Experimental therapy for rare metabolic disease shows early promise
Disease control
Terminated
This study tested a new medicine called ARCT-810 in 8 people aged 12 to 65 with ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder that causes dangerous ammonia buildup. The main goal was to check the drug's safety and how the body processes it. The trial was st…
Phase: PHASE2 • Sponsor: Arcturus Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:51 UTC