Drug intelligence / Profile preview

ASC1R

Development stage
Preclinical
Lead developer
Selecta Biosciences
Modality
Small Molecules, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

ASC1R is a novel therapeutic oligonucleotide designed to inhibit the replication of SARS-CoV-2, the virus responsible for COVID-19. It works by specifically binding to two regions of the viral RNA that are essential for coding a subunit of RNA-dependent RNA polymerase (RdRp), an enzyme critical for viral genome replication. By blocking this sequence, ASC1R effectively suppresses RdRp production and thereby halts viral replication within host cells. In preclinical studies, ASC1R demonstrated potent antiviral activity with over 94% reduction in viral RNA in cell cultures and similar efficacy in samples from COVID-19 patients. The compound also showed excellent safety and tolerability profiles in rodent models, even at doses much higher than those expected to be therapeutically effective. Notably, ASC1R can spontaneously enter cells without additional delivery agents and remains localized in the cytoplasm where it targets viral RNA[1][6].

02

Targets

SARS-CoV-2 RNA

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