Pharmaceutical Business review

QR Pharma, Rockefeller University enter research deal for Posiphen to treat Huntington’s disease

As part of the deal, the two parties will focus on preclinical research involving QR Pharma’s lead drug candidate Posiphen.

The deal will see investigators at Rockefeller 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 the drug promotes differentiation into neuronal cells as well as prolongs the cell life of the HD cells.

According to the partners, results from this collaboration could lead to clinical studies in HD patients.

QR Pharma CEO Maria Maccecchini said: "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."

Posiphen inhibits amyloid precursor protein (APP), a major pathway leading to Alzheimer’s disease (AD) and it also inhibits a-Synuclein, a major player in a pathway linked to Parkinson’s disease (PD).

The company said recently that the same compound was also shown to inhibit huntingtin, the protein that is seen at high levels in Huntington’s disease.