Nucleic Acid DDS: Drug Delivery Technology Realizing Gene-Targeted Therapy ~Analysis of R&D Trends in Patents, Papers, and Grants~
Astamuse Co., Ltd. has released a report analyzing research and development trends in nucleic acid DDS (Drug Delivery System), a technology realizing gene-targeted therapy, based on patents, papers, and grants. The report details challenges in intracellular delivery of nucleic acid drugs, the evolution of next-generation technologies (LNP, active targeting, etc.), and annual trends in related patents.
📋 Article Processing Timeline
- 📰 Published: April 28, 2026 at 23:11
- 🔍 Collected: April 28, 2026 at 14:31
- 🤖 AI Analyzed: April 28, 2026 at 14:49 (17 min after Collected)
Astamuse Co., Ltd. (Head office: Chiyoda-ku, Tokyo; Representative Director and President: Ayumu Nagai) has comprehensively analyzed trends in the technical field of nucleic acid DDS using its proprietary innovation database (innovation and R&D information such as papers, patents, startups, and grants) and compiled the findings into a report.
What is Nucleic Acid DDS?
Nucleic Acid DDS (Drug Delivery System) refers to a system of technologies for safely and efficiently delivering nucleic acid drugs, such as siRNA (small interfering RNA), ASO (antisense oligonucleotides), and mRNA (messenger RNA), into target cells.
Nucleic acid drugs are next-generation therapeutic agents that can regulate gene function from its very source. The mechanism for delivering drugs to target cells within the body (nucleic acid DDS) utilizes ultrafine lipid particles called lipid nanoparticles (LNPs). After attaching to the cell surface, LNPs are taken into the cell and enclosed within small sacs inside the cell called "endosomes."
For the nucleic acid (the active pharmaceutical ingredient) to exert its effect, it must break out of this sac and reach its site of action within the cell. To break the sac, it is necessary to act on the negatively charged membrane of the endosome and destroy it. However, conventional LNPs had a problem: they could not sufficiently acquire a positive charge in the acidic environment within the endosome, making them ineffective at breaking the membrane.
Furthermore, nucleic acids are easily broken down by enzymes (nucleases) present in the body and have the disadvantage of being difficult to pass through cell membranes due to their negative charge.
Latest developments in nucleic acid DDS aim to overcome these multiple challenges and maximize the effect of nucleic acid drugs.
What significantly differentiates next-generation nucleic acid DDS from conventional types is its aim not only to "deliver" the drug to the target cell but also to reliably "send it into" the exact location within the cell where the drug actually works.
Such technologies have already reached the practical application stage, and their effectiveness has been demonstrated globally through applications in COVID-19 mRNA vaccines and treatments for genetic diseases.
The main nucleic acid DDS technologies and their features, including both next-generation and conventional types, are as follows:
- Lipid Nanoparticles (LNP, conventional to next-generation): Encapsulate mRNA and siRNA in lipids to protect nucleic acids from degradation in vivo while delivering them to target cells.
- Active Targeting (next-generation): Attaching marker molecules (antibodies, glycan ligands) in advance to selectively deliver drugs to target tissues, such as cancer cells.
- Conjugate DDS (conventional to next-generation): A method that directly binds specific molecules (sugars, peptides, antibodies, etc.) that assist in cell binding to the nucleic acid, which is the drug's active ingredient, thereby enabling efficient uptake into targeted cells without the need for a separate "carrier" particle. This technology continues to be refined and evolve.
- Combination with Modified Nucleic Acid Technology: A technique that modifies the chemical structure of the nucleic acid itself (e.g., phosphorothioate modification, 2'-O-methyl modification, 2'-fluoro modification) to make it less susceptible to degradation in the body and prolong the drug's effect. This method has been utilized in combination with DDS for a long time.
- Endosomal Escape Function (conventional to next-generation): A function that allows drugs taken into cells to escape from being trapped in intracellular "sacs" by breaking the sac from the inside, enabling the drug to reach its actual site of action. Ionizable lipids become cationic in an acidic environment, disrupting the endosomal membrane and releasing nucleic acids into the cytoplasm. This has undergone years of improvement.
- Adaptability to Various Therapeutic Modalities (next-generation): Characterized by high versatility, capable of responding to a wide range of nucleic acid drugs (siRNA, ASO, mRNA, miRNA, CpG oligonucleotides, etc.) with different mechanisms of action, such as suppressing gene expression, repairing genes, or activating immunity.
This report utilized Astamuse's proprietary database to analyze technological trends related to "Nucleic Acid DDS" in patents, papers, and grants (competitive research funding).
Trends in Patents Related to Nucleic Acid DDS
From Astamuse's patent database, a patent population of 2,256 cases including technical keywords related to nucleic acid DDS such as "lipid nanoparticles," "mRNA delivery," and "nucleic acid drugs" in their abstracts was extracted. A "future prediction" analysis was conducted to identify technological elements that have seen recent advancements based on the annual trends of keywords. By understanding the changes in keywords, technologies predicted to be in the spotlight currently or in the future can be quantitatively evaluated.
Figure 1 shows the annual transition of keywords in nucleic acid DDS-related patents filed since 2015.
Figure 1: Annual Transition of Keywords in Nucleic Acid DDS-Related Patents (2015-2024)
What is Nucleic Acid DDS?
Nucleic Acid DDS (Drug Delivery System) refers to a system of technologies for safely and efficiently delivering nucleic acid drugs, such as siRNA (small interfering RNA), ASO (antisense oligonucleotides), and mRNA (messenger RNA), into target cells.
Nucleic acid drugs are next-generation therapeutic agents that can regulate gene function from its very source. The mechanism for delivering drugs to target cells within the body (nucleic acid DDS) utilizes ultrafine lipid particles called lipid nanoparticles (LNPs). After attaching to the cell surface, LNPs are taken into the cell and enclosed within small sacs inside the cell called "endosomes."
For the nucleic acid (the active pharmaceutical ingredient) to exert its effect, it must break out of this sac and reach its site of action within the cell. To break the sac, it is necessary to act on the negatively charged membrane of the endosome and destroy it. However, conventional LNPs had a problem: they could not sufficiently acquire a positive charge in the acidic environment within the endosome, making them ineffective at breaking the membrane.
Furthermore, nucleic acids are easily broken down by enzymes (nucleases) present in the body and have the disadvantage of being difficult to pass through cell membranes due to their negative charge.
Latest developments in nucleic acid DDS aim to overcome these multiple challenges and maximize the effect of nucleic acid drugs.
What significantly differentiates next-generation nucleic acid DDS from conventional types is its aim not only to "deliver" the drug to the target cell but also to reliably "send it into" the exact location within the cell where the drug actually works.
Such technologies have already reached the practical application stage, and their effectiveness has been demonstrated globally through applications in COVID-19 mRNA vaccines and treatments for genetic diseases.
The main nucleic acid DDS technologies and their features, including both next-generation and conventional types, are as follows:
- Lipid Nanoparticles (LNP, conventional to next-generation): Encapsulate mRNA and siRNA in lipids to protect nucleic acids from degradation in vivo while delivering them to target cells.
- Active Targeting (next-generation): Attaching marker molecules (antibodies, glycan ligands) in advance to selectively deliver drugs to target tissues, such as cancer cells.
- Conjugate DDS (conventional to next-generation): A method that directly binds specific molecules (sugars, peptides, antibodies, etc.) that assist in cell binding to the nucleic acid, which is the drug's active ingredient, thereby enabling efficient uptake into targeted cells without the need for a separate "carrier" particle. This technology continues to be refined and evolve.
- Combination with Modified Nucleic Acid Technology: A technique that modifies the chemical structure of the nucleic acid itself (e.g., phosphorothioate modification, 2'-O-methyl modification, 2'-fluoro modification) to make it less susceptible to degradation in the body and prolong the drug's effect. This method has been utilized in combination with DDS for a long time.
- Endosomal Escape Function (conventional to next-generation): A function that allows drugs taken into cells to escape from being trapped in intracellular "sacs" by breaking the sac from the inside, enabling the drug to reach its actual site of action. Ionizable lipids become cationic in an acidic environment, disrupting the endosomal membrane and releasing nucleic acids into the cytoplasm. This has undergone years of improvement.
- Adaptability to Various Therapeutic Modalities (next-generation): Characterized by high versatility, capable of responding to a wide range of nucleic acid drugs (siRNA, ASO, mRNA, miRNA, CpG oligonucleotides, etc.) with different mechanisms of action, such as suppressing gene expression, repairing genes, or activating immunity.
This report utilized Astamuse's proprietary database to analyze technological trends related to "Nucleic Acid DDS" in patents, papers, and grants (competitive research funding).
Trends in Patents Related to Nucleic Acid DDS
From Astamuse's patent database, a patent population of 2,256 cases including technical keywords related to nucleic acid DDS such as "lipid nanoparticles," "mRNA delivery," and "nucleic acid drugs" in their abstracts was extracted. A "future prediction" analysis was conducted to identify technological elements that have seen recent advancements based on the annual trends of keywords. By understanding the changes in keywords, technologies predicted to be in the spotlight currently or in the future can be quantitatively evaluated.
Figure 1 shows the annual transition of keywords in nucleic acid DDS-related patents filed since 2015.
Figure 1: Annual Transition of Keywords in Nucleic Acid DDS-Related Patents (2015-2024)