for conditions like metachromatic leukodystrophy and Wiskott Aldrich syndrome, both in late-stage development. This strategic decision allows GSK to concentrate on expanding its broader cell and gene therapy platform while ensuring the continued development of its rare disease treatments.

Financial and Operational Benefits

As part of the agreement, GSK will also benefit financially from royalties and commercial milestone payments related to these gene therapy drugs. This arrangement follows closely after GSK’s $13 billion proposal to fully acquire Novartis’ stake in their consumer healthcare joint venture, marking a significant period of strategic realignments for the company.

Orchard Therapeutics’ Enhanced Global Presence

For Orchard Therapeutics, this deal significantly bolsters its global presence in gene therapies for rare diseases. “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 of Orchard Therapeutics. He emphasized that the acquisition not only expands Orchard’s primary immune deficiency and inherited metabolic disorder franchises but also adds potential for future expansion into other areas.

Commitment to Patient Access and Advancement

John Lepore, Senior Vice President of GSK R&D pipeline, expressed pride in the developments achieved through their collaborations in Milan and highlighted the goal of finding the right steward for these therapies. “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,” Lepore stated.

Looking Forward

The acquisition deal not only includes current therapies but also provides Orchard Therapeutics with exclusive rights to license three additional preclinical programs post-clinical proof of concept trials for diseases like Hurler syndrome, chronic granulomatous disease, and globoid cell leukodystrophy. This strategic acquisition positions Orchard Therapeutics to further solidify its role as a leader in gene therapy for rare diseases while allowing GSK to focus on its strategic priorities.