Research Update

Oxford Biomedica PLC 10 June 2002 10 June 2002 OXFORD BIOMEDICA AND IMPERIAL COLLEGE ANNOUNCE BREAKTHROUGH IN GENE THERAPY TECHNOLOGY Gene delivery in utero widens scope to new disease areas Oxford and London, United Kingdom - 10 June 2002. Oxford BioMedica plc (LSE:OXB) ('BioMedica') and researchers at Imperial College of Science and Technology, London ('Imperial College') announced today that they had achieved very efficient gene transfer to mouse embryos in utero. The technology, if reproducible in man, creates the potential to cure diseases such as Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF), diseases that are incurable at present. These developments were presented at the American Society for Gene Therapy (ASGT) meeting in Boston on Saturday, June 8th by Dr. Mike Themis of the Gene Therapy Group, Division of Biomedical Sciences at Imperial College, London. Diseases that are caused by defects in single genes are obvious targets for gene therapy. In fact it was is in this area that gene therapy was originally conceived. Subsequently the applications of gene therapy have broadened to many other disease areas including cancer, cardiovascular disease and neurodegenerative diseases. Recently, the news of successful treatment in the UK, by gene therapy, of a genetic deficiency in the immune system of a young boy demonstrated the feasibility of the approach. However, part of the reason for this success was because the clinical benefit to the patient could be achieved by delivering the appropriate gene to a relatively small number of the patient's white blood cells. In many other applications of gene therapy, gene transfer to the target cells must be much more efficient. This includes, for example, neurodegenerative diseases and some single gene disorders such as DMD. However, in DMD the challenge goes beyond increasing the efficiency of gene transfer. One in three thousand males inherit a non-functional dystrophin gene and suffer from DMD. Life expectancy is about 20 years and the patients are confined to a wheelchair from about the age of 10. The disease causes severe muscle weakness and it affects every muscle in the body. In order to correct the disease in children or adults every muscle would need to receive a functional dystrophin gene. This is not feasible using many current gene therapy techniques because the tissue volume is too great. A solution to this problem is to deliver the gene when the tissue volume is small. That is, when the patient is a developing foetus in the womb. The Imperial College team, using BioMedica's highly efficient LentiVector(R), has now shown that this is possible in animal models. At the ASGT meeting Dr. Themis showed gene transfer to a wide range of tissues, including liver, brain and muscle, following administration of LentiVector(R) to the foetal blood supply. If this were to be recapitulated in humans it would provide a potential route to treating several genetic deficiencies. Commenting on the results Dr. Themis said: 'In utero, gene therapy opens many exciting opportunities to treat people with these severely debilitating and fatal diseases before suffering occurs. We hope that one day this may be offered to parents as an alternative to termination of pregnancy. In addition, the efficiency of the gene transfer that we see in animals enables us to study the function of genes in a way that has not been possible before. This may accelerate our understanding of a broad range of diseases and create new therapeutic strategies.' Professor Alan Kingsman, BioMedica's Chief Executive commented: 'We are very pleased to have provided the gene delivery technology for this elegant work at Imperial College. We are currently evaluating product opportunities from these studies as well consulting broadly on the regulatory and ethical issues raised by such groundbreaking work.' For further information, please contact: Oxford BioMedica plc Professor Alan Kingsman, Chief Executive Tel: +44 (0)1865 783 000 City/Financial Enquiries Mike Wort / James Chandler, Beattie Financial Tel: +44 (0)20 7398 3300 Scientific/Trade Press Enquiries Emma Murray / Emma Timewell, HCC•De Facto Group Tel: +44 (0)20 7496 3300 -Ends- Notes to Editors Oxford BioMedica plc Established in 1995, the Company specialises in the application of gene-based technology to the development of novel therapeutics. Its three principal activities are in the fields of gene therapy, immunotherapy and genomics, and its principal therapeutic areas are in cancer and neurodegenerative diseases. Oxford BioMedica plc was floated on the Alternative Investment Market of the London Stock Exchange in December 1996, and was promoted to the United Kingdom Listing Authority Official List in April 2001 following a successful £35.5 million fund-raising. Oxford BioMedica is headquartered in Oxford, UK and has operating centres in Oxford and San Diego, USA Currently Oxford BioMedica has corporate collaborations with Aventis, IDM, Nycomed Amersham, Valentis, Virbac and Wyeth. BioMedica has two products in Phase I/II clinical trials: MetXia(R) for late-stage breast cancer, and TroVax (R) for late-stage colorectal cancer. LentiVector(R) In gene therapy, the aim is to deliver a gene and its necessary regulatory elements (the gene construct) to the cell surface, using a vector to mediate the transfer across the cell membrane and, in some cases, into the nucleus. LentiVector(R) is a new and increasingly powerful vector system based on lentiviruses, which have similar features to retroviruses in the ease of manipulation, predictable integration and reliable gene expression and regulation. However, their main advantage over retroviruses is the ability to function in non-dividing cells or cells that are dividing slowly - a feature of many clinically important tissues including the central and peripheral nervous systems. Oxford BioMedica is a leader in the development and application of lentiviral vectors. Its proprietary LentiVector(R) technology is protected by international patents, including recently granted US patents. BioMedica is developing products to treat cancer, Parkinson's disease and retinopathy using vectors based on equine infective anaemia virus (EIAV). EIAV is one of the most simple lentiviruses and is not known to cause disease in humans. For use in gene therapy, the virus is engineered so that it delivers only therapeutic genes and not viral genes. LentiVector(R) also has important potential applications in product development and target validation of genomic targets. World Wide Web This release is also available on the World Wide Web at http:// www.oxfordbiomedica.co.uk This information is provided by RNS The company news service from the London Stock Exchange D RESQDLFBLQBFBBD
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