JP2023543218A - 遺伝子発現を阻害するための組成物及び方法 - Google Patents

遺伝子発現を阻害するための組成物及び方法 Download PDF

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JP2023543218A
JP2023543218A JP2023518775A JP2023518775A JP2023543218A JP 2023543218 A JP2023543218 A JP 2023543218A JP 2023518775 A JP2023518775 A JP 2023518775A JP 2023518775 A JP2023518775 A JP 2023518775A JP 2023543218 A JP2023543218 A JP 2023543218A
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expression
repressor
dna
sequence
suppression system
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JP2023543218A5 (https=
Inventor
ジョディ ミシェル ケネディ，
ジェレミア デイル ファレリ，
Original Assignee
フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド
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Publication of JP2023543218A publication Critical patent/JP2023543218A/ja
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    • 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
    • C12N15/11—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
    • 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]
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • C12N15/635—Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
    • C12Y201/01043—Histone-lysine N-methyltransferase (2.1.1.43)
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023518775A 2020-09-24 2021-09-24 遺伝子発現を阻害するための組成物及び方法 Pending JP2023543218A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063082555P 2020-09-24 2020-09-24
US63/082,555 2020-09-24
US202163242957P 2021-09-10 2021-09-10
US63/242,957 2021-09-10
PCT/US2021/051945 WO2022067033A1 (en) 2020-09-24 2021-09-24 Compositions and methods for inhibiting gene expression

Publications (2)

Publication Number Publication Date
JP2023543218A true JP2023543218A (ja) 2023-10-13
JP2023543218A5 JP2023543218A5 (https=) 2024-10-02

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JP2023518775A Pending JP2023543218A (ja) 2020-09-24 2021-09-24 遺伝子発現を阻害するための組成物及び方法

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US (1) US20230399640A1 (https=)
EP (1) EP4217477A4 (https=)
JP (1) JP2023543218A (https=)
KR (1) KR20230074525A (https=)
AU (1) AU2021350024A1 (https=)
MX (1) MX2023003424A (https=)
WO (1) WO2022067033A1 (https=)

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US20170151281A1 (en) 2015-02-19 2017-06-01 Batu Biologics, Inc. Chimeric antigen receptor dendritic cell (car-dc) for treatment of cancer
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
US10980836B1 (en) 2019-12-11 2021-04-20 Myeloid Therapeutics, Inc. Therapeutic cell compositions and methods of manufacturing and use thereof
EP4240367A4 (en) 2020-11-04 2024-10-16 Myeloid Therapeutics, Inc. MODIFIED CHIMERIC FUSION PROTEIN COMPOSITIONS AND METHODS OF USE THEREOF
WO2022197949A2 (en) 2021-03-17 2022-09-22 Myeloid Therapeutics, Inc. Engineered chimeric fusion protein compositions and methods of use thereof
BR112023023642A2 (pt) 2021-05-11 2024-01-30 Myeloid Therapeutics Inc Métodos e composições para integração genômica
WO2023057880A1 (en) * 2021-10-05 2023-04-13 Glaxosmithkline Intellectual Property Development Limited Crispr/cas9-based fusion proteins for modulating gene expression and methods of use
KR20250077608A (ko) * 2022-08-19 2025-05-30 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통한 b형 간염 바이러스 조절용 조성물, 시스템 및 방법
KR20250128349A (ko) * 2022-12-23 2025-08-27 에피제닉 테라퓨틱스 피티이 리미티드 융합체 및 이의 용도
EP4713027A1 (en) 2023-05-16 2026-03-25 Omega Therapeutics, Inc. Methods and compositions for modulating methylation of a target gene
WO2024238723A1 (en) 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
WO2025019742A1 (en) 2023-07-19 2025-01-23 Omega Therapeutics, Inc. Methods and compositions for modulating ctnnb1 expression
WO2025064469A1 (en) * 2023-09-18 2025-03-27 Omega Therapeutics, Inc. Methods for assessing dosage for epigenetic modifying agents
WO2025171047A1 (en) * 2024-02-05 2025-08-14 Bioventures, Llc Leveraging cancer-evolved resistance mechanisms to enhance ezh2 activity in adoptive t cells
WO2025261475A1 (zh) * 2024-06-21 2025-12-26 益杰立科(上海)生物科技有限公司 靶向血管内皮生长因子的工程化基因转录阻抑工具及用途

Citations (1)

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JP2017537611A (ja) * 2014-10-24 2017-12-21 オスペダーレ サン ラファエレ エス．アール．エル 持続的エピジェネティック遺伝子サイレンシング

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AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2017031370A1 (en) * 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US9896696B2 (en) * 2016-02-15 2018-02-20 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
US20220380760A1 (en) * 2018-10-15 2022-12-01 Flagship Pioneering Innovations V, Inc. Disrupting genomic complex assembly in fusion genes
US20220249625A1 (en) * 2019-01-29 2022-08-11 Flagship Pioneering Innovations V, Inc. Compositions comprising an endonuclease and methods for purifying an endonuclease

Patent Citations (1)

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Non-Patent Citations (1)

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Also Published As

Publication number Publication date
EP4217477A4 (en) 2024-10-16
AU2021350024A9 (en) 2023-07-13
US20230399640A1 (en) 2023-12-14
WO2022067033A1 (en) 2022-03-31
EP4217477A1 (en) 2023-08-02
KR20230074525A (ko) 2023-05-30
AU2021350024A1 (en) 2023-05-04
MX2023003424A (es) 2023-05-03

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