KR20240021170A - 변형된 mRNA, 변형된 비-코딩 RNA, 및 그의 용도 - Google Patents

변형된 mRNA, 변형된 비-코딩 RNA, 및 그의 용도 Download PDF

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KR20240021170A
KR20240021170A KR1020237042287A KR20237042287A KR20240021170A KR 20240021170 A KR20240021170 A KR 20240021170A KR 1020237042287 A KR1020237042287 A KR 1020237042287A KR 20237042287 A KR20237042287 A KR 20237042287A KR 20240021170 A KR20240021170 A KR 20240021170A
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modified
nucleotides
mrna
coding rna
sequence
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Korean (ko)
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샤오 왕
하이링 스
아브히쉑 아디트함
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더 브로드 인스티튜트, 인코퍼레이티드
메사추세츠 인스티튜트 오브 테크놀로지
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Publication of KR20240021170A publication Critical patent/KR20240021170A/ko
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    • 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
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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • 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
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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
    • C12N15/67—General methods for enhancing the expression
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    • 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)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • 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
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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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  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • 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)
KR1020237042287A 2021-05-12 2022-05-11 변형된 mRNA, 변형된 비-코딩 RNA, 및 그의 용도 Pending KR20240021170A (ko)

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

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KR20240021170A true KR20240021170A (ko) 2024-02-16

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US (1) US20240277872A1 (https=)
EP (1) EP4337331A1 (https=)
JP (1) JP2024518546A (https=)
KR (1) KR20240021170A (https=)
AU (1) AU2022273530A1 (https=)
CA (1) CA3218778A1 (https=)
WO (1) WO2022241045A1 (https=)

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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

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CA3218778A1 (en) 2022-11-17
AU2022273530A9 (en) 2023-11-30
EP4337331A1 (en) 2024-03-20
JP2024518546A (ja) 2024-05-01
AU2022273530A2 (en) 2023-12-07
WO2022241045A1 (en) 2022-11-17
US20240277872A1 (en) 2024-08-22
AU2022273530A1 (en) 2023-11-23

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