Proteostasis receives preclinical research funding for Parkinson’s disease

Using screening and structure-based medicinal chemistry optimization, the company identified potent and selective Usp14 inhibitors and expects to advance them as therapeutic candidates into clinical studies in 2015.

Inhibition of deubiquitinating enzyme Usp14 that modulates proteasome activity has shown to increase degradation of aggregation-prone proteins such as a-synuclein.

The Michael J. Fox Foundation CEO Todd Sherer said, "Of all the genetic targets discovered to date, alpha-synuclein has the strongest rationale for drug development toward a disease-modifying therapy for Parkinson’s disease."

The company designed an integrated platform to identify small molecules that modulate Proteostasis Network pathways involving protein folding, trafficking, and clearance.

Preclinical research revealed significant changes in a key cellular protein degradation pathway in PD patient brain samples.

Proteostasis chief medical officer Dr. David Weiner said, "We are pleased to collaborate with The Michael J. Fox Foundation as we work to advance proteostasis regulators into the clinic and closer to the many Parkinson’s disease patients in need of more effective treatments."