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Targeting USP14, a protein that suppresses proteostasis

Proteostasis Therapeutics is developing drugs that inhibit USP14, a protein that suppresses proteasomes, which are responsible for degrading unwanted proteins within cells. Their research suggests that this will reduce proteasomic function and so reduce the amount of dangerous aggregates within the human brain, such as the tau protein associated with Alzheimer’s disease and the α-synuclein associated with Parkinson’s.

Working under a proteostasis network model, which measures and defines proteostasis by the amounts and relationships of more than a thousand proteins, Proteostasis Therapeutics is using an -omics-based approach to develop therapeutic compounds that can restore balance to this network.

The company also has drugs in Phase 1 clinical trials that modulate the cystic fibrosis transmembrane conductance (CFTR) protein as a method of treating cystic fibrosis.