Arcturus Therapeutics to Develop ARCT-810 for Treatment of Ornithine Transcarbamylase (OTC) Deficiency
“We are pleased to select ARCT-810 as a candidate for clinical development,” said
“Our development candidate, ARCT-810, represents a novel approach to treat OTC deficiency,” said Dr. Pad Chivukula, Chief Scientific Officer of Arcturus. “We are moving into the clinic on the strength of preclinical proof-of-concept in our LUNAR-OTC program, demonstrating that LUNAR technology can deliver messenger RNA to liver cells and results in expression of functional OTC protein in animal models. Replacing the deficient OTC protein restores the urea cycle pathway, resulting in reduced plasma ammonia and urinary orotate concentrations. We look forward to IND-enabling studies which will allow the advancing of ARCT-810 into the clinic and investigation of its utility in humans.”
About Ornithine Transcarbamylase Deficiency (OTCD)
OTC deficiency is caused by mutations in the OTC gene which leads to a non-functional or deficient OTC enzyme. OTC is a critical enzyme in the urea cycle, which takes place in liver cells, and converts ammonia to urea. This conversion does not occur properly in patients with OTC deficiency and ammonia accumulates in their blood, acting as a neurotoxin and liver toxin. This can cause severe symptoms including vomiting, headaches, coma and death. OTC deficiency is an inherited disease that can cause developmental problems, seizures and death in newborn babies. It is an X-linked disorder, so is more common in boys. Patients with less severe symptoms may present later in life, as adults. There is currently no cure for OTC deficiency, apart from liver transplant. However, this treatment comes with significant risk of complications such as organ rejection, and transplant recipients must take immunosuppressant drugs for the rest of their lives. Current standard of care for OTC patients is a low-protein diet and ammonia scavengers to try and prevent patients from accumulating ammonia. These treatments do not address the underlying cause of disease.
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Source: Arcturus Therapeutics, Inc.