Oxford Biomedica awarded £0.5 million from the UK department of health gene therapy research programme

Oxford , UK : 29 March 2004 – Oxford BioMedica announced today that the Department of Health has awarded the Company £0.5 million to develop its proprietary LentiVector® technology for use in the treatment of single gene inherited disorders. The focus of the work is to be haemophilia A, a condition caused by a defective gene for Factor VIII, a key component of the mechanism for forming blood clots.

The LentiVector technology, developed primarily for the Company’s neurotherapy pipeline, can be used in many areas outside the field of neurobiology. It is ideally suited to the treatment of single gene disorders where therapy is based on functional correction of a defective gene in patients. In this situation, there is a need for long-term, stable and predictable gene activity and this can be achieved with the LentiVector technology.

Three years ago the Company initiated a programme to develop a product known as RequinateTM. Requinate comprises a LentiVector gene delivery system carrying a modified version of the human Factor VIII gene. During this exploratory programme the Company solved several problems that had been encountered by others attempting gene therapy for haemophilia. However, since haemophilia falls outside of the Company’s therapeutic focus of cancer and neurotherapy, the programme has received minimal internal resources. The new money from the Department of Health will enable the Company to progress the Requinate haemophilia programme without compromising progress of its cancer and neurotherapy products. The current market for Factor VIII treatment is in excess of $1.0 billion.

Commenting on the award, Oxford BioMedica’s Chief Executive, Prof. Alan Kingsman said “We are pleased to see the Department of Health implementing the government’s decision to place gene-based medicine in the mainstream of healthcare in the UK . We are also delighted to be the only Company selected to contribute to this initiative and to aid in the process of bringing products to the market that will directly benefit patients with genetic diseases. Oxford BioMedica is currently evaluating how it can expand the application of the LentiVector technology for other inherited disorders, in addition to haemophilia, while retaining its essential focus on bringing its current product pipeline in cancer and neurotherapy to the market as quickly as possible”.

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Notes to editors

1. Oxford BioMedica

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.

In addition to its technical expertise in gene delivery, Oxford BioMedica has in-house clinical, regulatory and manufacturing know-how. The development pipeline includes two novel anti-cancer products in clinical trials; and two neurotherapy products in advanced preclinical development for Parkinson’s disease and retinopathy. The Company is underpinned by an extensive preclinical and research portfolio and about 70 patent families, which represents one of the broadest patent estates in the field.

The Company has a staff of ~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, Merck & Co, Amersham and Kiadis.

Further information is available at http://www.oxfordbiomedica.co.uk

2. LentiVector® technology

Oxford BioMedica has three issued US patents 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. The EIAV system is Oxford BioMedica’s system of choice because of its superior safety profile.

Oxford BioMedica’s LentiVector gene delivery technology is arguably the most potent system currently available for treating diseases of the central nervous system, particularly chronic neurodegenerative disorders. Oxford BioMedica has shown that minimal lentiviral vectors are able to deliver genes to a wide range of dividing and non-dividing cells, including neurones in the brain.

The Company has established a neurotherapy pipeline of product candidates based on its LentiVector technology, which includes ProSavin® for Parkinson’s disease, RetinoStat® for retinopathy, MoNudin™ for motor neuron disease and Innurex® for nerve repair.

3. Requinate™ and Haemophilia A

Requinate comprises a LentiVector system encoding a modified human Factor VIII gene, which has been engineered to enhance protein production. Following successful preclinical studies Oxford BioMedica intends to progress clinical development in collaboration with the Oxford Haemophilia Centre at the Churchill Hospital under the guidance of Dr. Paul Giangrande and Dr. David Keeling.    

People with haemophilia A do not produce adequate amounts of Factor VIII, which is necessary to effectively clot blood. Without enough Factor VIII, patients can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating and damaging to joints. If untreated, patients with severe haemophilia A have a greatly reduced life expectancy.

According to the World Federation of Haemophilia, more than 400,000 people in the world may have haemophilia A and it affects 15-20 of every 100,000 males born worldwide. People with haemophilia A infuse themselves with clotting factors three times a month, on average, unless they are on a preventive regimen, in which case their physician may prescribe infusing a therapy as often as three or four times per week.

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Oxford BioMedica are the only commercial company to receive a share of the £3 million in funding which has been earmarked for research into single gene disorders, in particular haemophilia, announced in the Genetics White Paper in June 2003. Please find attached Oxford BioMedica's announcement regarding this funding.

If you would like a spokesperson to comment on the Department of Health's announcement or further information on Oxford BioMedica's announcement, please contact Katja Stout on 020 7886 8150 or Professor Alan Kingsman, CEO of Oxford BioMedica on 01865 783000.