5'Triphosphate oligonucleotide with blunt end and uses thereof

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

This invention relates to a specifically designed synthetic oligonucleotide (a short nucleic acid) with at least one blunt end carrying a 5' triphosphate. This molecular configuration robustly activates the RIG-I pathway in cells, leading to a potent type I interferon (IFN, especially IFN-α) response that is crucial for antiviral defense. The invention also covers its use for preparations and pharmaceutical compositions to induce antiviral states, and as adjuncts in treatment or prevention of infections, tumors, and immune disorders.

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

• Antiviral therapies, especially in viral infections where IFN response is beneficial (e.g., influenza, hepatitis B/C, RSV, HIV).
• Cancer immunotherapy—stimulating the immune system to attack tumors via RIG-I-mediated IFN production.
• Vaccine adjuvant—enhancing immune response to vaccines via IFN induction.
• Prevention or treatment of immune disorders that can benefit from modulation of IFN pathways, such as autoimmune diseases.
• In vitro research tool to study RIG-I signaling or type I IFN induction mechanisms.
• Gene silencing therapies combined with immune activation (using oligonucleotide for RNAi and IFN stimulation simultaneously).

BenefitsContent extracted from patent full text and abstract with AI.

• Precisely designed structure ensures high potency and specificity in activating RIG-I, leading to strong IFN responses.
• Minimal off-target immune activation compared to previous methods thanks to defined structural requirements (blunt end, 5' triphosphate).
• Can be used with high purity and reproducibility due to defined chemical synthesis processes.
• Effective at short lengths (≥21 base pairs), enabling ease of synthesis and delivery.
• Possibility to combine gene silencing (RNAi) and immune stimulation in one molecule, increasing therapeutic versatility.
• Applicable for both human and veterinary therapeutics, as well as basic research.
• Potential for local or systemic administration, and for use with various delivery methods (e.g., sprays, injections).

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Inventors & Applicants

Patent Abstract

The present invention provides an oligonucleotide which is capable of activating RIG-I and inducing an anti-viral, in particular, an IFN, response in cells expressing RIG-I. The oligonucleotide of the present invention has a double-stranded section of at least 21bp, at least one 5' triphosphate, and at least one blunt end which bears a 5' triphosphate. The present invention further provides the use said oligonucleotide for inducing an anti-viral, in particular, an IFN, response in vitro and in vivo. The present invention additionally provides the use of said oligonucleotide for preventing and/or treating diseases or conditions such as infections, tumors/cancers, and immune disorders.