JP2024533312A - Ｓｅｒｐｉｎａ調節組成物及び方法 - Google Patents

Ｓｅｒｐｉｎａ調節組成物及び方法 Download PDF

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JP2024533312A
JP2024533312A JP2024515068A JP2024515068A JP2024533312A JP 2024533312 A JP2024533312 A JP 2024533312A JP 2024515068 A JP2024515068 A JP 2024515068A JP 2024515068 A JP2024515068 A JP 2024515068A JP 2024533312 A JP2024533312 A JP 2024533312A
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sequence
domain
modification system
template rna
grna
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JP2024533312A5 (https=
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ロバート チャールズ アルトシュラー，
アン ヘレン ボスマー，
ダニエル レイモンド チー，
セシリア ジョバンナ シルビア コッタ－ラムシノ，
キュシク キム，
ランディ ミシェル コトラー，
グレゴリー デイビッド マカリスター，
アナーニャ レイ，
ナサニエル ロケ，
カルロス サンチェス，
バレット イーサン スタインバーグ，
ウィリアム エドワード サロモン，
ロバート ジェイムズ シトリク，
ウィリアム クルベス，
ルチアーノ エンリケ アポーニ，
ジャン ワン，
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フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー
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Publication of JP2024533312A publication Critical patent/JP2024533312A/ja
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    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • C07K14/8125—Alpha-1-antitrypsin
    • 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
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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
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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
    • 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.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • 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
    • 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)
  • Genetics & Genomics (AREA)
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  • Wood Science & Technology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
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  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024515068A 2021-09-08 2022-09-07 Ｓｅｒｐｉｎａ調節組成物及び方法 Pending JP2024533312A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163241970P 2021-09-08 2021-09-08
US63/241,970 2021-09-08
US202163253087P 2021-10-06 2021-10-06
US63/253,087 2021-10-06
US202263303905P 2022-01-27 2022-01-27
US63/303,905 2022-01-27
PCT/US2022/076073 WO2023039447A2 (en) 2021-09-08 2022-09-07 Serpina-modulating compositions and methods

Publications (2)

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JP2024533312A true JP2024533312A (ja) 2024-09-12
JP2024533312A5 JP2024533312A5 (https=) 2025-09-17

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JP2024515068A Pending JP2024533312A (ja) 2021-09-08 2022-09-07 Ｓｅｒｐｉｎａ調節組成物及び方法

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US (1) US20240084334A1 (https=)
EP (1) EP4399309A4 (https=)
JP (1) JP2024533312A (https=)
AU (1) AU2022344251A1 (https=)
CA (1) CA3231594A1 (https=)
MX (1) MX2024002988A (https=)
WO (1) WO2023039447A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
WO2025155772A1 (en) * 2024-01-19 2025-07-24 Gene Company (Pty)Ltd. Alpha-1 anti-trypsin upregulating polynucleotides and method of use and treatment thereof
US20250269059A1 (en) * 2024-02-27 2025-08-28 Crispr Therapeutics Ag Rt editing compositions and methods
WO2025194124A1 (en) * 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. Modified st1cas9 guide nucleic acids
US20250354138A1 (en) * 2024-03-15 2025-11-20 Beam Therapeutics Inc. Prime editing of single base mutations in alpha-1 antitrypsin deficiency

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9458457B2 (en) * 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
IL308824A (en) * 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof
CA3035534A1 (en) * 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214609A1 (en) * 2019-04-14 2020-10-22 Duke University Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
US12435330B2 (en) * 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA

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Publication number Publication date
MX2024002988A (es) 2024-06-11
EP4399309A2 (en) 2024-07-17
CA3231594A1 (en) 2023-03-16
AU2022344251A1 (en) 2024-03-28
WO2023039447A3 (en) 2023-06-01
WO2023039447A9 (en) 2023-12-21
WO2023039447A2 (en) 2023-03-16
EP4399309A4 (en) 2026-02-11
US20240084334A1 (en) 2024-03-14

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