JP2024518546A - 修飾されたｍＲＮＡ、修飾された非コードＲＮＡ、およびその使用 - Google Patents

修飾されたｍＲＮＡ、修飾された非コードＲＮＡ、およびその使用 Download PDF

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JP2024518546A
JP2024518546A JP2023570150A JP2023570150A JP2024518546A JP 2024518546 A JP2024518546 A JP 2024518546A JP 2023570150 A JP2023570150 A JP 2023570150A JP 2023570150 A JP2023570150 A JP 2023570150A JP 2024518546 A JP2024518546 A JP 2024518546A
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modified
nucleotides
mrna
nucleotide
coding rna
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JP2024518546A5 (enExample
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ワン，シャオ
シー，ハイリン
アディサム，アブヒシェク
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Broad Institute Inc
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Broad Institute Inc
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    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/53—Ligases (6)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1003—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor
    • C12N15/1006—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers
    • C12N15/1013—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers by using magnetic beads
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y605/00—Ligases forming phosphoric ester bonds (6.5)
    • C12Y605/01—Ligases forming phosphoric ester bonds (6.5) forming phosphoric ester bonds (6.5.1)
    • C12Y605/01003—RNA ligase (ATP) (6.5.1.3)
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Analytical Chemistry (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023570150A 2021-05-12 2022-05-11 修飾されたｍＲＮＡ、修飾された非コードＲＮＡ、およびその使用 Pending JP2024518546A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163187752P 2021-05-12 2021-05-12
US63/187,752 2021-05-12
US202163288522P 2021-12-10 2021-12-10
US63/288,522 2021-12-10
PCT/US2022/028849 WO2022241045A1 (en) 2021-05-12 2022-05-11 Modified mrna, modified non-coding rna, and uses thereof

Publications (2)

Publication Number Publication Date
JP2024518546A true JP2024518546A (ja) 2024-05-01
JP2024518546A5 JP2024518546A5 (enExample) 2025-05-20

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Country Status (7)

Country Link
US (1) US20240277872A1 (enExample)
EP (1) EP4337331A1 (enExample)
JP (1) JP2024518546A (enExample)
KR (1) KR20240021170A (enExample)
AU (1) AU2022273530A1 (enExample)
CA (1) CA3218778A1 (enExample)
WO (1) WO2022241045A1 (enExample)

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WO2023141474A1 (en) * 2022-01-18 2023-07-27 The Broad Institute, Inc. Poly-tailed and poly-capped mrna and uses thereof
CN114685586B (zh) * 2022-03-05 2025-07-18 武汉瑞佶生物科技有限公司 一种mRNA-脂肪酸靶向化合物及其制备方法和应用
JP2025519910A (ja) * 2022-06-24 2025-06-26 ザ・ブロード・インスティテュート・インコーポレイテッド キャップされた環状ｒｎａ分子を調製するための組成物及び方法
EP4655398A1 (en) * 2023-01-27 2025-12-03 Eleven Therapeutics Ltd Artificial polynucleotide molecules for enhanced stability and translation
KR20250166303A (ko) * 2023-03-29 2025-11-27 센젠 센신 바이오테크놀로지 컴퍼니 리미티드 폴리(a)를 포함하는 폴리뉴클레오타이드 분자 및 이의 용도
WO2025017029A1 (en) * 2023-07-17 2025-01-23 Eleven Therapeutics Ltd Non-natural building blocks for enhanced translation and stability of mrna
JP2025032058A (ja) * 2023-08-06 2025-03-11 シー‐ラン リン 新規なオキソ－ｒｎａ組成物及びその関連用途
WO2025111430A1 (en) * 2023-11-21 2025-05-30 Prime Medicine, Inc. CHEMICAL MODIFICATIONS IN mRNA POLY(A) TAIL
WO2025147660A2 (en) * 2024-01-04 2025-07-10 Trilink Biotechnologies, Llc Modified rna for increasing protein expression
WO2025175176A1 (en) * 2024-02-14 2025-08-21 Modernatx, Inc. Synthetic nucleic acids and methods of using the same for improved polypeptide production
WO2025217126A1 (en) * 2024-04-09 2025-10-16 Convergence Bio, Inc. Polynucleotides, 3' chain terminating tails, ligation and uses thereof
WO2025217135A1 (en) * 2024-04-09 2025-10-16 Convergence Bio, Inc. Rna ligation methods and products
WO2025227104A1 (en) * 2024-04-25 2025-10-30 Parcel Biosciences, Inc. Mrna complexes, their manufacturing, and their use for treatment
WO2026010855A1 (en) * 2024-07-02 2026-01-08 Trilink Biotechnologies, Llc Modified rna for increasing protein expression
WO2026011417A1 (en) * 2024-07-12 2026-01-15 The Hong Kong University Of Science And Technology Compositions and methods involving artificial poly (a) sequences

Citations (4)

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WO2013103659A1 (en) * 2012-01-04 2013-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Stabilizing rna by incorporating chain-terminating nucleosides at the 3'-terminus
WO2015048744A2 (en) * 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
JP2017537613A (ja) * 2014-11-02 2017-12-21 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． メッセンジャーｕｎａ分子およびその使用
WO2020061397A1 (en) * 2018-09-20 2020-03-26 Modernatx, Inc. Compositions and methods for delivery of nucleic acids

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CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
US9950065B2 (en) 2013-09-26 2018-04-24 Biontech Rna Pharmaceuticals Gmbh Particles comprising a shell with RNA
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
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Publication number Priority date Publication date Assignee Title
WO2013103659A1 (en) * 2012-01-04 2013-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Stabilizing rna by incorporating chain-terminating nucleosides at the 3'-terminus
WO2015048744A2 (en) * 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
JP2017537613A (ja) * 2014-11-02 2017-12-21 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． メッセンジャーｕｎａ分子およびその使用
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Publication number Publication date
KR20240021170A (ko) 2024-02-16
CA3218778A1 (en) 2022-11-17
AU2022273530A9 (en) 2023-11-30
EP4337331A1 (en) 2024-03-20
AU2022273530A2 (en) 2023-12-07
WO2022241045A1 (en) 2022-11-17
US20240277872A1 (en) 2024-08-22
AU2022273530A1 (en) 2023-11-23

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