Drug intelligence / Profile preview

REP 9

Development stage
Discontinued
Lead developer
Replicor
Modality
RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

REP 9 is a first-generation nucleic acid polymer (NAP) developed by Replicor Inc. for the treatment of chronic hepatitis B virus (HBV) infection. Chemically, it is a 40-mer random-sequence phosphorothioate oligonucleotide. The drug's primary mechanism of action is the inhibition of the release of hepatitis B surface antigen (HBsAg) from infected hepatocytes into the blood. By blocking the assembly and secretion of HBsAg-containing subviral particles, REP 9 reduces the circulating HBsAg levels that typically exhaust the host's immune system. This reduction is intended to allow for the restoration of the host's innate and adaptive immune responses, potentially leading to viral clearance. Although REP 9 served as a critical proof-of-concept molecule in early clinical studies, it has been succeeded in development by more optimized NAPs, such as REP 2139 and REP 2165, which offer improved safety and potency profiles.

Other names
Random sequence 40-mer phosphorothioate oligonucleotide
02

Targets

HBsAg (Hepatitis B surface antigen)

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