CTX340

CTX340 is an investigational in vivo gene-editing therapy developed by CRISPR Therapeutics. It is designed to inhibit the production of hepatic angiotensinogen (AGT), a validated target for modulating the renin-angiotensin-aldosterone system (RAAS) and normalizing blood pressure. The therapy uses lipid nanoparticle (LNP) delivery of Cas9 mRNA and guide RNA to achieve targeted editing of the AGT gene in liver cells. In preclinical studies, CTX340 demonstrated approximately 60% liver editing and about 90% reduction in AGT protein levels, resulting in a sustained reduction of around 30 mmHg in blood pressure for up to three months in hypertensive rat models. The primary indication under development is refractory hypertension, with additional investigation for acute hepatic porphyria.