U.S., Nov. 13 -- ClinicalTrials.gov registry received information related to the study (NCT07227207) titled 'A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)' on Nov. 03.
Brief Summary: This is a Phase 1/2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK/PD parameters of TSRA-196 in adults with the PiZZ genotype who have lung and/or liver disease associated with severe alpha-1 antitrypsin deficiency (AATD)
Study Start Date: Dec., 2025
Study Type: INTERVENTIONAL
Condition:
Alpha-1 Antitrypsin Deficiency (AATD)
Intervention:
DRUG: TSRA-196
TSRA-196 is an in-vivo genome editing product f...