New British Partnership set to Cure Rare Diseases with Gene Therapy

Oxford BioMedica and Orchard Therapeutics are partnering up to bring gene therapy to the market and treat rare diseases for which no cure is available yet.

Oxford BioMedica develops lentiviral vectors for gene and cell therapy applications. The company just announced a partnership with another biotech from the UK’s golden triangleOrchard Therapeutics. The deal is that Oxford BioMedica will develop and supply GMP-grade lentiviral vectors with exclusive IP licenses that Orchard will use in its ex-vivo stem cell gene therapies for rare diseases.

In exchange, Oxford Biomedica will receive a 1.95% equity stake in Orchard and performance incentives that could double this number. Additionally, the Oxford-based company will receive royalties on future sales of products arising from the collaboration.


The main targets of Orchard’s ex-vivo gene therapies are adenosine deaminase severe combined immunodeficiency (ADA-SCID) and Sanfilippo Syndrome type A. The new team will then be competing with Lysogene, one of the coolest biotechs in Paris, which was started by Karen Aiach, CEO, to find a cure for her daughter with Sanfilippo A.

oxford biomedica

Oxford BioMedica’s headquarters in… guess where? Oxford!

Rare diseases are particularly attractive for gene and cell therapy biotechs since most of them lack any treatments available, and although the market is smaller, it isn’t crowded with big pharma. However, this might result in several gene therapy companies targeting the same conditions, given that commercial failure is a serious concern. For example, UniQure’s Glybera was bought only once, leading the company to restructure its whole R&D strategy and shift to Hemophilia B.

With this new collaboration, Orchard strengthens its position in the gene therapy field, where commercialization is also a big challenge. Oxford BioMedica seems like the perfect partner to ensure success: its versatile LentiVector platform is being used in clinical trials for Parkinson’s, several ocular diseases and oncology, including a CAR-T program in partnership with Novartis.  Will the British team be able to make it to the short list of gene therapies on the market?

Images via Blablo101/; Oxford BioMedica

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