Pluvia develops small molecules as pharmacological chaperones to restore activity of misfolded PAH enzymes to help patients with Phenylketonuria (PKU) to live normal lives
The most common inborn metabolic failure: Incorrect folding of PAH enzyme which results in insufficient phenylalanine degradation. Untreated, this can cause severe brain damage. The condition has been screened by a simple blood test at birth since 1967. Approximately 50,000 are diagnosed with the condition in the western world. Pluvia is addressing a substantial unmet medical need in a vulnerable patient group with very limited alternative options. The company is working with a novel target with the potential to help a large subset of patients, as stand-alone and add-on therapy.