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2004/OB/21
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OXFORD
BIOMEDICA PUBLISHES PRECLINICAL EFFICACY RESULTS WITH ITS
PRODUCT CANDIDATE SMN1-G FOR SPINAL MUSCULAR ATROPHY
Oxford
BioMedica (LSE: OXB), a leading gene therapy company, is pleased
to announce the publication of promising preclinical results
with its product candidate, SMN1-G, for the treatment of spinal
muscular atrophy (SMA), in the Journal of Clinical Investigation.
SMA is a fatal genetic disease that affects the spinal cord
and brain stem. To date there are no effective drug treatments
for SMA. The preclinical results published in the Journal
of Clinical Investigation (Volume:
114, No: 12, pp: 1726-1731), suggest that SMN1-G may have
potential in the treatment of this genetic disease.
The preclinical
studies were supported by FightSMA, a US charitable organisation.
In these studies, mice with a defective SMN gene were given
intramuscular injections with either SMN1-G or a control.
The mice treated with SMN1-G showed a statistically significant
improvement in survival together with improved motor neuron
survival.
SMA is
one of the most common genetic diseases leading to death in
childhood. The disease, characterised by degeneration of motor
neurons in the spinal cord and brain stem leading to muscle
weakness, is caused by mutations or deletion of the SMN1 gene.
SMN1-G is Oxford BioMedica’s gene-based therapeutic
which utilises the Company’s LentiVector gene delivery
system carrying a corrected SMN1 gene. The Company anticipates
progressing SMN1-G into clinical development during 2006.
Martha
Slay, President of FightSMA, said, "For the first time ever,
researchers have replaced a missing gene in a mouse model
of SMA, a disease that kills more infants than any other genetic
disorder."
Oxford
BioMedica’s CEO, Professor Alan Kingsman, said, "We
are very encouraged by our preclinical data with SMN1-G and
its potential to reverse the effects of this fatal disease.
We believe that there is a significant commercial opportunity
in the development of a safe and effective treatment for this
unmet medical need."
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| Notes
| 1. |
Oxford
BioMedica plc |
|
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;
two neurotherapy products in advanced preclinical
development for Parkinson’s disease
and retinopathy; and three further preclinical
neurotherapy products. The Company is underpinned
by an extensive preclinical and research
portfolio and over 80 patent families, which
represents one of the broadest patent estates
in the field.
The
Company has a staff of approx. 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, Viragen
and Kiadis. |
| 2. |
SMN1-G
for spinal muscular atrophy |
| |
Spinal muscular atrophy (SMA) refers to a
group of diseases, which affect the motor
neurons of the spinal cord and brain stem.
These critically important cells are responsible
for supplying electrical and chemical messages
to muscle cells. Without the proper input
from the motor neurons, muscle cells cannot
function properly. The muscle cells will,
therefore, become much smaller (atrophy) and
will produce symptoms of muscle weakness.
It is caused by mutations or deletion of the
SMN-1 gene, leading to depletion in SMN protein
levels. SMA
is the most common genetically determined
cause of infant death. It is estimated that
SMA occurs in between one-in-6,000 and one-in-20,000
births. Based on the clinical severity and
age at onset, the childhood SMAs have been
divided into three types. Type I SMA is
the most severe, with onset occurring by
6 months of age and death by 2 years. There
are currently no approved treatments for
this disorder. Oxford BioMedica estimates
the market opportunity for an effective
treatment of SMA to be approximately $50
million.
Oxford
BioMedica is developing a novel gene therapy
candidate, SMN1-G, for the treatment of
SMA. The product is designed to replace
the defective SMN-1 gene using the Company’s
proprietary LentiVector gene delivery system
carrying the SMN-1 gene. The development
of SMN1-G is being supported by the US charitable
organisation, FightSMA. The Company expects
to enter clinical development with SMN1-G
in 2006.
The
article from the Journal of Clinical Investigation
(Volume: 114, No: 12, pp: 1726-1731), describing
the preclinical efficacy data with SMN1-G
in a mouse model of the disease, is available
at http://www.oxfordbiomedica.co.uk/news2004.asp |
| 3. |
FightSMA |
| |
FightSMA
is a non-profit corporation, founded in Richmond,
VA, in 1991 as "Andrew's Buddies",
to accelerate research for a treatment or
a cure for spinal muscular atrophy, the number-one
inherited cause of death of children under
two.
Further information is available
at http://www.fightsma.com/
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| 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-Jane
Johnson
Buchanan Communications |
Tel: +44 (0)20 7466 5000
|
Scientific/Trade
Press Enquiries
Sue
Charles, Katja
Stout, Ashley
Lilly
College Hill - Life Sciences |
Tel:
+44 (0)20 7886 8150 |
FightSMA
Martha Slay, President |
Tel:
+1 804 515 0080 |
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