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messenger RNA encoding human alpha-galactosidase A
- Development stage
- Preclinical
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
**Messenger RNA encoding human alpha-galactosidase A** is an investigational **mRNA-based biologic** formulated in **lipid nanoparticles** for systemic delivery of mRNA encoding the human **alpha-galactosidase A** enzyme for the treatment of **Fabry disease**. After administration, the mRNA is translated intracellularly to produce alpha-galactosidase A, aiming to restore deficient lysosomal enzyme activity and reduce accumulation of **globotriaosylceramide** and **lyso-globotriaosylceramide** in affected tissues. Based on the provided context, the program is in **preclinical development** and is being explored as an alternative to conventional enzyme replacement therapy, with animal studies showing restoration of enzyme activity and reduction of substrate burden in plasma, liver, kidney, heart, and spleen.
- Other names
- mRNA encoding human alpha-galactosidase Ahuman alpha-galactosidase A mRNALNP-formulated messenger RNA encoding human alpha-galactosidase A
02
Targets
IGF2R (Cation-independent mannose-6-phosphate receptor)Gb3 (Globotriaosylceramide)GLA (Alpha-galactosidase A)
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