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CRISPR Therapeutics Presents New Preclinical Data for CTX460™ Demonstrating In Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase™ Editing Platform
Globenewswire· 2025-10-10 12:00
-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 increase in total AAT levels, and >99% serum M-AAT:Z-AAT ratio in AATD disease models- ZUG, Switzerland and BOSTO ...