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.
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
Significant hepatic fibrosis or cirrhosis
Current or prior liver disease other than HBV
Other protocol-defined inclusion/exclusion criteria may apply
Sequential
CRMA-1001
GeneticEpigenetic gene silencing therapy delivered by intravenous (IV) infusion
Safety and tolerability of single and multiple doses of CRMA-1001
Incidence and severity of treatment-emergent adverse events
Long-term safety of single and multiple doses of CRMA-1001
Incidence and severity of treatment emergent adverse events
Pharmacokinetics of CRMA-1001 components (Cmax)
Maximum concentration (Cmax) in plasma
Pharmacokinetics of CRMA-1001 components (Tmax)
Time of maximum concentration (Tmax) in plasma
Pharmacokinetics of CRMA-1001 components (terminal clearance rate)
Clearance rate in terminal clearance phase (CL) in plasma
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