CRISPR Therapeutics has reported a string of positive R&D updates in recent weeks. On October 10, 2025, CRSP presented preclinical results for CTX460, its SyNTase-based therapy for alpha-1 antitrypsin deficiency. The data – highlighted at the ESGCT conference – showed that a single dose of CTX460 delivered by lipid nanoparticles corrected the disease-causing mutation in >90% of liver cells and boosted functional AAT protein ~5-fold in animal models globenewswire.com. “The preclinical results demonstrate the potential of CTX460, developed using our novel SyNTase editing platform, to correct the mutation with precision and efficiency,” said CRISPR CMO Dr. Naimish Patel globenewswire.com. These findings suggest a “best-in-class” approach to addressing AATD’s underlying cause, and the company aims to start a first-in-human trial in mid-2026.
