JP2024533313A - Ｈｂｂを調節する組成物及び方法 - Google Patents

Ｈｂｂを調節する組成物及び方法 Download PDF

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JP2024533313A
JP2024533313A JP2024515069A JP2024515069A JP2024533313A JP 2024533313 A JP2024533313 A JP 2024533313A JP 2024515069 A JP2024515069 A JP 2024515069A JP 2024515069 A JP2024515069 A JP 2024515069A JP 2024533313 A JP2024533313 A JP 2024533313A
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sequence
domain
template
grna
template rna
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JP2024533313A5 (https=
JPWO2023039440A5 (https=
Inventor
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，
ヤンファン フー，
ダニエル ジーン アバナシー，
マイケル クリストファー ホームズ，
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 シックス，エルエルシー
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Publication of JP2024533313A publication Critical patent/JP2024533313A/ja
Publication of JP2024533313A5 publication Critical patent/JP2024533313A5/ja
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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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    • C12N15/09—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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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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
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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
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
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  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2024515069A 2021-09-08 2022-09-07 Ｈｂｂを調節する組成物及び方法 Pending JP2024533313A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163241994P 2021-09-08 2021-09-08
US63/241,994 2021-09-08
US202163250143P 2021-09-29 2021-09-29
US63/250,143 2021-09-29
US202263303900P 2022-01-27 2022-01-27
US63/303,900 2022-01-27
PCT/US2022/076063 WO2023039440A2 (en) 2021-09-08 2022-09-07 Hbb-modulating compositions and methods

Publications (3)

Publication Number Publication Date
JP2024533313A true JP2024533313A (ja) 2024-09-12
JP2024533313A5 JP2024533313A5 (https=) 2025-09-17
JPWO2023039440A5 JPWO2023039440A5 (https=) 2025-09-17

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JP2024515069A Pending JP2024533313A (ja) 2021-09-08 2022-09-07 Ｈｂｂを調節する組成物及び方法

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US (1) US20240252682A1 (https=)
EP (1) EP4399307A4 (https=)
JP (1) JP2024533313A (https=)
AU (1) AU2022342169A1 (https=)
CA (1) CA3231679A1 (https=)
TW (1) TW202323524A (https=)
WO (1) WO2023039440A2 (https=)

Families Citing this family (11)

* 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
CN113286880A (zh) 2018-08-28 2021-08-20 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
CA3174483A1 (en) 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Improved methods and compositions for modulating a genome
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
KR20240099164A (ko) 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
EP4684023A2 (en) * 2023-03-21 2026-01-28 Tessera Therapeutics, Inc. Modified template guide rna molecules
WO2024206125A1 (en) * 2023-03-24 2024-10-03 The Broad Institute, Inc. Use of prime editing for treating sickle cell disease
WO2025059607A1 (en) * 2023-09-13 2025-03-20 Tessera Therapeutics, Inc. Lipid nanoparticles for delivery of therapeutic payloads to cells
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3445388T3 (da) * 2016-04-18 2024-06-03 Vertex Pharma Materialer og fremgangsmåder til behandling af hæmoglobinopatier
BR112019000057A2 (pt) * 2016-07-05 2019-04-02 The Johns Hopkins University composições e métodos com base em crispr/cas9 para o tratamento de degeneração de retina
JP7657726B2 (ja) * 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
CN114667149A (zh) * 2019-09-09 2022-06-24 比姆医疗股份有限公司 新型核碱基编辑器及其使用方法
US12435330B2 (en) * 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA

Also Published As

Publication number Publication date
WO2023039440A3 (en) 2023-05-19
US20240252682A1 (en) 2024-08-01
WO2023039440A9 (en) 2023-07-06
WO2023039440A2 (en) 2023-03-16
TW202323524A (zh) 2023-06-16
EP4399307A4 (en) 2026-01-21
EP4399307A2 (en) 2024-07-17
AU2022342169A1 (en) 2024-03-28
CA3231679A1 (en) 2023-03-16

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