JP2025504898A - Ｒｎａ誘導型ヌクレアーゼ及びその活性断片及びバリアント、並びに使用方法 - Google Patents

Ｒｎａ誘導型ヌクレアーゼ及びその活性断片及びバリアント、並びに使用方法 Download PDF

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JP2025504898A
JP2025504898A JP2024543890A JP2024543890A JP2025504898A JP 2025504898 A JP2025504898 A JP 2025504898A JP 2024543890 A JP2024543890 A JP 2024543890A JP 2024543890 A JP2024543890 A JP 2024543890A JP 2025504898 A JP2025504898 A JP 2025504898A
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seq
sequence
sequence identity
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tracrrna
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JPWO2023139557A5 (https=
JP2025504898A5 (https=
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エイチ．アリア グンジャン
コイル マイケル
ブライナー クロウリー アレグザンドラ
ディー．エリック テッド
エス．パーカー ジョエル
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ライフエディット セラピューティクス，インコーポレイティド
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    • A—HUMAN NECESSITIES
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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
    • A—HUMAN NECESSITIES
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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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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    • C12N9/1241—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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    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04002—Adenine deaminase (3.5.4.2)

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JP2024543890A 2022-01-24 2023-01-23 Ｒｎａ誘導型ヌクレアーゼ及びその活性断片及びバリアント、並びに使用方法 Pending JP2025504898A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263302271P 2022-01-24 2022-01-24
US63/302,271 2022-01-24
US202263386061P 2022-12-05 2022-12-05
US63/386,061 2022-12-05
PCT/IB2023/050560 WO2023139557A1 (en) 2022-01-24 2023-01-23 Rna-guided nucleases and active fragments and variants thereof and methods of use

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JP2025504898A true JP2025504898A (ja) 2025-02-19
JPWO2023139557A5 JPWO2023139557A5 (https=) 2026-02-02
JP2025504898A5 JP2025504898A5 (https=) 2026-02-02

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US (4) US12435325B2 (https=)
EP (2) EP4466349A1 (https=)
JP (1) JP2025504898A (https=)
KR (1) KR20250008537A (https=)
CN (1) CN119317713A (https=)
AU (1) AU2023208961A1 (https=)
CA (1) CA3243220A1 (https=)
IL (1) IL314488A (https=)
MX (1) MX2024009148A (https=)
TW (1) TW202346583A (https=)
WO (1) WO2023139557A1 (https=)

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EP4612284A2 (en) 2022-11-04 2025-09-10 Life Edit Therapeutics, Inc. Evolved adenine deaminases and rna-guided nuclease fusion proteins with internal insertion sites and methods of use
CA3277009A1 (en) * 2022-12-16 2024-06-20 Life Edit Therapeutics, Inc. RNA GUIDELINES TARGETING THE TRAC GENE AND METHODS OF USE
WO2024178397A2 (en) 2023-02-24 2024-08-29 Elevatebio Technologies, Inc. Modified immune effector cells and methods of use
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
TW202546224A (zh) 2024-02-12 2025-12-01 美商生命編輯治療學公司 新穎的ｒｎａ引導之核酸酶及用於聚合酶編輯之蛋白質
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof

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