SERPINA1(human)-AAV8-1 DP clinical trials

Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

This study is looking into a new treatment for people with a lung condition called alpha-1 antitrypsin deficiency (AATD), which can cause emphysema. It's the first time this treatment is being given to humans, and researchers want to check if it's safe and how the body handles it.