Lead Sponsor

BioMarin Pharmaceutical

Age

14 - No limit years

Sex

ALL

Healthy Volunteers

No

•Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
•Ability and willingness to maintain dietary protein intake consistent with baseline intake
•Willingness and capable per investigator opinion to comply with study procedures and requirements
•Plasma Phe Levels \> 600umol/L

•Clinically significant liver dysfunction or disease
•Prior treatment with gene therapy
•Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study