KR20240099164A - Pah-조절 조성물 및 방법 - Google Patents

Pah-조절 조성물 및 방법

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Publication number
KR20240099164A
KR20240099164A KR1020247011690A KR20247011690A KR20240099164A KR 20240099164 A KR20240099164 A KR 20240099164A KR 1020247011690 A KR1020247011690 A KR 1020247011690A KR 20247011690 A KR20247011690 A KR 20247011690A KR 20240099164 A KR20240099164 A KR 20240099164A
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KR
South Korea
Prior art keywords
sequence
domain
grna
template rna
genetic modification
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020247011690A
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English (en)
Korean (ko)
Inventor
로버트 찰스 알트슐러
앤 헬렌 보트머
다니엘 레이먼드 치
세실리아 지오반나 실비아 코타-라무시노
규식 김
랜디 미쉘 코틀라
그레고리 데이비드 맥앨리스터
아나냐 레이
나타니엘 로케트
카를로스 산체스
배럿 에단 스타인버그
윌리엄 에드워드 살로몬
로버트 제임스 시토리크
윌리엄 쿼르베스
루치아노 헨리케 아포니
잔 왕
Original Assignee
플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 filed Critical 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨
Publication of KR20240099164A publication Critical patent/KR20240099164A/ko
Pending legal-status Critical Current

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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
    • C12N15/1137—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 against enzymes
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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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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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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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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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    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C12N2310/00—Structure or type of the nucleic acid
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  • Health & Medical Sciences (AREA)
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  • Virology (AREA)
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  • Crystallography & Structural Chemistry (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
KR1020247011690A 2021-09-08 2022-09-07 Pah-조절 조성물 및 방법 Pending KR20240099164A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163241897P 2021-09-08 2021-09-08
US63/241,897 2021-09-08
US202263303927P 2022-01-27 2022-01-27
US63/303,927 2022-01-27
US202263367025P 2022-06-24 2022-06-24
US63/367,025 2022-06-24
PCT/US2022/076058 WO2023039435A2 (en) 2021-09-08 2022-09-07 Pah-modulating compositions and methods

Publications (1)

Publication Number Publication Date
KR20240099164A true KR20240099164A (ko) 2024-06-28

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KR1020247011690A Pending KR20240099164A (ko) 2021-09-08 2022-09-07 Pah-조절 조성물 및 방법

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Country Link
US (1) US12544458B2 (https=)
EP (1) EP4399306A4 (https=)
JP (1) JP2024533315A (https=)
KR (1) KR20240099164A (https=)
AU (1) AU2022342168A1 (https=)
CA (1) CA3231712A1 (https=)
IL (1) IL311218A (https=)
TW (1) TW202320810A (https=)
WO (1) WO2023039435A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
WO2024197103A2 (en) * 2023-03-21 2024-09-26 Tessera Therapeutics, Inc. Modified template guide rna molecules
US20250059567A1 (en) * 2023-08-17 2025-02-20 The Trustees Of The University Of Pennsylvania Compositions and methods for the management and treatment of phenylketonuria

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11120889B2 (en) * 2012-05-09 2021-09-14 Georgia Tech Research Corporation Method for synthesizing a nuclease with reduced off-site cleavage
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP3485032B1 (en) * 2016-07-12 2021-02-17 Life Technologies Corporation Compositions and methods for detecting nucleic acid regions
CA3073448A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
CN107723358B (zh) * 2017-10-26 2021-03-19 国家卫生健康委科学技术研究所 苯丙酮尿症检测试剂盒
CN112533627A (zh) * 2018-03-27 2021-03-19 G+Flas 生命科学公司 序列特异性体内细胞靶向
CA3105925A1 (en) * 2018-07-10 2020-01-16 Alia Therapeutics S.R.L. Vesicles for traceless delivery of guide rna molecules and/or guide rna molecule/rna-guided nuclease complex(es) and a production method thereof
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
CN113272428A (zh) * 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
JP7759311B2 (ja) 2019-07-19 2025-10-23 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 リコンビナーゼ組成物及び使用方法
EP4034659A4 (en) 2019-09-27 2024-10-16 The Broad Institute, Inc. PROGRAMMABLE POLYNUCLEOTIDE EDITORS FOR AMPLIFIED HOMOLOGOUS RECOMBINATION
CA3162499A1 (en) 2019-11-22 2021-05-27 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
EP4085141A4 (en) 2019-12-30 2024-03-06 The Broad Institute, Inc. GENOME EDITING USING REVERSE TRANSCRIPTASE FOR FULLY ACTIVE CRISPR COMPLEXES
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
US20230348939A1 (en) 2020-03-04 2023-11-02 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
BR112022017713A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
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
CA3174553A1 (en) 2020-03-05 2021-09-10 Flagship Pioneering Innovations Vi, Llc Host defense suppressing methods and compositions for modulating a genome
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
TW202208632A (zh) * 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2021248102A1 (en) 2020-06-05 2021-12-09 Flagship Pioneering Innovations Vi, Llc Template guide rna molecules
EP4274894A2 (en) 2021-01-11 2023-11-15 The Broad Institute, Inc. Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision
US20240132916A1 (en) 2021-02-09 2024-04-25 The Broad Institute, Inc. Nuclease-guided non-ltr retrotransposons and uses thereof
WO2022183210A1 (en) 2021-02-26 2022-09-01 Flagship Pioneering Innovations Vi, Llc Tissue-specific methods and compositions for modulating a genome
AU2022240747A1 (en) 2021-03-19 2023-10-05 Flagship Pioneering Innovations Vi, Llc Ltr transposon compositions and methods
EP4314257A4 (en) 2021-04-01 2025-07-09 Prime Medicine Inc METHODS AND COMPOSITIONS FOR EDITING NUCLEOTIDE SEQUENCES
EP4347859A4 (en) 2021-05-26 2025-07-02 Flagship Pioneering Innovations Vi Llc INTEGRASE COMPOSITIONS AND METHODS
EP4352230A4 (en) 2021-06-03 2025-06-11 Prime Medicine, Inc. Genome editing compositions and methods for treating Wilson's disease
WO2023283092A1 (en) 2021-07-06 2023-01-12 Prime Medicine, Inc. Compositions and methods for efficient genome editing
EP4373939A4 (en) 2021-07-23 2025-11-19 Prime Medicine Inc Genome editing compositions and treatment methods for chronic granulomatous disease
WO2023015318A2 (en) 2021-08-05 2023-02-09 Prime Medicine, Inc. Genome editing compositions and methods for treatment of cystic fibrosis
JP2024530487A (ja) 2021-08-06 2024-08-21 ザ ブロード インスティテュート，インコーポレーテッド 改善されたプライムエディターおよび使用方法
CA3231679A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Hbb-modulating compositions and methods
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
IL311219A (en) 2021-09-08 2024-05-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for genome modulation
MX2024002988A (es) 2021-09-08 2024-06-11 Flagship Pioneering Innovations Vi Llc Composiciones y metodos moduladores de la serpina.
EP4419688A2 (en) 2021-10-21 2024-08-28 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
EP4430185A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
EP4430188A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of amyotrophic lateral sclerosis
EP4430189A1 (en) 2021-11-11 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fragile x syndrome
EP4437106A2 (en) 2021-11-24 2024-10-02 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
IL313027A (en) 2021-11-24 2024-07-01 Prime Medicine Inc Modified prime editing guide rnas
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
EP4504921A4 (en) 2022-04-01 2026-04-15 Prime Medicine Inc METHODS AND COMPOSITIONS FOR EDITING NUCLEOTID SEQUENCES

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