CRISPR Therapeutics

CRISPR Therapeutics Presents New Preclinical Data for CTX460 Demonstrating In Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase Editing Platform

Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) highlight a potential best-in-class profile The AATD program, CTX460, is the first investigational candidate to emerge from the SyNTase editing platform and the Company expects to initiate a clinical trial with CTX460 in mid-2026 CTX460 showed specific and durable effects, with >90% mRNA correction, a 5-fold...
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