RheoGene receives grant for Parkinson’s research

RheoGene has developed technology to manage gene expression, a key component of gene-based therapies. RheoGene’s therapeutic system uses a patented small-molecule mediator that can turn genes ‘on’ or ‘off’ as well as adjust the level of gene activity similar to the way a rheostat regulates electric current.

“This project has the potential to revolutionize the clinical application of gene therapy, not only for the millions of people with Parkinson’s disease, but for countless numbers afflicted by other health ailments as well,” said Deborah Brooks, president and CEO of the Michael J Fox Foundation for Parkinson’s Research (MJFF).

Although research continues on gene-based treatments for a variety of ills, much of the strategy’s early promise remains to be realized because of risks associated with failure to regulate gene expression, which can bring about worrisome side effects.

A RheoGene-led team will work to further the technology, called the RheoSwitch Therapeutic System (RTS), for safe and effective use to control the intensity and timing of gene expression. This will be accomplished using a specially developed activator drug that is administered orally to act as an ‘on switch’. When the drug is no longer taken, gene expression stops. Preclinical testing in animal models has indicated that RTS can work as envisioned by researchers.

Gene therapy for Parkinson’s disease is the first target for RheoGene. Future research will concentrate on applications to cancers and diabetes.

Parkinson’s disease gradually destroys brain cells that produce dopamine, a chemical messenger crucial for the cellular communication that controls muscle movement. The National Institutes of Health estimates that between 500,000 and 1.5 million people have Parkinson’s disease in the US alone. Because risk increases with age, these rates will increase as the population ages.