New hope for those living with Cystic Fibrosis

LunairCF is a clinical trial investigating the use of mRNA therapy (also known as ARCT-032) as an alternative to treating Cystic Fibrosis lung disease.

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.

Is the LunairCF trial right for you?

LunairCF is a Phase 2, open-label study investigating whether mRNA therapy is safe and effective for the treatment of people living with CF. The study is being enrolled at trial sites around the world. Find out if it’s right for you or someone you love with CF.

FAQ

What is Cystic Fibrosis?

CF is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that results in reduced or absent CFTR function in the membranes that line the airways, intestinal tract, and other organs. CFTR is a membrane channel that transports salt ions that are critical to maintain hydration in the airways. In CF, the CFTR is absent or does not function well, which leads to sticky mucus, inflammation, and progressive lung damage.

What is a Clinical Study?

Clinical studies (or trials) are a type of research performed to learn about the effects of investigational new drugs or treatments. The studies follow predetermined procedures, called the clinical study protocol. Clinical studies are the most important way for researchers to better understand whether a new drug or treatment is safe and effective for a particular condition.

What is ARCT-032 and How is it Administered?

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 that lack a functional copy. These cells then use the newly delivered instructions provided by the mRNA to create functional CFTR and restore a proper salt and water balance in the airways.

To learn more about mRNA therapy in CF, please visit the Cystic Fibrosis Foundations website.