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.
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