Parkinson's disease (ReN004)

Parkinson's disease is a progressive neurodegenerative disease of the basal ganglia region of the brain, with tremor, rigidity and difficulty initiating movement being the most common symptoms.

The condition is associated with a deficiency of the chemical dopamine in the brain. The appropriate cell therapy treatment of patients suffering from Parkinson's disease is the introduction of purified dopamine-producing neurons into the patient's brain.

We intend to take a dopaminergic stem cell line, ReN004, into development as a cell therapy treatment for Parkinson's disease, targeted at those patients with advanced disease who are no longer responding to drug therapy. Fast track or equivalent accelerated status will be sought for the treatment.

Market opportunity

The worldwide market for Parkinson's disease is currently worth approximately $2 billion and is dominated by drugs that raise the level of dopamine in the brain. In the US alone, some one million patients are affected and 50,000 new patients are added annually.

Progress

ReNeuron has developed a stem cell line, ReN004, that can be induced to generate highly enriched, functional dopaminergic neurons. We are not aware of any other organisation that has generated dopamine-producing cells at these concentrations. ReN004 is being re-derived to GLP standards, ahead of pre-clinical efficacy testing.

We have research collaboration with a UK tissue scaffold company to develop a novel cell delivery scaffold that will enhance dopaminergic cell survival and differentiation of ReN004 once implanted.

We have also been awarded a grant from the US-based Michael J. Fox Foundation for Parkinson's Research (MJFF) to develop our ReN004 stem cell therapy for Parkinson's disease. The award was made under MJFF's Therapeutics Development Initiative, designed to catalyse and expand industry investment in pre-clinical Parkinson's drug development.

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