This study is a single-arm, open-label, dose-escalation, exploratory study to evaluate the safety, tolerability, and efficacy of a single administration of GS1168 Injection in Chinese adult phenylketonuria (PKU) with PAH mutation.
Understand the purpose and risks of the study and voluntarily sign Informed Consent Forms (ICF).
Male or female, aged 18-55 years.
Diagnosis of PKU due to PAH mutation, and be inadequately controlled with current therapy (e.g., dietary management) in the judgement of the investigator.
Intolerant of unresponsive to available treatment such as Kuvan or Palynziq, and have come off the medication for at least 28 days prior to signing the ICF.
Diagnosis of PKU due to other gene mutations.
Body weight ≥100 kg or Body Mass Index (BMI) ≥30 kg/m² at screening.
Presence of anti-AAV8 neutralizing antibody.
ALT or AST >1.25 × Upper Limit of Normal (ULN)