Proteostasis Therapeutics


    Proteostasis Therapeutics is developing novel therapeutics designed to pharmacologically control the PN, either by restoring its normal state or enhancing the capacity of the compromised PN to create a therapeutic state sufficient to control or delay progression of disease. This novel approach of rebalancing the PN offers a broader application to genetic diseases than protein replacement therapy by providing access to many cell types, organelles, and tissues where recombinant proteins cannot be delivered. It further allows for improvement relative to single chaperones with the potential to address multiple diseases using the same therapeutic intervention.

    Our drug candidates should also complement existing strategies, with improved protein folding synergizing with molecular chaperones and intracellular clearance, thereby enhancing extracellular protein clearance approaches.

    Focus and Stage