JP2024533315A - Ｐａｈ調節性組成物及び方法 - Google Patents

Ｐａｈ調節性組成物及び方法 Download PDF

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JP2024533315A
JP2024533315A JP2024515071A JP2024515071A JP2024533315A JP 2024533315 A JP2024533315 A JP 2024533315A JP 2024515071 A JP2024515071 A JP 2024515071A JP 2024515071 A JP2024515071 A JP 2024515071A JP 2024533315 A JP2024533315 A JP 2024533315A
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sequence
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grna
template rna
template
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JP2024533315A5 (https=
JPWO2023039435A5 (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，
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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 JP2024533315A publication Critical patent/JP2024533315A/ja
Publication of JP2024533315A5 publication Critical patent/JP2024533315A5/ja
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JP2024515071A 2021-09-08 2022-09-07 Ｐａｈ調節性組成物及び方法 Pending JP2024533315A (ja)

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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

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JP2024533315A true JP2024533315A (ja) 2024-09-12
JP2024533315A5 JP2024533315A5 (https=) 2025-09-17
JPWO2023039435A5 JPWO2023039435A5 (https=) 2025-09-17

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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
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
KR20240099164A (ko) 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
JP2024533311A (ja) 2021-09-08 2024-09-12 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 ゲノムを調節するための方法及び組成物
EP4444362A4 (en) 2021-12-10 2026-04-01 Flagship Pioneering Innovations Vi Llc CFTR COMPOSITIONS AND MODULATION METHODS
EP4684023A2 (en) * 2023-03-21 2026-01-28 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
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
EP3875603A1 (en) * 2016-07-12 2021-09-08 Life Technologies Corporation Compositions and methods for detecting nucleic acid regions
CN118530967A (zh) 2017-08-23 2024-08-23 通用医疗公司 具有改变的PAM特异性的工程化的CRISPR-Cas9核酸酶
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
WO2020012335A1 (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
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
US20200268906A1 (en) * 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
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
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
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
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
US20230348939A1 (en) 2020-03-04 2023-11-02 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
JP2023516692A (ja) 2020-03-04 2023-04-20 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 ゲノムを調節するための方法及び組成物
CA3174483A1 (en) 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Improved methods and compositions for modulating a genome
AU2021232069A1 (en) 2020-03-05 2022-11-03 Flagship Pioneering Innovations Vi, Llc Host defense suppressing 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
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
US20240042058A1 (en) 2021-02-26 2024-02-08 Flagship Pioneering Innovations Vi, Llc Tissue-specific methods and compositions for modulating a genome
CA3214277A1 (en) 2021-03-19 2022-09-22 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
WO2022251356A1 (en) 2021-05-26 2022-12-01 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
WO2023015309A2 (en) 2021-08-06 2023-02-09 The Broad Institute, Inc. Improved prime editors and methods of use
JP2024533311A (ja) 2021-09-08 2024-09-12 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 ゲノムを調節するための方法及び組成物
CA3231594A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Serpina-modulating compositions and methods
MX2024002930A (es) 2021-09-08 2024-05-29 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
KR20240099164A (ko) 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
AU2022342169A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Hbb-modulating compositions and methods
WO2023070062A2 (en) 2021-10-21 2023-04-27 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
US20250011766A1 (en) 2021-11-09 2025-01-09 Prime Medicine, Inc. Genome Editing Compositions and Methods for Treatment of Amyotrophic Lateral Sclerosis
EP4430185A1 (en) 2021-11-09 2024-09-18 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
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
JP2024545947A (ja) 2021-11-24 2024-12-16 プライム メディシン， インコーポレイテッド 修飾されたプライム編集ガイドｒｎａ
EP4444362A4 (en) 2021-12-10 2026-04-01 Flagship Pioneering Innovations Vi Llc CFTR COMPOSITIONS AND MODULATION METHODS
EP4504921A4 (en) 2022-04-01 2026-04-15 Prime Medicine Inc METHODS AND COMPOSITIONS FOR EDITING NUCLEOTID SEQUENCES

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