The goal of the study is to determine the safety and efficacy of the treatment. Efficacy will be measured by standardized tests for Parkinson’s patients, as well as brain imaging studies.
CERE-120 is a novel gene therapy product that delivers the neurturin (NTN) gene via an adeno-associated virus (AAV) type2 vector delivery system.
The naturally occurring NTN gene encodes the NTN protein that maintains survival of dopamine-producing nerve cells that are required for normal bodily movement and are the nerves that degenerate in Parkinson’s disease patients.
“CERE-120 has the potential to significantly slow or even halt the progression of symptoms of this devastating disease,” said Dr Jeffrey Ostrove, president and CEO of Ceregene. “Numerous studies in animal models, including the most widely accepted animal models of Parkinson’s disease, consistently demonstrated that this NTN gene therapy product may be able to improve symptoms as well as retard the progression of the disease.”
The study is being conducted at the University of California, San Francisco Medical Center and Rush University Medical Center in Chicago.