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2005/OB/09
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OXFORD BIOMEDICA DEMONSTRATES THAT ITS LENTIVECTOR® TECHNOLOGY IS THE SYSTEM OF CHOICE FOR RNAI APPLICATIONS
Oxford BioMedica (LSE:OXB.L), the leading gene therapy company, announced today the publication, in the journal Nature Medicine, of a paper demonstrating efficacy of the Company’s LentiVector technology in an animal model of inherited (familial) amyotrophic lateral sclerosis (ALS) or motor neuron disease. The paper describes the delivery, by the LentiVector technology, of a specific RNAi molecule that shuts down the gene that causes the disease.
Familial ALS is caused by a series of mutations in a gene called SOD1. The disease represents about 2% of the total ALS cases. These mutations cause the SOD1 gene to acquire a new function that destroys motor neurons. The neurons die and the patient loses muscle control in a similar way to the more common acquired ALS or Lou Gehrig’s disease. Patients generally die from respiratory failure because they are unable to control their diaphragm muscle. Switching off the gene stops the development of the disease but the challenge so far has been to achieve that goal.
RNAi is a new technology that uses small RNA molecules to switch off any gene of choice. It has been adopted widely by the pharmaceutical industry as a means of identifying genes with a causative relationship to specific diseases. The Company’s LentiVector technology is perfectly suited to deliver these molecules and this is what has been reported in the Nature Medicine paper. A LentiVector has been used to deliver a highly specific RNAi molecule to a selection of motor neurons in a mouse that has the mutated SOD1 gene. The data show that delivery was very efficient and there was a 100% extension of the time to onset of the disease as well as a 70% increase in survival.
Commenting on the results the Company’s CEO, Prof. Alan Kingsman said: “These data show that our LentiVector technology is the system of choice for the genetic delivery of RNAi molecules. This is a particularly powerful illustration of the point and is assisting us with our ongoing out-licensing of the LentiVector technology for drug discovery and transgenic applications. We have already completed four such deals and we have five further term sheets under discussion.”
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Notes
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Oxford
BioMedica |
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Oxford BioMedica (LSE: OXB) is a biopharmaceutical company specialising in the development of novel gene-based therapeutics with a focus on the areas of oncology and neurotherapy. The Company was established in 1995 as a spin out from Oxford University, and is listed on the London Stock Exchange.
Oxford BioMedica has core expertise in gene delivery, as well as in-house clinical, regulatory and manufacturing know-how. In oncology, the pipeline includes an immunotherapy and a gene therapy in multiple Phase II trials, and a preclinical targeted antibody therapy in collaboration with Wyeth. In neurotherapy, the Company’s lead product is a gene therapy for Parkinson’s disease, which is expected to enter clinical development in 2005, and four further preclinical candidates. The Company is underpinned by over 80 patent families, which represent one of the broadest patent estates in the field.
The Company has a staff of approximately 65 split between its main facilities in Oxford and its wholly owned subsidiary, BioMedica Inc, in San Diego, California. Oxford BioMedica has corporate collaborations with Wyeth, Intervet, Amersham, Viragen, MolMed and Kiadis; and has licensed technology to a number of companies including Merck & Co and Biogen Idec.
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LentiVector technology |
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Oxford BioMedica’s LentiVector gene delivery technology, based on lentiviral vectors, is arguably the most potent system currently available for treating a range of diseases, particularly those of the central nervous system. Oxford BioMedica has shown that its lentiviral vectors are able to deliver genes with high efficiency to a variety of both dividing and non-dividing cells, including neurons in the brain.
Oxford BioMedica has three issued US patents and a European patent for its LentiVector technology. These include broad composition of matter claims and methods of production claims for lentiviral vector gene delivery systems of both human and non-human origin. The patents also cover derivatives of lentiviral vector systems that, unlike many versions of lentiviral vectors, have real clinical utility because of their safety. The Oxford BioMedica team was the first to construct lentiviral vectors that contain no viral genes at all, and which comprise the minimum number of viral components in the viral particles. It is this minimisation of the vectors that is the subject of these patents. This work was done using vectors based on HIV and Equine Infectious Anaemia Virus (EIAV), a horse virus that is not linked to any disease in humans. |
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further information please contact:
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Oxford BioMedica plc
Professor Alan
Kingsman, Chief Executive |
Tel: +44 (0)1865 783 000 |
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Enquiries
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Baderoon/Mark
Court/Mary-Jane
Johnson
Buchanan Communications |
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Charles, Katja
Stout, Ashley
Lilly
College Hill - Life Sciences |
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