US National PKU Alliance invests in Pluvia Biotech
National PKU Alliance (NPKUA), the US patient advocacy organization for PKU, is investing in Pluvia’s drug development program
NPKUA is joining Pluvia’s investors (Sarsia, Investinor and Trond Mohn Foundation) to support the final preclinical development steps for PBAS499, Pluvia’s lead therapeutic candidate, towards clinical studies.
PBAS499 can potentially restore endogenous phenylalanine hydroxylase (PAH) activity by stabilizing and increasing the functional performance of misfolded PAH variants associated with PKU. Acting as a pharmacological chaperone, it targets the root cause of the disorder and is designed to provide an oral disease-modifying treatment approach.