Clinical Trials
Clinical Trials
Clinical trials are an important step to understand whether a medicine works for its intended purpose. Please see our active clinical trials below. For any questions email [email protected].CYSTIC FIBROSIS
LunairCF is a clinical trial investigating the use of mRNA therapy (also known as ARCT-032) as an alternative to treat Cystic Fibrosis. While ARCT-032 is a potential therapy for all those living with CF, it may be especially helpful for those individuals with nonsense and rare CF mutations that do not respond to CFTR modulators.
Overview
ARCT-032 (study treatment) is an inhaled messenger RNA (mRNA) encased in tiny fat particles, i.e., a lipid-mediated delivery system called LUNAR®. ARCT-032 is delivered by a special nebulizer directly to the lungs to target a normal copy of CFTR mRNA directly to lung cells. These cells then use the instructions provided by the mRNA to create functional CFTR and restore a proper salt and water balance in the airways.Visit LunairCF
Clinical Trial Website
Visit ClinicalTrials.gov
Website
OTC
The mRNA in LUNAR-OTC (also known as ARCT-810) is designed to be a template for making OTC enzyme. Arcturus uses its lipid nanoparticle technology, called LUNAR® to deliver the mRNA template for OTC to the liver, the place where the OTC enzyme is made.
Overview
Arcturus is conducting a clinical trial to determine whether ARCT-810 can address the root cause of OTC deficiency and lessen the disease burden experienced by those living with the condition. The goal of the ARCT-810-04 study is to evaluate the safety and tolerability of ARCT-810 in people with OTC deficiency and its efficacy in restoring urea cycle function.Visit LUNAR-OTC
Clinical Trial Website
Visit ClinicalTrials.gov
Website