CRISPR Therapeutics registers Phase 1/2 trial of CTX460 for AATD-related emphysema

CRISPR Therapeutics registered one new trial this week.

• CTX460 is a lipid nanoparticle formulation of a synthetic nucleotide template polymerase editor designed for in vivo gene editing of the SERPINA1 gene in adults with emphysema associated with severe alpha-1 antitrypsin deficiency.¹ The Phase 1/2 study, titled "A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)," is not yet recruiting, with an estimated enrollment of 68 participants¹ and an estimated start date of October 31, 2026. The study plans to assess safety and tolerability in Phase 1 to determine the recommended Phase 2 dose, measured by incidence of dose-limiting toxicities and adverse events¹, and in Phase 2 will evaluate absolute blood levels of total alpha-1 antitrypsin at Month 6¹.