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ARCT-2601
- Development stage
- Unknown
- Lead developer
- Arcturus Therapeutics
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
ARCT-2601 is an intravenously administered, next-generation investigational mRNA therapeutic being developed by Arcturus Therapeutics for the treatment of ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder. The drug is designed to deliver mRNA encoding the functional OTC enzyme to the liver, restoring urea cycle function and preventing toxic ammonia buildup. ARCT-2601 utilizes Arcturus' proprietary LUNAR 2.0™ lipid nanoparticle (LNP) delivery platform, which features improved lipids and an enhanced formulation process that has demonstrated significantly greater potency and protein expression compared to its predecessor, ARCT-810.
- Other names
- LUNAR-OTC
02
Targets
OTC (Ornithine carbamoyltransferase)
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