Clinical Research Directory

Key Inclusion Criteria: 1. Male sex 2. Gestational or adjusted (corrected) gestational age ≥ 37 weeks 3. Age at screening is 24 hours to 7 months 4. Weight ≥ 3.5 kg and ≤ 13.5 kg at screening 5. Has received age-appropriate vaccinations 6. Genetically confirmed OTCD 7. Severe neonatal OTCD defined by hyperammonemic crisis with elevated ammonia level of \>560 μmol/L and clinical symptoms within first week of life 8. Current or historical biochemical profile consistent with OTCD 9. Participant's parent(s)/LAR must be able to comprehend and be willing to provide a signed IRB/IEC-approved ICF. Key Exclusion Criteria: 1. Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to birth injury 2. Requiring urgent liver transplant due to liver failure as assessed by the PI. 3. Contiguous gene deletion involving the OTC gene and including at least the CYBB gene on the telomeric side or the TSPAN7 gene on the centromeric side. 4. Known or suspected major organ injury/dysfunction/anomalies. 5. Vital sign abnormalities 6. Laboratory abnormalities outside of laboratory normal ranges for urinalysis, complete blood count, and comprehensive metabolic panel that are attributable to comorbidities unrelated to OTCD 7. Treatment with any other gene therapy or gene editing therapy 8. Co-enrollment in any other clinical study unless approved by the sponsor. 9. Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data 10. Documented vertical transmission of HepA/HepB/HepC 11. Documented in-utero teratogen, substance, and/or alcohol exposure, which in the opinion of the Investigator may increase the participant's risk of developmental delays, congenital anomalies, and/or significant medical complications