JP2024534575A - Ｐｃｓｋ９又はａｎｇｐｔｌ３の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法 - Google Patents

Ｐｃｓｋ９又はａｎｇｐｔｌ３の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法 Download PDF

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JP2024534575A
JP2024534575A JP2024518410A JP2024518410A JP2024534575A JP 2024534575 A JP2024534575 A JP 2024534575A JP 2024518410 A JP2024518410 A JP 2024518410A JP 2024518410 A JP2024518410 A JP 2024518410A JP 2024534575 A JP2024534575 A JP 2024534575A
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sequence
nucleic acid
pharmaceutical composition
editing system
dna
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JP2024534575A5 (enExample
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ベリンジャー，アンドリュー・エム
ラジーブ，カラントッタチル・ジー
レイス，キャロライン
デニジオ，ジェイミー
ジャヤラム，ハリハラン
アイヤー，ソウミャ
アルメイダ・ピント・ガルシア，サラ・クリスティナ デ
ワーン，クイ
チャドウィック，アレクサンドラ
チェン，クリストファー
リー，リチャード・グレン
ローデ，エレン
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ヴァーヴ・セラピューティクス，インコーポレーテッド
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Publication of JP2024534575A publication Critical patent/JP2024534575A/ja
Priority to JP2025171713A priority Critical patent/JP2026012737A/ja
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    • 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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JP2024518410A 2021-09-22 2022-09-22 Ｐｃｓｋ９又はａｎｇｐｔｌ３の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法 Pending JP2024534575A (ja)

Priority Applications (1)

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JP2025171713A JP2026012737A (ja) 2021-09-22 2025-10-10 Ｐｃｓｋ９又はａｎｇｐｔｌ３の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163247236P 2021-09-22 2021-09-22
US63/247,236 2021-09-22
US202163255333P 2021-10-13 2021-10-13
US63/255,333 2021-10-13
US202263389679P 2022-07-15 2022-07-15
US63/389,679 2022-07-15
PCT/US2022/044453 WO2023049299A2 (en) 2021-09-22 2022-09-22 Gene editing of pcsk9 or angptl3 and compositions and methods of using same for treatment of disease

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JP2025171713A Division JP2026012737A (ja) 2021-09-22 2025-10-10 Ｐｃｓｋ９又はａｎｇｐｔｌ３の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法

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JP2024534575A true JP2024534575A (ja) 2024-09-20
JP2024534575A5 JP2024534575A5 (enExample) 2025-10-21

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JP2024518410A Pending JP2024534575A (ja) 2021-09-22 2022-09-22 Ｐｃｓｋ９又はａｎｇｐｔｌ３の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法
JP2025171713A Pending JP2026012737A (ja) 2021-09-22 2025-10-10 Ｐｃｓｋ９又はａｎｇｐｔｌ３の遺伝子編集、並びに疾患の治療のためにそれらを使用する組成物及び方法

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US (1) US20240390520A1 (enExample)
EP (1) EP4405470A2 (enExample)
JP (2) JP2024534575A (enExample)
KR (2) KR20250160515A (enExample)
AU (1) AU2022349449A1 (enExample)
CA (1) CA3232742A1 (enExample)
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CA2990699A1 (en) * 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
AU2019336245B2 (en) 2018-09-07 2026-02-26 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
CN114040977B (zh) 2019-01-31 2024-09-03 比姆医疗股份有限公司 非靶脱氨反应减低的核碱基编辑器和用于定性核碱基编辑器的测定
AU2020223314A1 (en) 2019-02-13 2021-08-12 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
SG11202108346WA (en) 2019-02-13 2021-08-30 Beam Therapeutics Inc Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
CN115279398B (zh) 2019-09-27 2025-07-04 比姆医疗股份有限公司 治疗液态癌症的组合物和方法
US12576151B2 (en) 2020-09-25 2026-03-17 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
WO2025137465A1 (en) * 2023-12-21 2025-06-26 Beam Therapeutics Inc. Base editors with modified editing windows
WO2025167914A1 (zh) * 2024-02-07 2025-08-14 益杰立科新加坡有限公司 通过表观编辑多靶点治疗疾病的方法

Family Cites Families (3)

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Publication number Priority date Publication date Assignee Title
US8691750B2 (en) * 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
SI3250691T1 (sl) * 2015-01-28 2023-10-30 Caribou Biosciences, Inc. Hibridni dna/rna-polinukleotidi crispr in postopki za uporabo
SG10202104041PA (en) * 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof

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EP4405470A2 (en) 2024-07-31
US20240390520A1 (en) 2024-11-28
GB202509407D0 (en) 2025-07-30
WO2023049299A3 (en) 2023-05-19
KR20250160515A (ko) 2025-11-13
CA3232742A1 (en) 2023-03-30
KR20240099200A (ko) 2024-06-28
MX2024003570A (es) 2024-05-31
GB202405381D0 (en) 2024-05-29
IL311607A (en) 2024-05-01
AU2022349449A1 (en) 2024-04-18
GB2700667A (en) 2026-02-25
WO2023049299A2 (en) 2023-03-30
GB202509406D0 (en) 2025-07-30
GB2628238A (en) 2024-09-18
GB2701689A (en) 2026-05-06
JP2026012737A (ja) 2026-01-27

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