CN110249052B - 合成的指导分子、组合物和与其相关的方法 - Google Patents

合成的指导分子、组合物和与其相关的方法 Download PDF

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CN110249052B
CN110249052B CN201780085248.4A CN201780085248A CN110249052B CN 110249052 B CN110249052 B CN 110249052B CN 201780085248 A CN201780085248 A CN 201780085248A CN 110249052 B CN110249052 B CN 110249052B
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guide molecule
guide
molecule
integer
inclusive
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CN110249052A (zh
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J·海尔
S·金
S·萨科曼诺
S·凯普哈特
C·费尔南德斯
H·嘉亚拉穆
B·伊顿
K·Z·贝里
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Editas Medicine Inc
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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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/319—Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
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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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    • C12N2310/50—Physical structure
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    • C12N2320/00—Applications; Uses
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
CN201780085248.4A 2016-12-30 2017-12-29 合成的指导分子、组合物和与其相关的方法 Active CN110249052B (zh)

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US201662441046P 2016-12-30 2016-12-30
US62/441,046 2016-12-30
US201762492001P 2017-04-28 2017-04-28
US62/492,001 2017-04-28
PCT/US2017/069019 WO2018126176A1 (en) 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto

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EP (1) EP3565895A1 (enExample)
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KR (2) KR102758180B1 (enExample)
CN (2) CN118345072A (enExample)
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CA (1) CA3048434A1 (enExample)
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JP2025016776A (ja) 2025-02-04
CN110249052A (zh) 2019-09-17
US20230111575A1 (en) 2023-04-13
KR102618864B1 (ko) 2024-01-02
JP2022173349A (ja) 2022-11-18
JP7167029B2 (ja) 2022-11-08
MX420023B (es) 2025-01-14
AU2017388753A1 (en) 2019-07-11
AU2024205204A1 (en) 2024-09-05
WO2018126176A1 (en) 2018-07-05
MX2019007750A (es) 2019-10-15
CN118345072A (zh) 2024-07-16
CA3048434A1 (en) 2018-07-05
KR20190110554A (ko) 2019-09-30
KR20230175330A (ko) 2023-12-29
JP2020503049A (ja) 2020-01-30
EP3565895A1 (en) 2019-11-13
KR102758180B1 (ko) 2025-01-23
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