Modified Nucleic Acid Conjugates

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This patent discloses modified nucleic acid conjugates featuring a 2',3'-O ketal moiety, which can be chemically attached to nucleic acids (such as DNA or RNA, including antisense, siRNA, or miRNA molecules). The resulting conjugates enhance the selective and efficient delivery of therapeutic nucleic acids into target cells (including human cells), improving their stability and performance for gene therapy, gene silencing, or gene modulation applications.

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

• Targeted delivery of therapeutic siRNA or antisense oligonucleotides for gene silencing in human diseases (e.g., cancer, genetic disorders).
• Development of RNAi-based drugs with improved delivery to specific tissues or organs (e.g., liver, lung, heart) for in vivo therapy.
• Gene upregulation or downregulation therapies for conditions associated with specific gene expression.
• Ex vivo modification of cells (e.g., in cell therapy protocols) prior to transplantation into patients.
• Delivery of mRNA or gene-editing molecules (like CRISPR components) into mammalian cells.
• Basic research for studying gene function by introducing modified nucleic acids into various cell types.

BenefitsContent extracted from patent full text and abstract with AI.

• Enhanced efficiency and specificity of nucleic acid delivery into target cells, including difficult-to-transfect cells.
• Increased stability of therapeutic nucleic acids in biological fluids (e.g., protecting siRNA from degradation).
• Versatility: compatible with a variety of nucleic acid types (DNA, RNA, modified bases) and applicable to both therapeutic and research settings.
• Potential for reduced dosing and off-target effects (side effects) due to improved delivery and targeting.
• Applicability to both in vitro (laboratory) and in vivo (clinical) scenarios.
• Enables development of advanced RNA-based therapeutics with improved pharmacological profiles.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The present invention refers to a nucleic acid conjugate comprising at least one 2',3'-O ketal moiety. This conjugate is suitable for the target-specific or target-selective delivery of nucleic acid molecules into cells such as mammalian cells including human cells with high efficacy. Thus, a new delivery vehicle for therapeutic nucleic acid molecules including antisense molecules, siRNA molecules, miRNA molecules, antagomirs or precursors of such molecules is provided.