Therapeutic oligonucleotides have attracted great interest due to their potency and potential for changing the therapeutic landscape of many pathological conditions – including those of viral origin.
The COVID-19 pandemic highlighted the need for antiviral therapies that can respond to both current and emerging viral threats. Alongside preventive measures, effective treatments remain essential, particularly for people at increased risk of severe disease – the elderly, individuals with comorbidities, and immunocompromised patients.
Advances in our understanding of SARS-CoV-2 have created new opportunities for RNA-targeted therapies. Therapeutic oligonucleotides offer a precise way to inhibit viral replication by binding directly to viral RNA before viral proteins can be produced. Our approach focuses on a highly conserved RNA sequence that is essential for viral replication. Because this region changes very little as the virus evolves, it represents a stable therapeutic target with the potential to remain effective across multiple viral variants.
Built on the ESiNAR-X® platform, ASC1R is a highly specific therapeutic oligonucleotide designed to selectively target a conserved SARS-CoV-2 RNA sequence.
ASC1R is a therapeutic oligonucleotide developed using the ESiNAR-X® platform to selectively target a highly conserved SARS-CoV-2 RNA sequence that encodes the viral RNA-dependent RNA polymerase (RdRp), an enzyme essential for viral replication. By binding to this sequence, ASC1R triggers degradation of the viral RNA, preventing the virus from producing the proteins it needs to replicate.
Preclinical studies demonstrated potent antiviral activity together and excellent tolerability. ASC1R reduced viral RNA levels by more than 98% after a single application in lung cell lines and completely eliminated its target RNA in vivo. No evidence of organ toxicity was observed, even at doses 100 times higher than the effective dose.
Selectively binds to the conserved SARS-CoV-2 RdRp sequence.
More than 30 million molecules delivered per lung cell.
Reduced SARS-CoV-2 RNA levels by up to 100% after a single application in mice.
Excellent tolerability in preclinical studies with no evidence of organ toxicity even at high doses.
The properties demonstrated by ASC1R may support the development of next-generation RNA-targeted antivirals for other diseases of viral origin.