Rare Diseases Targeted by Arnatar’s ASO and RNA Silencing Combo

RNA therapeutics biotech Arnatar Therapeutics came out of stealth mode this week to announce its candidate antisense oligonucleotide (ASO) for the treatment of the rare disease Alagille syndrome had received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.

ART4 is an ASO designed to boost levels of the Jagged-1 (JAG1) protein, which is found at abnormally low levels in the majority people with the rare disease Alagille syndrome, which affects as many as one in 30,000 births in the U.S., due to mutations in the JAG1 gene (95% of cases).

Alagille syndrome doesn’t have a cure, and current treatments focus on managing the symptoms, which in addition to liver damage, include congenital heart defects, itching and poor growth, as well as vertebral abnormalities in