WO2022230990A1 - 人工核酸及びそれを用いた核酸の送達方法 - Google Patents

人工核酸及びそれを用いた核酸の送達方法 Download PDF

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WO2022230990A1
WO2022230990A1 PCT/JP2022/019343 JP2022019343W WO2022230990A1 WO 2022230990 A1 WO2022230990 A1 WO 2022230990A1 JP 2022019343 W JP2022019343 W JP 2022019343W WO 2022230990 A1 WO2022230990 A1 WO 2022230990A1
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nucleic acid
cationic
delivery
group
hydrophilic polymer
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French (fr)
Japanese (ja)
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寛子 宮本
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Japan Science and Technology Agency
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Japan Science and Technology Agency
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Priority to EP22795903.8A priority Critical patent/EP4331617A4/en
Priority to US18/288,674 priority patent/US20240238325A1/en
Priority to AU2022266383A priority patent/AU2022266383A1/en
Priority to CN202280031158.8A priority patent/CN117295528A/zh
Priority to JP2023517627A priority patent/JPWO2022230990A1/ja
Publication of WO2022230990A1 publication Critical patent/WO2022230990A1/ja
Anticipated expiration legal-status Critical
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A61K47/6931—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Definitions

  • X + is a functional group containing the cationic group
  • Z represents O or S
  • W represents -O- or -NR 4 -, where R 4 is hydrogen or 10 alkyl groups. * means a bond with the adjacent structural unit.
  • nucleic acid delivery structure of [9] which is a nanoscale structure in which a plurality of the nucleic acid delivery structures are assembled.
  • FIG. 1 is a schematic diagram illustrating the use of a nucleic acid analogue as a carrier.
  • the nucleic acid analog has a cationic group and a hydrophilic polymer as its primary structure, and the nucleic acid to be delivered, such as a natural nucleic acid, has anionic properties (“synthetic” in the figure). reference).
  • Such a structure has high degradation stability because the nucleic acid to be delivered is located inside the spherical structure.
  • the structure since the structure has a hydrophilic polymer segment in the outermost shell, it has excellent retention in blood. Then, the structure is wrapped in endosomes and taken up into the cytoplasm of the target site, such as a cell, and then escapes from the endosome and releases the target nucleic acid or low-molecular-weight drug, which is a drug, into the cytoplasm or nucleus (Fig. (see “Biochemical Evaluation” in Physiotherapy).
  • a nucleotide supported on a solid phase is reacted with a diisopropylamide phosphite compound and a nucleotide monomer ("1. Coupling” in the figure). Some hydroxyl groups are protected with a protecting group (“2. Capping”), and oxidized or borated with an oxidizing agent or a boronizing agent (“3. Oxidation or Boration”).
  • a cationic group is introduced by reacting an amino group compound by iodine oxidation.
  • a nucleic acid analog is synthesized according to the scheme shown in "Strategy 1: Cation introduction oligonucleic acid synthesis” in the figure.
  • phosphoramidite nucleotides synthesized according to the above scheme, phosphoramidite polyethylene glycol, and phosphoramidite disulfide having a disulfide bond and a phosphoramidite in the molecule are used as raw materials.
  • reactions such as protection with a protecting group and deprotection are performed to bind a hydrophilic polymer to the 5'-position of the cationic artificial nucleic acid.
  • cationic artificial nucleic acids may be synthesized in one step by solid-phase synthesis, as in the scheme shown in the figure "Strategy 2: Synthesis of cation-introduced oligonucleic acid".
  • the structure for nucleic acid delivery associates the nucleic acid to be delivered with the nucleic acid analogue to form a complex (association step). If there is a complementarity between the cationic group of the nucleic acid analog and the phosphate group of the nucleic acid to be delivered, the two are joined by annealing to form a duplex.
  • the ratio of the nucleic acid analogue to the nucleic acid to be delivered during annealing is not particularly limited, but the range of nucleic acid analogue to nucleic acid to be delivered is preferably 1:1 to 1:10.
  • FIG. 5 shows the results of evaluation of structural change due to pH response of the structure and stability of the structure in serum. From (A, B) in the figure, under the condition of pH 7, the positive peak at 260 nm, which is characteristic of the A-type helical structure observed in AN2, shifted to the longer wavelength side for both AN4 and AN5, and a strong negative peak at 210 nm. No drop in was observed. When the pH was changed to 5, AN4 showed no change, while AN5 decreased the intensity of the positive peak at 260 nm. The results of measuring RH at this time are shown in (C, D) of the figure. AN4 increased the RH from 35 nm to 85 nm.
  • Intracellular uptake of ligand RION miR-143 DDS in systemic administration includes “passive targeting” and “active targeting”.
  • passive targeting utilization of Enhanced Permeability and Retention effect (EPR effect), which is a characteristic of vascular endothelium around tumors, is known.
  • EPR effect Enhanced Permeability and Retention effect
  • Active targeting is expected to efficiently deliver nucleic acids to cancer with a smaller dose by specifically delivering only to target cells and tissues. Therefore, a ligand-introduced structure (AN30) was examined as active targeting.

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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PCT/JP2022/019343 2021-04-28 2022-04-28 人工核酸及びそれを用いた核酸の送達方法 Ceased WO2022230990A1 (ja)

Priority Applications (5)

Application Number Priority Date Filing Date Title
EP22795903.8A EP4331617A4 (en) 2021-04-28 2022-04-28 ARTIFICIAL NUCLEIC ACID AND METHOD OF ADMINISTRATING NUCLEIC ACID USING IT
US18/288,674 US20240238325A1 (en) 2021-04-28 2022-04-28 Artificial Nucleic Acid and Method for Delivery of Nucleic Acid Using the Same
AU2022266383A AU2022266383A1 (en) 2021-04-28 2022-04-28 Artificial nucleic acid and nucleic acid delivering method using same
CN202280031158.8A CN117295528A (zh) 2021-04-28 2022-04-28 人工核酸以及使用该人工核酸的核酸递送方法
JP2023517627A JPWO2022230990A1 (https=) 2021-04-28 2022-04-28

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JP2021-075651 2021-04-28
JP2021075651 2021-04-28
JP2022026023 2022-02-22
JP2022-026023 2022-02-22

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JP (1) JPWO2022230990A1 (https=)
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WO (1) WO2022230990A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024242065A1 (ja) 2023-05-19 2024-11-28 国立研究開発法人科学技術振興機構 核酸送達用構造体の造血器腫瘍の治療への使用
WO2025225509A1 (ja) * 2024-04-22 2025-10-30 国立研究開発法人科学技術振興機構 核酸送達用構造体、核酸送達用構造体の製造方法、及び医薬

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JPH10509740A (ja) * 1995-08-04 1998-09-22 カイロン コーポレイション カチオン性オリゴヌクレオチド、ならびに関連の合成および使用の方法
WO2005100447A1 (ja) 2004-04-16 2005-10-27 Japan Science And Technology Agency Ｐｅｇ−機能性核酸コンジュケート
US20060241071A1 (en) * 2003-06-16 2006-10-26 Grinstaff Mark W Functional synthetic molecules and macromolecules for gene delivery
WO2008062909A1 (en) 2006-11-22 2008-05-29 The University Of Tokyo ENVIRONMENT-RESPONDING siRNA CARRIER USING DISULFIDE-BRIDGED POLYMERIC MICELLE
JP2014522862A (ja) * 2011-07-19 2014-09-08 ウェイブ ライフ サイエンス プライベート リミテッド 官能化核酸の合成のための方法

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EP1689443B1 (en) * 2003-12-05 2019-03-13 Arrogene, Inc. Polymalic acid-based multifunctional drug delivery system
MX2009002859A (es) * 2006-09-15 2009-03-30 Enzon Pharmaceuticals Inc Enlazadores biodegradables a base de ester impedido para suministro de oligonucleotidos.
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JPH10509740A (ja) * 1995-08-04 1998-09-22 カイロン コーポレイション カチオン性オリゴヌクレオチド、ならびに関連の合成および使用の方法
US20060241071A1 (en) * 2003-06-16 2006-10-26 Grinstaff Mark W Functional synthetic molecules and macromolecules for gene delivery
WO2005100447A1 (ja) 2004-04-16 2005-10-27 Japan Science And Technology Agency Ｐｅｇ−機能性核酸コンジュケート
WO2008062909A1 (en) 2006-11-22 2008-05-29 The University Of Tokyo ENVIRONMENT-RESPONDING siRNA CARRIER USING DISULFIDE-BRIDGED POLYMERIC MICELLE
JP2014522862A (ja) * 2011-07-19 2014-09-08 ウェイブ ライフ サイエンス プライベート リミテッド 官能化核酸の合成のための方法

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SCOMPARIN ANNA, POLYAK DINA, KRIVITSKY ADVA, SATCHI-FAINARO RONIT: "Achieving successful delivery of oligonucleotides — From physico-chemical characterization to in vivo evaluation", BIOTECHNOLOGY ADVANCES, vol. 33, no. 6, 1 November 2015 (2015-11-01), GB , pages 1294 - 1309, XP055981359, ISSN: 0734-9750, DOI: 10.1016/j.biotechadv.2015.04.008 *
See also references of EP4331617A4
SUN XIAOLI, ZHANG NA: "Cationic Polymer Optimization for Efficient Gene Delivery", MINI-REVIEWS IN MEDICINAL CHEMISTRY, vol. 10, no. 2, 1 February 2010 (2010-02-01), NL , pages 108 - 125, XP055981348, ISSN: 1389-5575, DOI: 10.2174/138955710791185109 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024242065A1 (ja) 2023-05-19 2024-11-28 国立研究開発法人科学技術振興機構 核酸送達用構造体の造血器腫瘍の治療への使用
JPWO2024242065A1 (https=) * 2023-05-19 2024-11-28
JP7725042B2 (ja) 2023-05-19 2025-08-19 国立研究開発法人科学技術振興機構 造血器腫瘍治療用組成物、造血器腫瘍治療用医薬、及び造血器腫瘍における癌化細胞の増殖抑制用組成物
WO2025225509A1 (ja) * 2024-04-22 2025-10-30 国立研究開発法人科学技術振興機構 核酸送達用構造体、核酸送達用構造体の製造方法、及び医薬

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EP4331617A1 (en) 2024-03-06
EP4331617A4 (en) 2025-11-12
AU2022266383A1 (en) 2023-11-16
US20240238325A1 (en) 2024-07-18
JPWO2022230990A1 (https=) 2022-11-03

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