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.
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