This is a Phase 1/2, open-label, multi-center clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of TSRA-196, a gene editing compound, in adults with severe alpha-1 antitrypsin deficiency (PiZZ genotype) and associated lung and/or liver disease. Participants will receive a single intravenous dose of TSRA-196 in a dose-escalation phase followed by dose-expansion cohorts.
The study will assess safety outcomes, pharmacokinetics, and changes in serum alpha-1 antitrypsin levels and lung function to determine whether TSRA-196 can safely increase functional AAT levels and inform selection of an appropriate dose for further clinical development.