Have you or a loved one been diagnosed with OTC deficiency?

At Arcturus, we are developing mRNA medicines designed to treat the underlying cause of rare genetic conditions like OTC deficiency.

What is OTC Deficiency?

ORNITHINE TRANSCARBAMYLASE (or-nuh-theen trans-car-bamma-lace) DEFICIENCY, or OTC DEFICIENCY, is a rare genetic disorder caused by a missing or deficient OTC enzyme. OTC is an important enzyme of the urea cycle. When someone has OTC deficiency, ammonia does not get removed but instead builds in the body, which can cause serious illness or life-threatening symptoms.

People living with OTC deficiency must take extra steps to monitor their condition and ensure ammonia levels stay low, which may include:

Low-protein diet
Amino acid supplements
Medical foods
Medicines that remove excess ammonia

New treatments are currently being studied to address the root cause of OTC deficiency and lessen the disease burden experienced by those living with this condition.

What is ARCT-810?

ARCT-810 is a new investigational treatment that is designed to address the cause of OTC deficiency.

Messenger RNA, or mRNA, carries the template for making functional OTC enzyme.
Lipid nanoparticle sphere contains tiny fat particles that protect the mRNA and allow it to be delivered into liver cells.
We believe these 2 parts work together in ARCT‑810 to enable the body to make functioning OTC enzyme to remove excess ammonia.

ARCT-810 is different than gene therapy. From our current understanding: the mRNA is short-lived and does not change one’s genes or cause permanent and irreversible effects. Also, ARCT-810 does not use viruses to carry the mRNA to the liver cells. Instead, by using LUNAR® technology, ARCT-810 addresses the cause of OTC deficiency by helping produce OTC enzymes using the cell’s own processes.