Companies in #Metabolic Rare Disease (OTC deficiency)
Found 1 company operating in this sector.
Arcturus Therapeutics is a messenger RNA medicines company focused on liver and respiratory rare diseases, built on the STARR self-amplifying mRNA platform and the LUNAR lipid-mediated delivery system. Its pipeline includes ARCT-032 (LUNAR-CF, an mRNA therapeutic candidate for cystic fibrosis in Phase 2), ARCT-810 (LUNAR-OTC for ornithine transcarbamylase deficiency, Phase 2), KOSTAIVE (a self-amplifying COVID-19 vaccine approved in Japan), and Phase 1 influenza (ARCT-2138) and pandemic-influenza (ARCT-2304) programs. Headquartered in San Diego, the company beat Q2 2026 earnings estimates by $0.16 per share and traded up 21.9% on August 7, 2026 on above-average volume.