Arcturus Therapeutics: Clinical Trials for OTC

Who are Arcturus Therapeutics?

Founded in 2013, Arcturus Therapeutics, is a United States based pharmaceutical company, which is focused on developing life-changing therapies and vaccines for those with rare disorders, including OTC deficiency. Arcturus develops medicines made with a molecule known as messenger RNA (mRNA). Each mRNA is uniquely designed to treat a specific disease.

What work is Arcturus Therapeutics doing?

Arcturus is developing ARCT-810, an investigational mRNA therapy that is designed to address the underlying cause of OTC deficiency. When a person has OTC deficiency, the body is not able to process ammonia in the same way and a buildup of ammonia can occur, causing distress and illness.

 The mRNA in ARCT-810 is designed to be a template for making a healthy OTC enzyme, which removes excess ammonia from the body through a process known as the urea cycle. Arcturus uses its lipid nanoparticle technology called LUNAR to deliver the mRNA template for OTC to the liver, where the OTC enzyme is made. Arcturus believes that mRNA and the LUNAR lipid nanoparticle work together to make a functioning OTC enzyme, which removes excess ammonia from the body.

ARCT-810-03 Study

The goal of the ARCT-810-03 study is to evaluate the safety and tolerability of six single doses of ARCT-810 in individuals ages 12-65 with a confirmed diagnosis of OTC deficiency. This study will help measure whether repeated doses of ARCT-810 can increase OTC activity and improve urea cycle function in the body.


The ARCT-810-03 study will take up to 30 weeks to complete and all travel expenses for participants such as meals, transportation, and lodging will be covered. Interested individuals should contact the study doctor listed under the following UK sites on for more information.

  • Great Ormond Street Hospital for Children
  • University College London Hospitals
  • Queen Elizabeth Hospital Birmingham
  • Salford Royal Hospital

More information

If you or someone you know are interested in the ARCT-810-03 study or learning more about Arcturus, please visit

For all other inquiries, contact or visit

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