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RG-101
- Development stage
- Discontinued
- Lead developer
- Regulus Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
RG-101 is a GalNAc-conjugated antisense oligonucleotide therapeutic that targets microRNA-122 (miR-122), a liver-specific microRNA essential for hepatitis C virus (HCV) replication. By inhibiting miR-122, RG-101 disrupts the HCV life cycle, leading to significant reductions in viral load. The drug was developed primarily for the treatment of chronic hepatitis C infection and was administered via subcutaneous injection. Clinical studies demonstrated substantial antiviral effects after single-dose administration, but development was discontinued due to safety concerns related to hyperbilirubinemia caused by inhibition of bilirubin transport[1][2][3][6][8][9].
- Other names
- RG 101RG101RG-101
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