RNA therapeutics for metabolic disorders
Thuy-Duong Vu1, Sheng-Che Lin2, Chia-Ching Wu3
1Center for Biomedicine and Community Health, International School, Vietnam National University, Hanoi, Vietnam.
Progress in Molecular Biology and Translational Science
|February 15, 2024
Summary
RNA therapeutics offer new hope for metabolic disorders, with seven approved antisense oligonucleotide (ASO) and small interfering RNA (siRNA) drugs targeting liver proteins. mRNA therapeutics also show promise.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Metabolic disorders are rising globally, with existing treatments showing limitations.
- RNA therapeutics have emerged as a promising new class of treatments for these conditions.
- Significant advancements in chemical modifications have enhanced the potential of RNA-based therapies.
Purpose of the Study:
- To review the current landscape of RNA therapeutics for metabolic disorders.
- To highlight approved RNA drugs and their targets.
- To discuss the potential of emerging mRNA therapeutics.
Main Methods:
- Review of clinical studies and market-approved RNA drugs.
- Analysis of RNA therapeutic mechanisms, including antisense oligonucleotides (ASO) and small interfering RNA (siRNA).
- Examination of chemical modifications enhancing RNA drug efficacy and safety.
Main Results:
- Seven RNA drugs, primarily ASO and siRNA, are approved for metabolic disorders affecting liver proteins.
- These drugs target conditions like hereditary transthyretin-mediated amyloidosis and hypercholesterolemia.
- Ongoing development shows potential for mRNA therapeutics.
Conclusions:
- RNA therapeutics, particularly ASO and siRNA, have demonstrated significant success in treating metabolic disorders.
- Chemical modifications are key to the efficacy and safety of these oligonucleotide-based drugs.
- mRNA therapeutics represent a future frontier in managing metabolic diseases.
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