CRISPR Therapeutics AG today reported new preclinical data for its SyNTase‑based candidate CTX460, a therapy targeting alpha‑1 antitrypsin deficiency (AATD). The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress and were released in a press statement dated 2025‑10‑12.
The preclinical studies demonstrated >90% mRNA correction, a 5‑fold increase in total alpha‑1 antitrypsin levels, and a >99% serum M‑AAT:Z‑AAT ratio in both mouse and rat models of AATD. The effect was durable following a single dose of the lipid‑nanoparticle‑encapsulated CTX460.
CTX460 is the first investigational candidate to use CRISPR Therapeutics’ SyNTase editing platform, which combines compact Cas9 proteins with engineered polymerases for precise gene correction. The company expects to initiate a clinical trial in mid‑2026, marking the next step toward potential regulatory approval.
This breakthrough expands CRISPR Therapeutics’ pipeline into a rare disease with significant unmet need, showcases the versatility of its SyNTase platform, and positions the company for future clinical development and potential revenue streams.
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