The Proteostasis Network consists of more than 1,000 proteins organized into pathways that can be regulated
The PN ensures that every protein within a cell will reach its final destination correctly folded with appropriate function or be degraded and cleared to prevent damage. Disease, genetic mutations, environmental factors, and aging can cause the PN to become imbalanced, which can lead to a decrease in protein quality control contributing to diseases categorized as loss-of-function or gain-of-toxic function disorders. Loss-of-function diseases, such as cystic fibrosis, are often caused by inherited mutations resulting in inefficient folding and excessive degradation. Gain-of-toxic-function disorders, which include Alzheimer’s, Huntington’s, and Parkinson’s diseases, appear to arise from aggregation-associated toxicity.
Proteostasis Therapeutics is developing novel therapeutics designed to pharmacologically control or rebalance 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.