Oxford Biomedica publishes 'promising' preclinical efficacy results with its product candidate smn1-g for spinal muscular atrophy
Oxford BioMedica announces 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."
