Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency - NCT05222178

To determine incidence and severity of Treatment Emergent Adverse Events and adverse events of special interest of a single administration of HMI-103, To evaluate the efficacy of HMI-103 on reduction of plasma Phe concentration at each dose level, To evaluate the effect of HMI-103 on plasma Phe concentration relative to treatment guidelines for PKU, (and 2 more...)