European Orphan Drug Designat

RNS Number : 8079F

Oxford Biomedica PLC

20 January 2010

 



For Immediate Release

20 JANUARY 2010

OXFORD BIOMEDICA'S USHSTAT™ FOR USHER SYNDROME RECEIVES 
EUROPEAN ORPHAN DRUG DESIGNATION

Oxford, UK - 20 January 2010: Oxford BioMedica (LSE: OXB), a leading gene therapy company,  announced today that UshStat, the Company's gene therapy for the treatment of Usher syndrome 1B, has received orphan designation from the Committee for Orphan Medicinal Products of the European Medicines Agency. 

Usher syndrome 1B is an inherited condition that results in hearing loss and progressive loss of vision from retinitis pigmentosa. It is caused by abnormalities in a gene called Myosin VIIA (MYO7A). UshStat is designed to deliver a corrected version of the MYO7A gene into the cells of the retina using the Company's LentiVector® gene delivery technology. In collaboration with sanofi-aventis, clinical development is expected to start in 2011. 

The European Medicines Agency grants orphan drug designation to products that may provide a significant advantage over current treatments, if any exist, for life-threatening or chronically debilitating conditions affecting up to five in 10,000 people in the European Union. Companies with European orphan  drug  designation benefit from incentives, including ten years of marketing exclusivity and reduced regulatory fees.

Oxford BioMedica's Chief Executive Officer, John Dawson, commented: "We are delighted to have received orphan designation from the European Medicines Agency for another of our innovative LentiVector-based candidates targeting debilitating and progressive ocular diseases. This designation is an important step towards the start of clinical trials of UshStat for Usher syndrome 1B as part of our landmark  collaboration with sanofi-aventis to develop gene therapies in the field of ophthalmology." 

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For further information, please contact:
Oxford BioMedica plc:  John Dawson, Chief Executive Officer Nick Woolf, Chief Business Officer	Tel: [phone]
JPMorgan Cazenove Limited: James Mitford/ Gina Gibson	Tel: [phone]
Media/Financial Enquiries: Lisa Baderoon/ Mark Court  Buchanan Communications	Tel: [phone]
US Enquiries: Thomas Fechtner The Trout Group LLC	Tel: [phone]

Notes to editors

1. Oxford BioMedica

Oxford BioMedica (LSE: OXB) is a biopharmaceutical company developing innovative gene-based medicines and therapeutic vaccines that aim to improve the lives of patients with high unmet medical needs. The Company's technology platform includes a highly efficient gene delivery system (LentiVector®), which has specific advantages for targeting diseases of the central nervous system and the eye; and a unique tumour antigen (5T4), which is an ideal target for anti-cancer therapy. Through in-house and collaborative research, Oxford BioMedica has a broad pipeline and its partners include sanofi-aventis, Sigma-Aldrich and Pfizer. Technology licensees include Biogen Idec, GlaxoSmithKline, Merck & Co and Pfizer. Further information is available at www.oxfordbiomedica.co.uk

2. UshStat™ for Usher Syndrome 1B

Usher syndrome affects four in every 100,000 live births and is a leading cause of deaf-blindness. One of the most common subtypes is Usher syndrome 1B, which is associated with a mutation of the gene encoding Myosin VIIA (MYO7A). This leads to progressive retinitis pigmentosa combined with a congenital hearing defect. Approximately 8,000 people in the USA and the EU suffer from Usher syndrome 1B and, currently, there is no effective treatment available. UshStat uses Oxford BioMedica's LentiVector® technology to deliver a corrected version of the MYO7A gene to retinal cells. Promising results from preclinical studies suggest that a single administration of UshStat could provide long-term or potentially permanent stabilisation of ocular function in patients with Usher syndrome 1B.

 

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