(QR) announced today a new collaborative research agreement with The Rockefeller University aimed at developing a treatment for Huntington’s disease (HD).
The collaboration will focus on preclinical research involving QR’s lead drug candidate, Posiphen®. Investigators at Rockefeller will conduct experiments in stem cell culture models of HD to determine, if Posiphen lowers the levels of the toxic huntingtin protein in these cells, if it promotes differentiation into neuronal cells and, if it prolongs the cell life of the HD cells. Results from the collaboration could lead to clinical studies in HD patients.
Posiphen inhibits amyloid precursor protein (APP) and therefore a major pathway leading to Alzheimer’s disease. It also inhibits α-Synuclein, an important player in a pathway linked to Parkinson’s disease. Recently, the same compound also was shown to inhibit huntingtin, the protein that is seen at high levels in HD.
“QR Pharma’s technology targets the mRNA of a number of proteins that are over expressed in several neurological disorders, such as Alzheimer’s disease, Parkinson’s disease, Down Syndrome, and Huntington’s disease in a manner that has the potential to impact disease progression,” said Maria Maccecchini, Ph.D., CEO of QR Pharma.
Source: Business Wire