Lentivector is the 'system of choice' for RNAI

The paper describes the delivery, by the LentiVector technology, of a specific RNAi molecule that shuts down the gene that causes the disease.

Familial ALS is caused by a series of mutations in a gene called Sod1.

The disease represents about 2% of the total ALS cases.

These mutations cause the Sod1 gene to acquire a new function that destroys motor neurons.

The neurons die and the patient loses muscle control in a similar way to the more common acquired ALS or Lou Gehrig's disease.

Patients generally die from respiratory failure because they are unable to control their diaphragm muscle.

Switching off the gene stops the development of the disease but the challenge so far has been to achieve that goal.

RNAi is a new technology that uses small RNA molecules to switch off any gene of choice.

It has been adopted widely by the pharmaceutical industry as a means of identifying genes with a causative relationship to specific diseases.

The Company's LentiVector technology is perfectly suited to deliver these molecules and this is what has been reported in the Nature Medicine paper.

A LentiVector has been used to deliver a highly specific RNAi molecule to a selection of motor neurons in a mouse that has the mutated Sod1 gene.

The data show that delivery was very efficient and there was a 100% extension of the time to onset of the disease as well as a 70% increase in survival.

Commenting on the results the company's CEO, Alan Kingsman said: "These data show that our LentiVector technology is the system of choice for the genetic delivery of RNAi molecules.

"This is a particularly powerful illustration of the point and is assisting us with our ongoing out-licensing of the LentiVector technology for drug discovery and transgenic applications".

"We have already completed four such deals and we have five further term sheets under discussion."