KR102778328B1 - 유전체 편집 효율 증가를 위한 화합물 - Google Patents

유전체 편집 효율 증가를 위한 화합물 Download PDF

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KR102778328B1
KR102778328B1 KR1020237017487A KR20237017487A KR102778328B1 KR 102778328 B1 KR102778328 B1 KR 102778328B1 KR 1020237017487 A KR1020237017487 A KR 1020237017487A KR 20237017487 A KR20237017487 A KR 20237017487A KR 102778328 B1 KR102778328 B1 KR 102778328B1
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dna
compound
target cell
cell
cells
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KR20230084312A (ko
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스테판 리센버그
토미스라브 마리칙
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막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우.
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Priority claimed from EP17203591.7A external-priority patent/EP3489357A1/en
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    • 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
    • A61K31/16—Amides, e.g. hydroxamic acids
    • A61K31/165—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
    • 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
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/194—Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
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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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    • 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
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
    • 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
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Pyrane Compounds (AREA)
  • Management, Administration, Business Operations System, And Electronic Commerce (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
KR1020237017487A 2017-04-10 2018-04-10 유전체 편집 효율 증가를 위한 화합물 Active KR102778328B1 (ko)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
EP17165784.4A EP3388517A1 (en) 2017-04-10 2017-04-10 Compounds for increasing genome editing efficiency
EP17165784.4 2017-04-10
EP17203591.7 2017-11-24
EP17203591.7A EP3489357A1 (en) 2017-11-24 2017-11-24 Compounds for increasing genome editing efficiency
PCT/EP2018/059173 WO2018189186A1 (en) 2017-04-10 2018-04-10 Compounds for increasing genome editing efficiency
KR1020197032518A KR102537447B1 (ko) 2017-04-10 2018-04-10 유전체 편집 효율 증가를 위한 화합물

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KR20230084312A KR20230084312A (ko) 2023-06-12
KR102778328B1 true KR102778328B1 (ko) 2025-03-11

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KR1020197032518A Active KR102537447B1 (ko) 2017-04-10 2018-04-10 유전체 편집 효율 증가를 위한 화합물

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US (1) US12173309B2 (enExample)
EP (1) EP3610008A1 (enExample)
JP (2) JP7319194B2 (enExample)
KR (2) KR102778328B1 (enExample)
CN (1) CN110691848B (enExample)
CA (1) CA3058697A1 (enExample)
IL (1) IL269887B2 (enExample)
SG (1) SG11201909432SA (enExample)
WO (1) WO2018189186A1 (enExample)

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WO2019051316A1 (en) * 2017-09-08 2019-03-14 Life Technologies Corporation ENHANCED APPROVAL RECOMBINATION METHODS AND COMPOSITIONS THEREOF
JP7235153B2 (ja) * 2017-12-29 2023-03-08 株式会社三洋物産 遊技機
JP7483612B2 (ja) 2018-01-17 2024-05-15 バーテックス ファーマシューティカルズ インコーポレイテッド ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット
CN111741955B (zh) * 2018-01-17 2024-02-23 沃泰克斯药物股份有限公司 Dna-pk抑制剂
JP7235154B2 (ja) * 2018-02-15 2023-03-08 株式会社三洋物産 遊技機
JP7231076B2 (ja) * 2018-03-08 2023-03-01 株式会社三洋物産 遊技機
EP3911330B1 (en) 2018-12-21 2025-03-05 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Dna-pkcs inhibitors for increasing genome editing efficiency
JP2020103418A (ja) * 2018-12-26 2020-07-09 株式会社三洋物産 遊技機
JP2020130466A (ja) * 2019-02-15 2020-08-31 株式会社三洋物産 遊技機
JP7234740B2 (ja) * 2019-03-28 2023-03-08 株式会社三洋物産 遊技機
JP7234741B2 (ja) * 2019-03-28 2023-03-08 株式会社三洋物産 遊技機
JP7234761B2 (ja) * 2019-04-11 2023-03-08 株式会社三洋物産 遊技機
JP7234760B2 (ja) * 2019-04-11 2023-03-08 株式会社三洋物産 遊技機
JP2021186294A (ja) * 2020-05-29 2021-12-13 株式会社三洋物産 遊技機
CN112359065B (zh) * 2020-09-21 2023-04-07 中国医学科学院血液病医院(中国医学科学院血液学研究所) 一种提高基因敲入效率的小分子组合物
KR102545913B1 (ko) * 2021-02-02 2023-06-22 재단법인 아산사회복지재단 히스톤 탈아세틸화 억제제를 포함하는 염기교정 기술의 효율성 및 정확도 향상용 조성물 및 이의 용도
EP4347815A2 (en) * 2021-05-24 2024-04-10 New York Genome Center, Inc. Compositions and methods for increasing efficiency of precise editing repair
CN113403309B (zh) * 2021-05-24 2022-08-16 珠海舒桐医疗科技有限公司 非同源双链寡聚核苷酸片段在基因敲除系统中的应用
EP4095246A1 (en) 2021-05-27 2022-11-30 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Novel crispr grnas
US20230070047A1 (en) * 2021-07-06 2023-03-09 Regents Of The University Of Minnesota Method to enhance gene editing
JP2023105110A (ja) * 2021-10-27 2023-07-28 株式会社三洋物産 遊技機
JP2023105108A (ja) * 2021-10-27 2023-07-28 株式会社三洋物産 遊技機
JP2023105111A (ja) * 2021-10-27 2023-07-28 株式会社三洋物産 遊技機
JP2023063369A (ja) * 2022-01-07 2023-05-09 株式会社三洋物産 遊技機
JP2023053387A (ja) * 2022-02-04 2023-04-12 株式会社三洋物産 遊技機
JP2023060269A (ja) * 2022-04-01 2023-04-27 株式会社三洋物産 遊技機
JP2023060270A (ja) * 2022-04-01 2023-04-27 株式会社三洋物産 遊技機
CN114717207B (zh) * 2022-04-25 2023-03-07 苏州泓迅生物科技股份有限公司 一种酵母细胞同源重组酶系、dna体外组装试剂及其应用
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KR20190139239A (ko) 2019-12-17
JP2020516258A (ja) 2020-06-11
US20200392540A1 (en) 2020-12-17
JP2023093479A (ja) 2023-07-04
SG11201909432SA (en) 2019-11-28
CN110691848A (zh) 2020-01-14
CN110691848B (zh) 2024-04-02
WO2018189186A1 (en) 2018-10-18
IL269887B1 (en) 2024-09-01
US12173309B2 (en) 2024-12-24
IL269887A (en) 2019-11-28
IL269887B2 (en) 2025-01-01
CA3058697A1 (en) 2018-10-18
KR20230084312A (ko) 2023-06-12
EP3610008A1 (en) 2020-02-19
JP7319194B2 (ja) 2023-08-01
KR102537447B1 (ko) 2023-05-30

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