CTX450

CTX450 is an investigational in vivo gene-editing therapy developed by CRISPR Therapeutics. It utilizes lipid nanoparticle (LNP) delivery of CRISPR/Cas9 to target and edit the ALAS1 gene in the liver. By inhibiting hepatic production of 5’-aminolevulinate synthase 1 (ALAS1), CTX450 prevents accumulation of neurotoxic aminolevulinic acid (ALA) and porphobilinogen (PBG), which are implicated in acute hepatic porphyria (AHP). Preclinical studies have demonstrated approximately 70% liver editing and a ~97% reduction in ALAS1 protein, normalizing disease biomarkers in AHP mouse models. The therapy is being positioned as a potential one-time treatment for AHP, with plans to initiate clinical trials in the second half of 2025[1][3][4][5][9].

Brand names

CTX450CTX-450CTX 450

Other names

CTX-450CTX450CTX 450