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PMM2 mRNA
- Development stage
- Preclinical
- Lead developer
- Moderna
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
PMM2 mRNA is an experimental mRNA-based therapy being developed by Moderna in collaboration with the University of Utah for the treatment of Phosphomannomutase 2-Congenital Disorder of Glycosylation (PMM2-CDG). The therapy utilizes lipid nanoparticle (LNP) technology to deliver mRNA encoding the human PMM2 enzyme, primarily targeting the liver to restore enzyme activity and correct coagulation abnormalities and other hepatic phenotypes associated with the disease. It is being explored as a standalone treatment or as a bridge therapy for patients awaiting AAV-based gene replacement, particularly those with pre-existing AAV antibodies or severe liver disease.
- Other names
- human PMM2 mRNALNP-encapsulated PMM2 mRNA
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