CRISPR Therapeutics announced the acceptance of an abstract for oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, introducing its novel SyNTase gene editing technology. This proprietary, next-generation, site-specific gene correction platform represents a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. This technology aims for greater efficiency and precision in gene editing.
The preclinical data highlighted the application of SyNTase editing in single-dose in vivo gene correction to treat Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder. SyNTase editing produced high levels of editing, up to 95%, in SERPINA1-E342K human hepatocyte cell models without any detectable off-target effects, defined as less than 0.5%. This precision and efficiency are critical for developing safe and effective gene therapies.
In a humanized mouse model, SyNTase editing components delivered via lipid nanoparticles (LNP) enabled highly efficient, specific, and potentially curative gene correction with a single intravenous dose of ≤0.5 mg/kg, demonstrating a well-tolerated safety profile. Furthermore, in a humanized rat model of AATD, SyNTase editing achieved potent gene correction of the E342 mutation with over 70% mRNA correction and more than a threefold total serum AAT upregulation, exceeding the established clinically protective threshold. These results provide strong proof-of-concept for a potentially best-in-class therapeutic modality.
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