Structure and use of 5' phosphate oligonucleotides

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The invention describes the use of oligonucleotides (short strands of nucleic acids) that have a free, uncapped 5' phosphate group, such as 5' triphosphate RNA, which can activate a specific immune pathway in cells via the RIG-I receptor. This activation leads to the production of key immune cytokines, including type I interferon (IFN), IL-18, and IL-1β, which help mount strong anti-viral, anti-bacterial, and anti-tumor immune responses. The approach can be used to both stimulate the immune system to fight infections (viruses, bacteria, parasites), cancer, allergies, and immunodeficiencies, and to selectively target responses to diseased or infected cells without harming healthy ones.

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

• Developing new antiviral therapies or vaccines by using oligonucleotides to robustly induce type I interferon production and other immune activators.
• Enhancing antibacterial or antiparasitic treatments, including therapy for intracellular bacterial infections.
• Cancer immunotherapy, including direct induction of tumor cell death (apoptosis) and immune activation against cancer cells.
• Immunostimulatory agents for combating immunodeficiency or immunosuppression (e.g., after chemotherapy).
• Targeting autoimmune diseases by selectively modulating or inhibiting inflammatory cytokine responses.
• Molecular adjuvants for vaccines, boosting immune responses to antigens in vaccine formulations.
• In vitro tools for studying innate immune responses, cytokine production, and cell-specific immune activation.
• Potentially as part of personalized medicine approaches, where oligonucleotides are designed to target unique disease-related RNA sequences in specific patient cells.

BenefitsContent extracted from patent full text and abstract with AI.

• Enables precise activation of the body's innate immunity by mimicking viral or bacterial molecular patterns, leading to potent and rapid immune responses.
• Can selectively target diseased cells (e.g., infected or tumor cells) while sparing healthy tissues, reducing undesirable side effects.
• Facilitates the creation of more effective, localized therapies for infections and cancer, potentially overcoming limitations and side effects of conventional interferon therapy.
• Allows for lower-cost alternatives to recombinant protein interferon therapies, as the oligonucleotides can be synthesized more economically and induce natural cytokine production in patients.
• Broad range of potential indications – viral, bacterial, parasitic infections, tumors, allergies, autoimmune diseases, immunodeficiencies, and immunosuppression.
• The structure-activity relationship knowledge enables rational design of highly effective drug candidates or adjuvants.
• Bacterial RNA can be used as a safe, inexpensive therapeutic agent compared to attenuated viruses or synthetic oligonucleotides.
• Oligonucleotides can be engineered for sequence and cell specificity, increasing safety and therapeutic efficacy.

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Inventors & Applicants

Patent Abstract

Oligonucleotides bearing free, uncapped 5' phosphate group(s) are recognized by RIG-I, leading to the induction of type I IFN, IL-18 and IL-1β production. Bacterial RNA also induces type I IFN production. 5' phosphate oligonucleotides and bacterial RNA can be used for inducing an anti-viral response or an anti-bacterial response, in particular, type I IFN and/or IL-18 and/or IL-1β production, in vitro and in vivo and for treating various disorders and diseases such as viral infections, bacterial infections, parasitic infections, tumors, allergies, autoimmune diseases, immunodeficiencies and immunosuppression. Single- stranded 5' triphosphate RNA can be used for inducing an anti-viral response, an anti- bacterial response, or an anti-tumor response, in particular, type I IFN and/or IL-18 and/or IL- 1β production, in a target cell-specific manner.