UPR: Researching the effect of modulating the unfolded protein response pathway

The Proteostasis Network and cellular stress

The unfolded protein response (UPR) is a part of the Proteostasis Network (PN) that is activated when cells experience stress due to the accumulation of unfolded proteins within the endoplasmic reticulum (ER). UPR dysfunction is associated with many diseases that are now recognized as protein conformational disorders, including:

  • Genetic
  • Neuro-degenerative
  • Retinal-degenerative

Mediating the cellular stress response

Proteostasis Therapeutics sees the modulation of the UPR as a promising therapeutic approach to restore and improve the proper cellular stress response in protein conformational disorders. UPR modulators have high potential as novel therapies for a variety of diseases with high unmet medical need.

This program is developed in collaboration with Astellas Pharma.