Rna for Inhibiting Sars-Cov-2 Replication

Simple SummaryContent extracted from patent full text and abstract with AI.

This invention discloses specifically designed double-stranded RNA molecules (siRNA and shRNA) that can efficiently inhibit replication of the SARS-CoV-2 virus, which causes COVID-19. These RNAs target a highly conserved region (the 5'-UTR leader sequence) in the viral genome, leading to the degradation of both genomic and subgenomic viral RNAs. The invention includes pharmaceutical compositions (including inhalable forms and those delivered via viral vectors) containing these RNAs for therapeutic use against SARS-CoV-2 and potentially related coronaviruses including SARS-CoV-1.

Use CasesContent extracted from patent full text and abstract with AI.

• Therapeutic treatment for COVID-19 patients to directly inhibit SARS-CoV-2 replication.
• Inhalation-based medication to deliver siRNA/shRNA directly to the lungs, the primary site of infection.
• Treatment of infections caused by other related coronaviruses (e.g., SARS-CoV-1 and emerging variants) due to targeting of highly conserved genomic sequences.
• Prevention of coronavirus infections in high-risk or exposed populations by prophylactic administration.
• Research tool for studying virus replication and host-virus interactions in cellular models.
• Pharmaceutical formulations for extended delivery, including viral vector-based (AAV) systems or combinations of multiple RNAs to reduce development of viral escape mutants.

BenefitsContent extracted from patent full text and abstract with AI.

• Highly specific: Targets a highly conserved region in the SARS-CoV-2 genome, minimizing the chance of viral escape and mutations reducing effectiveness.
• Broad-spectrum antiviral activity: Effective against multiple SARS-CoV-2 variants, the original SARS-CoV-1, and possibly future coronaviruses sharing the target sequence.
• Efficient inhibition: Demonstrated up to 99% reduction of viral replication in vitro at nanomolar concentrations.
• Potential for inhalation therapy: Enables direct delivery to the lungs for rapid and localized effect with reduced systemic side effects.
• Combination strategies: Allows use of multiple siRNAs/shRNAs to further prevent viral escape and resistance.
• Reduced risk of off-target effects: Target site is chosen to avoid matches in the human transcriptome, increasing safety.
• Compatibility with advanced delivery: Can be administered as aerosols or with gene therapy vectors like AAV for targeted and efficient delivery.

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Patent Abstract

The invention relates to an RNA for inhibiting severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) replication. According to an aspect of the invention, the RNA is an at least partially double-stranded RNA having a sequence being at least 95 % identical to a sequence chosen from the group consisting of SEQ ID NO. 6 and SEQ ID NO. 11.