Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Exclusion Criteria

• •1. History of clinically significant disease(s), in the opinion of the Investigator
• •2. Clinically significant screening laboratory values
• •3. Uncontrolled diabetes
• •4. Clinically significant anemia
• •5. Subjects who develop infection during screening must be asymptomatic for at least 7 days prior to dosing
• •6. Unwillingness to comply with study requirements
• •7. History of positive HIV, hepatitis C, or chronic hepatitis B
• •8. Uncontrolled hypertension
• •9. Malignancy within 5 years prior to study
• •10. Treatment with another investigational drug, biological agent, or device within 30 days of screening, or 5 half-lives of investigational drug
• •11. Treatment with any oligonucleotide or mRNA within 6 months of screening, with exceptions for some vaccinations and investigational treatments
• •12. History of gene therapy, hepatocyte or mesenchymal stem cell transplantation
• •13. Prior organ transplant
• •14. History of severe allergic reaction to a liposomal product
• •15. Recent history of, or current, drug or alcohol abuse
• •16. Dependence on inhaled (smoked or vaped) or oral cannabis products
• •17. Systemic corticosteroids within 6 weeks prior to screening
• •18. Blood donation of 50 to 499 mL within 30 days of screening or of .499 mL within 60 days of screening
• •19. Other conditions, in the opinion of the Investigator, that would make the subject unsuitable for participation