SAN DIEGO — September 18, 2026
Arcturus Therapeutics announced plans to provide an update on its ARCT-810 Phase 2 clinical program in ornithine transcarbamylase (OTC) deficiency and its broader mRNA liver therapeutics platform during a virtual presentation on September 23, 2026. The presentation will include an overview of the clinical program and platform technologies, with Marshall Summar, M.D., a recognized expert in urea cycle disorders and former member of the NIH UCD Consortium, participating in the event. Arcturus said it will issue a press release summarizing the presentation before the event.
ARCT-810 Targets the Underlying Cause of OTC Deficiency
ARCT-810 is an investigational intravenously administered mRNA therapeutic designed to deliver functional OTC enzyme expression in the liver. OTC deficiency is an inherited urea cycle disorder caused by mutations in the X-linked OTC gene, resulting in deficient or non-functional OTC enzyme and impaired conversion of toxic ammonia into urea. Elevated ammonia can lead to metabolic crises and serious neurological complications. ARCT-810 is designed to restore OTC enzyme activity in hepatocytes and potentially improve urea cycle function, reduce abnormal glutamine levels and maintain normal ammonia concentrations.
Arcturus Advances mRNA Liver Therapeutics Platform
ARCT-810 uses Arcturus’ LUNAR® lipid-mediated delivery platform, proprietary lipid library, mRNA design construct and manufacturing technologies to deliver OTC mRNA to liver cells. The candidate has received Orphan Drug, Fast Track and Rare Pediatric Disease designations from the U.S. FDA, as well as Orphan Medicinal Product designation and an approved Pediatric Investigation Plan from the European Medicines Agency. The program represents a key application of Arcturus’ mRNA technology toward rare metabolic diseases in which conventional supportive treatments do not directly address the underlying genetic deficiency.
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Arcturus Expands Rare Disease RNA Therapeutics Pipeline
The ARCT-810 program forms part of Arcturus’ broader strategy to develop RNA medicines for liver and respiratory rare diseases. The company’s technology portfolio includes LUNAR lipid-mediated delivery, STARR® self-amplifying mRNA technology and mRNA drug-substance and drug-product manufacturing capabilities. Its pipeline also includes RNA therapeutic candidates for cystic fibrosis, while the company has established collaborations involving mRNA manufacturing and pandemic preparedness. The upcoming ARCT-810 presentation is expected to provide further clinical and platform updates as Arcturus advances its OTC deficiency program.
Source :Arcturus Therapeutics press release
