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RO7062931
- Development stage
- Phase 1
- Lead developer
- Roche
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
RO7062931 is an N-acetylgalactosamine (GalNAc)-conjugated single-stranded locked nucleic acid (LNA) antisense oligonucleotide designed to target hepatitis B virus (HBV) RNA transcripts. The drug specifically binds to a highly conserved sequence in the shared 3′ region common to all HBV RNA transcripts, leading to RNase H-mediated degradation of viral RNA and subsequent reduction in the translation of viral antigens such as hepatitis B surface antigen (HBsAg). The GalNAc conjugation enables hepatocyte-specific delivery via the asialoglycoprotein receptor, which is predominantly expressed on liver cells. RO7062931 was developed for subcutaneous administration and has been evaluated in phase 1 clinical trials for chronic hepatitis B, demonstrating dose-dependent reductions in HBsAg with a favorable safety profile[1][2][4].
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