T9001

During the past decade it was discovered that a gene (DYT-1) and the protein it encodes, called “Torsin”, is critical for normal cellular function in the brain. Torsin is a “chaperone protein” that prevents mutations of proteins that cause neurological disorders such as dystonia and Parkinson’s disease. The core Torsin technology licensed by QRxPharma was developed at the University of Alabama in the US, where scientists demonstrated that a known and already approved drug appears to activate the Torsin system, preventing mutations in other proteins and improving movement disorders in preclinical models of these diseases. Preliminary anecdotal clinical observations with patients suffering from dystonia also support these findings.

QRxPharma intends to co-develop this research in a Phase II study coordinated through the University of Alabama. It is believed that investigators at the Beth Israel Medical Center in New York City will agree to host clinical studies, and the Dystonia Medical Research Foundation will agree to collaborate on the trial which should be completed during 2008.
QRxPharma has exclusively licensed the molecules and portfolio of IP that surrounds these inventions at the University of Alabama.

http://www.qrxpharma.com/go.cfm?do=Page.View&pid=37

paula

Thank you Paula!

CNS = Central Nervous System

http://www.qrxpharma.com/go.cfm?do=Page.View&pid=21



Many neurological diseases, including Parkinson’s,
dystonias,
Huntington’s,
Amytrophic Lateral Sclerosis (ALS)
and Alzheimer’s

involve the toxic effects of misfolded proteins. While there are no cures for these diseases of the brain, over the past decade significant advances have been made in understanding the molecular and cellular biology of Parkinson’s and dystonia. These discoveries have pointed to the potential for new therapeutics that treat the root cause of such neurodegenerative disorders and not simply the effects of disease.