At Arcturus, we are focused on advancing mRNA therapies designed to treat the underlying cause of rare genetic conditions like OTC deficiency.
Kelsey Bobka, OTCD Carrier and Parent
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:
Our bodies constantly create our own mRNA to relay the instructions from our genes to the “assembly line” to make components like enzymes that are essential for our bodies to function. Genes are the “recipes” for creating our body’s components and are located in a protected place called the nucleus, which acts as the “command center” of our cells. The job of mRNA is to carry the instructions (or the “message”) to the cell’s assembly line outside the nucleus and act as a template to gather the necessary ingredients to assemble the final components.
Sometimes, a person’s genes can carry instructions that are different from the typical instructions included in that gene. This difference is called a variant or mutation, and it causes the gene to perform differently, which can lead to faulty components (e.g., enzymes) that do not work properly.
What is ARCT-810?
ARCT-810 is an investigational mRNA treatment developed to target the root cause of OTC deficiency. The mRNA in ARCT-810 is designed to act as a template for making OTC enzyme to help remove excess ammonia from the body.
ARCT-810 IS DIFFERENT THAN GENE THERAPY. The mRNA appears to be short-lived and does not alter one’s genes. 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 to produce OTC enzymes using the cell’s own processes.
