Arcturus Therapeutics Advances with ARCT-810 Study for OTC Deficiency

Arcturus Therapeutics ((ARCT)) announced an update on their ongoing clinical study.

Study Overview: Arcturus Therapeutics is conducting a Phase 2a study titled ‘A Phase 2a, Open-label, Multiple Ascending Dose Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency.’ The study aims to assess the safety and pharmacodynamics of ARCT-810, a potential treatment for OTC deficiency, a rare genetic disorder. This research is significant as it could lead to new therapeutic options for managing this condition.

Intervention/Treatment: The study tests ARCT-810, a biological intervention. ARCT-810 is an investigational medicinal product that includes Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP). It is designed to address the enzyme deficiency in patients with OTCD.

Study Design: This interventional study follows a sequential model where all participants receive five doses of ARCT-810 via intravenous infusion at 14-day intervals. The study is open-label, meaning no masking is involved, and its primary purpose is treatment-focused.

Study Timeline: The study began on June 28, 2024, and is currently recruiting participants. The latest update was submitted on March 13, 2025. These dates are crucial as they indicate the study’s progress and potential timelines for results.

Market Implications: The ongoing study of ARCT-810 could significantly impact Arcturus Therapeutics’ stock performance. Positive outcomes may boost investor confidence and position the company as a leader in genetic disorder treatments. This development is particularly relevant in the competitive landscape of mRNA-based therapies, where advancements can lead to substantial market shifts.

The study is ongoing, and further details are available on the ClinicalTrials portal.