ART4

ART4 is an upregulating antisense oligonucleotide (ASO) therapeutic candidate developed by Arnatar Therapeutics for the treatment of Alagille Syndrome (ALGS). It utilizes the proprietary ACT-UP1 platform to increase the expression of endogenous JAG1 (Jagged-1) protein, specifically targeting the haploinsufficiency caused by mutations in the JAG1 gene, which occurs in approximately 95% of ALGS patients. By restoring JAG1 levels, ART4 aims to promote proper bile duct development and reduce liver injury markers such as bile acids and ALT. The drug is administered subcutaneously on a once-monthly schedule and has received Orphan Drug and Rare Pediatric Disease Designations from the FDA.