Oxford BioMedica announced in December 2002 that it has been awarded a grant of $0.5 million to support its Motor Neuron Disease (MND) programme
The grant comes from the US charity Andrew's Buddies/FightSMA, which is dedicated to the development of treatments for Spinal Muscular Atrophy (SMA), a form of motor neuron disease that is one of the most common causes of death in childhood.
SMA is caused by a defect in the SMN-1 gene.
It affects one in every 6000 births and one in 40 people are carriers.
The defect leads to a degeneration of motor neurons in the spinal cord with concomitant systemic loss of muscle function.
In the most severe form of the disease, weakness of the respiratory muscles results in death usually before the age of two years.
The product strategy is to replace the defective gene using the company's proprietary LentiVector carrying the SMN-1 gene.
Oxford BioMedica retains all commercial rights arising from the grant-funded programme and the company anticipates having a clinical stage product within two years.
The SMA product is part of Oxford BioMedica's motor neuron disease programme that also includes the development candidate MoNuDina for the most common form of MND, amyotrophic lateral sclerosis (ALS).
ALS has an estimated market of $200 million.
Both products use Oxford BioMedica's proprietary LentiVector gene delivery technology and are at the preclinical stage of development.
At present there are no products that make a major impact on these devastating diseases.
"We are delighted to fund this ground-breaking initiative.
"The work of Oxford BioMedica breathes new hope into the lives of countless parents and their children who suffer with SMA." said Martha Slay, president of Andrew's Buddies/FightSMA.
Commenting on the grant from Andrew's Buddies/FightSMA, Oxford BioMedica's chief executive, professor Alan Kingsman said "The development of novel products for neurological disease is the largest growth area in pharmaceutical research.
"Oxford BioMedica has an exceptional capability in this area and is developing a pipeline of products of which the lead is ProSavin for Parkinson's disease.
"We are delighted that Andrew's Buddies/FightSMA have recognised the quality of our technology and we look forward to working together towards an effective treatment for this inherited disease that brings so much sorrow to afflicted families."
