ARCT-810 interim Phase 2 update reports urea-cycle biomarker activity in OTC deficiency
Company / institutional announcement · Arcturus Therapeutics
Source published: · Event / reporting date: 2026-09-23
Site published: 2026-11-03 · Updated: 2026-11-03
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What changed
Arcturus reported interim Phase 2 findings for ARCT-810 in ornithine transcarbamylase deficiency, including lower or maintained-normal fasting ammonia and reduced glutamine, with generally favorable tolerability. The company plans to incorporate next-generation ARCT-2601 into an amended protocol near year-end. Separately reported large expression gains for its new delivery platform came from nonhuman-primate studies, not comparative human efficacy data.
Why it may matter
The update supports continued investigation of hepatic mRNA enzyme replacement, while long-term clinical benefit and next-generation dosing still require human evidence.
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