Strategic Alliance with Orchard Therapeutics

RNS Number : 3629Q
Oxford Biomedica PLC
29 November 2016









Oxford BioMedica Announces a Strategic Alliance with Orchard Therapeutics


Oxford, UK & London, UK- 29 November 2016: Oxford BioMedica plc ("Oxford BioMedica" or "the Group") (LSE: OXB), a leading gene and cell therapy group, today announces it has entered into a strategic alliance with Orchard Therapeutics ("Orchard"), a biotechnology company dedicated to bringing transformative ex-vivo stem cell based gene therapies to patients with serious and life-threatening orphan diseases.


As part of the agreement the Group will develop and supply lentiviral vectors used by Orchard for the manufacture of ex-vivo gene therapy products in primary immune deficiency disorders and inherited metabolic disorders, including adenosine deaminase severe combined immunodeficiency (ADA-SCID), Mucopolysaccharidosis-IIIA (MPS-IIIA or Sanfilippo Syndrome type A) and undisclosed follow-on indications. Orchard will lead the global clinical development and commercialisation of collaboration programmes in Europe, the United States and in other regions.


Under the terms of the collaboration and licence agreement, Oxford BioMedica will receive a 1.95% equity stake in Orchard and will be entitled to royalties on future sales of products covered by the collaboration. The Group will provide process development services and manufacture clinical and commercial GMP-grade lentiviral vectors for Orchard. The process development arrangements include performance-related incentives through which Oxford BioMedica could receive a further 1.95% equity stake in Orchard. The Group has also granted an exclusive intellectual property licence to Orchard for collaboration programmes.



John Dawson, Chief Executive Officer of Oxford BioMedica, commented: "We are delighted to initiate a Strategic Alliance with Orchard Therapeutics and look forward to working with them to develop and launch much needed treatments for patients in desperate need of better treatment options. The alliance combines Oxford BioMedica's world-leading capabilities in lentiviral vector process development and bioprocessing expertise with Orchard's expertise in the development and commercialisation of gene therapies for orphan diseases. This further demonstrates Oxford BioMedica's position as a "go to" partner for companies and academic institutions working with lentiviral vector based products."


Stewart Craig, Chief Manufacturing Officer of Orchard Therapeutics Ltd., commented: "Orchard is a leader in bringing transformative gene therapies to patients with serious and life threatening orphan diseases. This alliance with Oxford BioMedica represents a key element of the supply chain for manufacture of our ex vivo gene-modified stem cell products. We expect that Oxford BioMedica's expertise in the development and manufacture 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. We are excited about the potential for this alliance to deliver real patient benefits."





For further information, please contact:



Oxford BioMedica plc:

John Dawson, Chief Executive Officer

Tim Watts, Chief Financial Officer


Tel: +44 (0)1865 783 000


Financial and corporate communications enquiries:

Consilium Strategic Communications

Mary-Jane Elliott/Matthew Neal/Chris Welsh/Laura Thornton


Tel: +44 (0)20 3709 5700


Orchard Therapeutics Limited:

Sylvie Blanchier                                                                   Tel: +44 (0)20 3823 2149




Notes to editors


About Oxford BioMedica®

Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy company focused on developing life changing treatments for serious diseases. Oxford BioMedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector®) through which the Group develops in vivo and ex-vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The Group has also entered into a number of partnerships, including with Novartis, Sanofi, GSK, and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 250 people. Further information is available at


About Orchard Therapeutics Ltd.

Orchard Therapeutics is a clinical-stage biotechnology company dedicated to bringing transformative ex-vivo gene therapies to patients with serious and life-threatening orphan diseases. The company was recently named a Fierce 15 Company for 2016 by Fierce Biotech. For more information, visit


About Adenosine Deaminase Deficiency Severe Combined Immunodeficiency ("ADA-SCID")

ADA-SCID is a rare inherited disorder of the immune system. The incidence of ADA-SCID is estimated between 1 in every 375,000 - 660,000 live births, according to literature sources. ADA-SCID is caused by mutations in the gene encoding for the enzyme adenosine deaminase, which result in a severe deficiency in white blood cells and life-threatening infections. In the absence of treatment, ADA-SCID is fatal within the first months of life. Despite currently available treatment options, there remains significant need for therapies that reduce the mortality, morbidity and burden of disease on patients and families.


About Mucopolysaccharidosis type-A (Sanfilippo Syndrome type A)

Mucopolysaccharidosis type-A is a rare neurodegenerative lysosomal storage disease caused by mutations in the sulfoglycosamine sulfohydrolase (SGSH) gene. There are no effective treatments for MPS-IIIA to date. The disease affects children in early life, with a progressive decline in cognitive and behavioural function and a subsequent decline in motor function and results in severe dementia and early death, usually in the teens or early twenties.


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