ART104

ART104 is a first-in-class antisense oligonucleotide (ASO) developed by Arnatar Therapeutics for the treatment of Alagille Syndrome (ALGS). ALGS is a rare genetic disorder primarily caused by haploinsufficiency of the *JAG1* gene, which leads to a deficiency in the Jagged1 ligand and subsequent impairment of the Notch signaling pathway. This impairment results in multi-organ defects, most notably chronic cholestasis and liver damage. ART104 is designed to address the genetic root cause of the disease by increasing the expression of the Jagged1 protein, thereby restoring functional Notch signaling. The drug has received Orphan Drug Designation and Pediatric Rare Disease Designation from the FDA. Clinical development is currently in the early stages, with the first patient dosed in a Phase 1 trial in early 2025. Note: The identifier ART104 was also historically used by Arana Therapeutics for an unrelated antibody-based therapeutic targeting solid tumors.