JP2022507269A - カプセル化ポリヌクレオチド及び使用方法 - Google Patents

カプセル化ポリヌクレオチド及び使用方法 Download PDF

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JP2022507269A
JP2022507269A JP2021525757A JP2021525757A JP2022507269A JP 2022507269 A JP2022507269 A JP 2022507269A JP 2021525757 A JP2021525757 A JP 2021525757A JP 2021525757 A JP2021525757 A JP 2021525757A JP 2022507269 A JP2022507269 A JP 2022507269A
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sequence
mir
target sequence
lnp
recombinant dna
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JPWO2020102285A5 (https=
JP2022507269A5 (https=
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ミッチェル エイチ． ファイナー，
エドワード ケネディ，
ロレーナ ラーナー，
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オンコラス， インコーポレイテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/768—Oncolytic viruses not provided for in groups A61K35/761 - A61K35/766
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/5107—Excipients; Inactive ingredients
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
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    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2809—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against the T-cell receptor (TcR)-CD3 complex
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    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32034—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2770/00011—Details
    • C12N2770/32011—Picornaviridae
    • C12N2770/32041—Use of virus, viral particle or viral elements as a vector
    • C12N2770/32043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07006—DNA-directed RNA polymerase (2.7.7.6)

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Dispersion Chemistry (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2021525757A 2018-11-13 2019-11-13 カプセル化ポリヌクレオチド及び使用方法 Pending JP2022507269A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862760422P 2018-11-13 2018-11-13
US62/760,422 2018-11-13
PCT/US2019/061093 WO2020102285A1 (en) 2018-11-13 2019-11-13 Encapsulated polynucleotides and methods of use

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JP2022507269A true JP2022507269A (ja) 2022-01-18
JPWO2020102285A5 JPWO2020102285A5 (https=) 2022-11-07
JP2022507269A5 JP2022507269A5 (https=) 2022-11-07

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US (1) US20210403950A1 (https=)
EP (1) EP3880812A4 (https=)
JP (1) JP2022507269A (https=)
KR (1) KR20210093285A (https=)
CN (1) CN113348246A (https=)
AU (1) AU2019381698A1 (https=)
BR (1) BR112021009226A2 (https=)
CA (1) CA3117924A1 (https=)
IL (1) IL282992A (https=)
MX (1) MX2021005448A (https=)
SG (1) SG11202104887UA (https=)
WO (1) WO2020102285A1 (https=)

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US11130787B2 (en) * 2020-06-11 2021-09-28 MBF Therapeutics, Inc. Alphaherpesvirus glycoprotein d-encoding nucleic acid constructs and methods
MX2023009345A (es) * 2021-02-10 2023-10-24 Oncorus Inc Compuestos, composiciones y metodos de uso de estos.
IT202200016386A1 (it) * 2022-08-02 2024-02-02 Univ Degli Studi Roma La Sapienza Nanoparticelle lipidiche multicomponenti ad elevata fusogenicità cellulare per la veicolazione di acidi nucleici e relativo processo di preparazione
EP4658782A2 (en) * 2023-02-02 2025-12-10 The Regents of the University of California Self-amplifying non-coding rna for targeted gene knockdown
EP4683655A1 (en) * 2023-03-20 2026-01-28 Dimberg, Anna Tumor blood vessel-targeted aav therapy for cancer treatment
CN117487762A (zh) * 2023-11-06 2024-02-02 中科佰乘(深圳)生物科技有限公司 一种细胞外囊泡、制备方法、药物组合物及应用
CN119242712A (zh) * 2024-09-27 2025-01-03 南京师范大学 特异性靶向骨骼肌细胞的携带miRNA-486基因的腺相关病毒载体、药物组合物

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SG11202104887UA (en) 2021-06-29
EP3880812A1 (en) 2021-09-22
CN113348246A (zh) 2021-09-03
KR20210093285A (ko) 2021-07-27
EP3880812A4 (en) 2022-09-07
AU2019381698A1 (en) 2021-05-27
IL282992A (en) 2021-06-30
BR112021009226A2 (pt) 2021-10-26
WO2020102285A1 (en) 2020-05-22
CA3117924A1 (en) 2020-05-22
MX2021005448A (es) 2021-08-11
US20210403950A1 (en) 2021-12-30

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