Pluvia Biotech PAH chaperone

Pluvia Biotech is developing a small-molecule pharmacological chaperone for the treatment of phenylketonuria (PKU), a rare genetic metabolic disorder. PKU is caused by mutations in the liver enzyme phenylalanine hydroxylase (PAH), which leads to the accumulation of neurotoxic levels of phenylalanine in the blood and brain. This pharmacological chaperone is designed to bind to and stabilize the PAH enzyme, preventing mutation-associated misfolding and restoring its native enzymatic activity. By enhancing the stability and function of the endogenous mutated enzyme, the therapy aims to reduce phenylalanine levels and provide a treatment option for patients, including those who may not respond to existing therapies like sapropterin dihydrochloride. The program is currently in preclinical development and originated as a spin-out from the University of Bergen.

Other names

Pluvia Biotech PKU programPAH pharmacological chaperone