Oxford BioMedica and Orchard ink gene therapy deal
- In a hook-up between two U.K. biotechs, gene and cell therapy company Oxford BioMedica and 2015 startup Orchard Therapeutics will work on ex-vivo gene therapy for primary immune deficiency disorders and inherited metabolic disorders. These will include adenosine deaminase severe combined immunodeficiency, mucopolysaccharidosis-IIIA and others.
- Oxford BioMedica will develop and manufacture lentiviral vectors for Orchard, and Orchard will lead development and commercialization in Europe, the U.S. and elsewhere.
- Oxford BioMedica gets a 1.95% equity stake in Orchard, and will get royalties and performance-related incentives. No other financial details have been revealed.
While the market for individual therapies in rare diseases are small, it is overall a huge field, and one that pharma and biotech companies are finding increasingly attractive. According to EvaluatePharma, the global sales for orphan drugs is expected to grow at 11.7% per year between 2015 and 2020 from a baseline of $178 billion. This is almost double the rate of the prescription drug market overall, and is driven by opportunities of unmet needs, along with fast-track reviews, lower R&D costs, tax breaks and research grants, as well as longer market exclusivity.
Orchard Therapeutics' lead program is an ex vivo gene lentiviral therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID), an inherited immune disorder. This is in clinical development, with more than 30 patients treated in the U.S. and U.K. as of March 2016. The patients showed signs of reconstitution of their immune system. A first in human clinical trial is planned for an ex vivo mucopolysaccharidosis-IIIA (MPS-IIIA; Sanfilippo syndrome type A) gene therapy once preclinical studies have been completed. This disorder affects around 1-2 in 100,000 births.
"This alliance with Oxford BioMedica represents a key element of the supply chain for manufacturing of our ex vivo gene-modified stem cell products. We expect that Oxford BioMedica’s expertise in the development and manufacturing of lentiviral vectors, along with their proven experience of working with global pharma companies will accelerate our ability to potentially address a series of devastating genetic diseases," said Stewart Craig, chief manufacturing officer of Orchard Therapeutics.
- Oxford BioMedica Statement
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