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mRNA-3210
- Development stage
- Discontinued
- Lead developer
- Moderna
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
mRNA-3210 is an investigational mRNA-based therapy developed by Moderna for the treatment of phenylketonuria (PKU), a rare genetic disorder caused by deficiency of the enzyme phenylalanine hydroxylase (PAH). The drug consists of messenger RNA encoding human PAH, encapsulated in a lipid nanoparticle (LNP) delivery system. Once administered intravenously, the mRNA instructs cells to produce functional PAH enzyme, aiming to restore normal metabolism of phenylalanine and reduce its toxic accumulation in patients with PKU. This approach addresses an unmet need for effective therapies beyond dietary management and currently available medications[2][3][5][7].
02
Targets
PAH (Phenylalanine hydroxylase)
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