Crispr Therapeutics to present preclinical data on AATD at ESGCT 2025

CRISPR Therapeutics (CRSP) announced the acceptance of an abstract for oral presentation at the European Society of Gene and Cell Therapy, ESGCT, 2025 Annual Congress, taking place October 7-10, 2025. The presentation will introduce the Company’s novel SyNTase gene editing technology and highlight its application in single-dose in vivo gene correction to treat Alpha-1 Antitrypsin Deficiency, AATD, a rare genetic disorder. The abstract describes that SyNTase editing produces high levels of editing in SERPINA1-E342K human hepatocyte cell models without any detectable off-target effects. In a humanized mouse model, SyNTase editing components encapsulated in a lipid nanoparticle enabled highly efficient, specific, and potentially curative gene correction with a single intravenous dose with a well-tolerated safety profile. In a custom humanized rat model of AATD, SyNTase editing achieved potent gene correction of the E342 mutation with greater than70% mRNA correction and greater than3-fold total serum AAT upregulation, exceeding the established clinically protective threshold.