The deficiency of ornithine Transcarbamylase (OTC) is a rare genetic disease that provides severe abnormalities in the urea cycle. There are nearly 10,000 cases by the prevalence of OTC deficiency globally. The shortage of the OTC enzyme in liver cells leads to elevated levels of ammonia in the blood, which may cause seizures, coma, and death in untreated patients. Currently, no treatment is approved by FDA for OTC deficiency.
OTC protein replacement therapy or OTC mRNA therapy are drug candidates because the expression of the OTC enzyme in the liver of patients with OTC deficiency may be able to restore normal cycle activity, preventing neurological damage and the need for liver transplantation.
A mRNA therapeutics is developed for OTC deficiency. ARCT-810 is based on the lipid-mediated LUNAR delivery platform of Arcturus, which delivers OTC mRNA to liver cells and enables them to produce functional OTC enzymes. Arct-810 has demonstrated a favorable safety profile and was well tolerated in Phase 1a (healthy volunteers) and Phase 1b studies (OTC deficient patients). Currently, Arcturus has now implemented a phase 2 multiple-dose study targeting OTC deficiency adolescents and adults. US FDA granted orphan drug designation to ARCT-810.
Moderna rolled out a new program for ornithine transcarbamylase (OTC) deficiency in 2022 —— mRNA-3139 encoding OTC. And mRNA-3139 is based on a lipid nanoparticles (LNP) delivery system.
Yaohai Bio-Pharma Offers One-Stop Solution for RNA
Custom Deliverables
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Grade
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Deliverables
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Specification
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Applications
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non-GMP
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Drug Substance, mRNA
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0.1~10 mg (mRNA)
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Preclinical research such as cell transfection, Analytical method development, Pre-stability studies, Formulation development
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Drug Product, LNP-mRNA
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GMP, Sterility
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Drug Substance, mRNA
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10 mg~70 g
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Investigational new drug (IND), Clinical trial authorisation (CTA), Clinical trial supply, Biologic license application (BLA), Commercial supply
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Drug Product, LNP-mRNA
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5000 vials or pre-filled syringes/ cartridges
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mRNA Therapeutics Pipelines for OTC Deficiency
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Code Name
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Target Protein
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Indications
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Manufacturer
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Latest Stage
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ARCT-810, LUNAR-OTC
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OTC enzyme
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Ornithine Transcarbamylase (OTC) deficiency
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Arcturus
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Phase II
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mRNA-3139
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OTC enzyme
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OTC deficiency
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Moderna
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Pre-clinical
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