Crispr presents new preclinical data from SyNTase gene editing platform

CRISPR Therapeutics (CRSP) 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. CTX460 Key Preclinical Data Highlights: A single dose of CTX460 achieved significant, dose-dependent correction of liver DNA in both rat and mouse AATD models, with near saturating editing in hepatocytes at doses as low as 0.1 mg/kg. Furthermore, a single dose of CTX460 was able to achieve greater than90% mRNA correction at a clinically relevant dose of 0.5 mg/kg in PiZ mice. Relative to pre-dose values, editing with CTX460 yielded a greater than5-fold increase in total serum AAT levels with an M-AAT:Z-AAT ratio of over 99% in the serum of PiZ rats. AAT upregulation was linearly correlated with editing efficiency. Durability of editing was maintained in both rat and mouse models for up to 7 weeks and 9 weeks, respectively.