INFECTIOUS DISEASES
COVID-19
Nearly three years after the outbreak of the COVID-19 pandemic, the SARS-CoV-2 virus is still causing lasting damage around the world, impacting the health and lives of millions. By September 2022, the World Health Organization reported more than 600 million cumulative cases as well as 6.5 million deaths associated with the disease.
Preventive measures, such as widespread administration of vaccines, is playing a role in reducing the severity of cases. For those who require treatment, clinicians can try to treat the disease and its symptoms with existing drugs. However, an effective therapy specifically targeted against SARS-CoV-2 in patients is still lacking.
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. Targeting conserved SARS-CoV-2 RNA sequences essential for viral replication offers a rational approach to inhibit viral infection and thereby halt disease progression.
The ESiNAR-X® platform served as basis for the development of highly specific oligonucleotide-based modalities against SARS-CoV-2. Among them, ASC1R was selected due to its potency.
ASC1R
The therapeutic potential of ASC1R demonstrated by the results of in vitro and ex vivo studies could translate into substantial clinical benefits for patients with COVID-19 (Read more). ASC1R is a potent inhibitor of the SARS-CoV-2 RNA which targets the conserved region encoding the RNase dependent RNA polymerase and induces the enzymatic cleavage of the viral genome thereby inhibiting the replication of the virus. Reduction of the viral load could effectively prevent a cytokine storm with consequent multi-organ damage. ASC1R is intended to treat patients with COVID-19 irrespective of the viral load and the severity of symptoms and may be especially beneficial for the most at-risk group such as the elderly, patients with comorbidities and immunocompromised people.
Recognizes exclusively the target RNA
specifically binds to the RdRp sequence
Exhibits spontaneous delivery to lung cells
more than 30 million molecules per cell
Exceptional efficacy in patient samples
reduction of SARS-CoV-2 levels after a single dose by >98%
Favorable safety profile
preclinical studies showed excellent tolerability
Furthermore, in the context of infectious diseases, these results may provide implications for R&D of analogous antivirals for other diseases of viral origin.