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Clinical trial

Official Title: A Phase 1/2 Multicenter, Open-label, First-in-Human Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460, a Lipid Nanoparticle Formulation of SyNTase Editing Components for In Vivo Editing of the Serine Protease Inhibitor A1 (SERPINA1) Gene in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)

NCT07850076 is registered in ClinicalTrials.gov with CRISPR Therapeutics as lead sponsor.

NCT NCT07850076
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Registry record

As filed in ClinicalTrials.gov.

Title
Official Title: A Phase 1/2 Multicenter, Open-label, First-in-Human Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460, a Lipid Nanoparticle Formulation of SyNTase Editing Components for In Vivo Editing of the Serine Protease Inhibitor A1 (SERPINA1) Gene in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)
Lead sponsor
CRISPR Therapeutics
Phase
Phase 1/2
Overall status
Not yet recruiting
Start date
October 31, 2026
Completion date
June 30, 2030
Enrollment
68 (estimated)
Sites
Not reported

Conditions

As the registry lists them; a condition with its own page is linked.

alpha-1 antitrypsin deficiency (aatd)