On October 1, 2025 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported 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 (Press release, CRISPR Therapeutics, OCT 1, 2025, View Source [SID1234656376]). 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.
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CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform. SyNTase editors represent a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. Together, these components enable gene editing with greater efficiency and precision, while also supporting scalable manufacturing.
Using AI-guided structural modeling and large-scale screening, the polymerase was optimized to support gene correction activity based on synthetic nucleotide templates. When integrated with Cas9, SyNTase editors can utilize engineered templates with improved serum stability, enabling higher target correction efficiency.
The abstract describes that SyNTase editing produces high levels of editing (up to 95%) in SERPINA1-E342K human hepatocyte cell models without any detectable (<0.5%) off-target effects. In a humanized mouse model, SyNTase editing components encapsulated in a lipid nanoparticle (LNP) enabled highly efficient, specific, and potentially curative gene correction with a single intravenous (IV) dose (≤0.5 mg/kg) 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 >70% mRNA correction and >3-fold total serum AAT upregulation, exceeding the established clinically protective threshold. Together, these data provide proof-of-concept for a potentially best-in-class therapeutic modality to address the underlying cause of AATD and support SyNTase editing as a promising platform for the treatment of many monogenic disorders. Additional results beyond those included in the abstract will be presented at the conference.
Presentation Details
Title: Single-dose in vivo gene correction of AATD via LNP-delivered SyNTase editors
Abstract Number: OR096
Session Type: Oral Presentation
Session Title: SESSION 12c: Gene Editing III: Technology & applications
Session Date and Time: Friday, October 10, 2025, 11:00 a.m. – 1:00 p.m. CEST
The accepted abstract is available online on the ESGCT website for congress registrants. Any updated data, new graphics, and follow-up information to be presented during the oral presentation sessions is embargoed until 8:00 a.m. CEST on the day of the presentation. A copy of the presentation will be available at www.crisprtx.com once the presentation concludes.