A Phase 1/2, Open-Label, Multi-Center, Dose Escalation, Dose Expansion, and Single Repeat Dose Study of TSRA-196 in Adults With the PiZZ Genotype Who Have Lung and/or Liver Disease Associated with Severe Alpha-1 Antitrypsin Deficiency

Date Added
April 9th, 2026
PRO Number
Pro00149000
Researcher
Charlie Strange

List of Studies


Keywords
Drug Studies, Liver, Lung, Pulmonary, Rare Diseases
Summary

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.

Institution
MUSC
Recruitment Contact
Kristin Neff
843-792-1219
neffk@musc.edu



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