A Phase 1/2, Open-Label, Single and Multiple Ascending Dose Study of CRMA-1001 in Adults With Chronic Hepatitis B

A Multi-Center, Phase 1/2, Open-Label, Single and Multiple Ascending Dose Study of CRMA-1001 to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy in Adults With Chronic Hepatitis B

This is an open-label study with single- and multiple-ascending dose arms followed by a dose expansion arm. The primary objective of the study is to determine the safety and tolerability of CRMA-1001 in adult participants with Chronic Hepatitis B. In addition, the pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of CRMA-1001 will be evaluated. CRMA-1001 is an epigenetic gene therapy delivered via intravenous (IV) infusion. Up to four dose levels will be tested. Participants will receive a single or multiple doses of CRMA-1001 and will remain on antiviral therapy during the dosing process.

Inclusion Criteria:

• Male/Female, weight 45-150 kg, age 18-64, inclusive
• Diagnosed with Chronic Hepatitis B
• On oral antiviral therapy
• ALT and AST <= 1.5 x ULN
• Total bilirubin <= ULN

Exclusion Criteria:

• Significant hepatic fibrosis or cirrhosis
• Current or prior liver disease other than HBV
• Other protocol-defined inclusion/exclusion criteria may apply