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LentiVector Technology

14th Mar 2005 12:47

Oxford Biomedica PLC14 March 2005 FOR IMMEDIATE RELEASE 14 MARCH 2005 OXFORD BIOMEDICA DEMONSTRATES THAT ITS LENTIVECTOR(R) TECHNOLOGY IS THE SYSTEM OF CHOICE FOR RNAI APPLICATIONS Oxford, UK - 14 March 2005: Oxford BioMedica (LSE:OXB.L), the leading genetherapy company, announced today the publication, in the journal NatureMedicine, of a paper demonstrating efficacy of the Company's LentiVectortechnology in an animal model of inherited (familial) amyotrophic lateralsclerosis (ALS) or motor neuron disease. The paper describes the delivery, bythe LentiVector technology, of a specific RNAi molecule that shuts down the genethat causes the disease. Familial ALS is caused by a series of mutations in a gene called SOD1. Thedisease represents about 2% of the total ALS cases. These mutations cause theSOD1 gene to acquire a new function that destroys motor neurons. The neurons dieand the patient loses muscle control in a similar way to the more commonacquired ALS or Lou Gehrig's disease. Patients generally die from respiratoryfailure because they are unable to control their diaphragm muscle. Switching offthe gene stops the development of the disease but the challenge so far has beento achieve that goal. RNAi is a new technology that uses small RNA molecules to switch off any gene ofchoice. It has been adopted widely by the pharmaceutical industry as a means ofidentifying genes with a causative relationship to specific diseases. TheCompany's LentiVector technology is perfectly suited to deliver these moleculesand this is what has been reported in the Nature Medicine paper. A LentiVectorhas been used to deliver a highly specific RNAi molecule to a selection of motorneurons in a mouse that has the mutated SOD1 gene. The data show that deliverywas very efficient and there was a 100% extension of the time to onset of thedisease as well as a 70% increase in survival. Commenting on the results the Company's CEO, Prof. Alan Kingsman, said: "Thesedata show that our LentiVector technology is the system of choice for thegenetic delivery of RNAi molecules. This is a particularly powerful illustrationof the point and is assisting us with our ongoing out-licensing of theLentiVector technology for drug discovery and transgenic applications. We havealready completed four such deals and we have five further term sheets underdiscussion." -Ends- For further information, please contact:Oxford BioMedica plc:Professor Alan Kingsman, Chief Executive Tel: +44 (0)1865 783 000 City/Financial Enquiries:Lisa Baderoon/ Mark Court/ Mary-JaneJohnson Buchanan Communications Tel: +44 (0)20 7466 5000 Scientific/Trade Press Enquiries:Sue Charles/ Katja Stout/ Ashley LillyNorthbank Communications Tel: +44 (0)20 7886 8150 Notes to editors 1. Oxford BioMedica Oxford BioMedica (LSE: OXB) is a biopharmaceutical company specialising in thedevelopment of novel gene-based therapeutics with a focus on the areas ofoncology and neurotherapy. The Company was established in 1995 as a spin outfrom Oxford University, and is listed on the London Stock Exchange. Oxford BioMedica has core expertise in gene delivery, as well as in-houseclinical, regulatory and manufacturing know-how. In oncology, the pipelineincludes an immunotherapy and a gene therapy in multiple Phase II trials, and apreclinical targeted antibody therapy in collaboration with Wyeth. Inneurotherapy, the Company's lead product is a gene therapy for Parkinson'sdisease, which is expected to enter clinical development in 2005, and fourfurther preclinical candidates. The Company is underpinned by over 80 patentfamilies, which represent one of the broadest patent estates in the field. The Company has a staff of approximately 65 split between its main facilities inOxford 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 companiesincluding Merck & Co and Biogen Idec. Further information is available at www.oxfordbiomedica.co.uk. 2. LentiVector technology Oxford BioMedica's LentiVector gene delivery technology, based on lentiviralvectors, is arguably the most potent system currently available for treating arange of diseases, particularly those of the central nervous system. OxfordBioMedica has shown that its lentiviral vectors are able to deliver genes withhigh efficiency to a variety of both dividing and non-dividing cells, includingneurons in the brain. Oxford BioMedica has three issued US patents and a European patent for itsLentiVector technology. These include broad composition of matter claims andmethods of production claims for lentiviral vector gene delivery systems of bothhuman and non-human origin. The patents also cover derivatives of lentiviralvector systems that, unlike many versions of lentiviral vectors, have realclinical utility because of their safety. The Oxford BioMedica team was thefirst to construct lentiviral vectors that contain no viral genes at all, andwhich comprise the minimum number of viral components in the viral particles. Itis this minimisation of the vectors that is the subject of these patents. Thiswork 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. This information is provided by RNS The company news service from the London Stock Exchange

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