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-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 BOSTON, Oct. 10, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported new preclinical data from its novel SyNTase™ gene editing platform for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The data are featured in an oral presentation titled "Single-dose in vivo gene correction of AATD via LNP-delivered SyNTase editors" at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress. The AATD program, designated CTX460™, is the first investigational candidate to use SyNTase editing and is expected to enter the clinic in mid-2026.
Posted In: CRSP
