OXFORD BIOMEDICA PUBLISHES PRECLINICAL EFFICACY RESULTS WITH ITS PRODUCT CANDIDATE SMN1-G FOR SPINAL MUSCULAR ATROPHY.
RNS Number:4672G
Oxford Biomedica PLC
16 December 2004
FOR IMMEDIATE RELEASE 16 DECEMBER 2004
OXFORD BIOMEDICA PUBLISHES PRECLINICAL EFFICACY RESULTS WITH ITS PRODUCT
CANDIDATE SMN1-G FOR SPINAL MUSCULAR ATROPHY
Oxford, UK - 16 December 2004: 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." |
