JP2023510352A5 - - Google Patents

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Publication number
JP2023510352A5
JP2023510352A5 JP2022542340A JP2022542340A JP2023510352A5 JP 2023510352 A5 JP2023510352 A5 JP 2023510352A5 JP 2022542340 A JP2022542340 A JP 2022542340A JP 2022542340 A JP2022542340 A JP 2022542340A JP 2023510352 A5 JP2023510352 A5 JP 2023510352A5
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JP
Japan
Application number
JP2022542340A
Other languages
Japanese (ja)
Other versions
JPWO2021142342A5 (https=
JP2023510352A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/012804 external-priority patent/WO2021142342A1/en
Publication of JP2023510352A publication Critical patent/JP2023510352A/ja
Publication of JPWO2021142342A5 publication Critical patent/JPWO2021142342A5/ja
Publication of JP2023510352A5 publication Critical patent/JP2023510352A5/ja
Pending legal-status Critical Current

Links

JP2022542340A 2020-01-10 2021-01-08 Pcsk9の標的化のための組成物および方法 Pending JP2023510352A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062959685P 2020-01-10 2020-01-10
US62/959,685 2020-01-10
PCT/US2021/012804 WO2021142342A1 (en) 2020-01-10 2021-01-08 Compositions and methods for the targeting of pcsk9

Publications (3)

Publication Number Publication Date
JP2023510352A JP2023510352A (ja) 2023-03-13
JPWO2021142342A5 JPWO2021142342A5 (https=) 2024-01-16
JP2023510352A5 true JP2023510352A5 (https=) 2024-01-16

Family

ID=74505368

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022542340A Pending JP2023510352A (ja) 2020-01-10 2021-01-08 Pcsk9の標的化のための組成物および方法

Country Status (9)

Country Link
US (1) US20230167424A1 (https=)
EP (1) EP4087930A1 (https=)
JP (1) JP2023510352A (https=)
KR (1) KR20220125332A (https=)
CN (1) CN115427570A (https=)
AU (1) AU2021206270A1 (https=)
CA (1) CA3163714A1 (https=)
IL (1) IL294620A (https=)
WO (1) WO2021142342A1 (https=)

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US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
PH12021553076A1 (en) 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
US20250388885A1 (en) * 2022-03-11 2025-12-25 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
WO2023235818A2 (en) * 2022-06-02 2023-12-07 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
WO2023240076A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240074A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
US20250361525A1 (en) 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ, インコーポレーテッド Mecp2発現を低減する化合物及び方法
TW202444906A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 用於靶向pcsk9之組合物及方法
WO2024238723A1 (en) * 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
CN118086311B (zh) * 2023-05-25 2024-08-09 苏州时安生物技术有限公司 抑制PCSK9基因表达的siRNA、其缀合物和药物组合物及用途
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions
CN116978457B (zh) * 2023-09-22 2023-12-22 成都斯马特科技有限公司 避免rna检测过程中假基因干扰的引物和探针组合及其设计方法
CN119506351B (zh) * 2024-11-20 2025-08-19 中国人民解放军陆军军医大学第二附属医院 一体化pcsk9腺嘌呤碱基编辑系统及应用

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US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
CA2999649A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
CA3038960A1 (en) 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
EP3559223A1 (en) * 2016-12-23 2019-10-30 President and Fellows of Harvard College Gene editing of pcsk9
EP3585894A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders
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WO2019126762A2 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
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