Oxford BioMedicas UshStat For Usher Syndrome Receives European Orphan Drug Designation - 1/20/2010
2010/OB/02
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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Notes to editors
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Oxford BioMedica plc
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. Partners include sanofi-aventis, Sigma-Aldrich and Wyeth. Technology licensees include Biogen-Idec, GlaxoSmithKline, Merck & Co and Pfizer. Further information is available at www.oxfordbiomedica.co.uk
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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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Oxford BioMedica plc
Lara Mott, Head of Corporate Communications |
Tel: +44 (0)1865 783 000 |
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JPMorgan Cazenove Limited
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Emma Thompson/Rob Newman/Ben Simons |
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US Enquiries
Thomas Fechtner
The Trout Group LLC |
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