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
Prior art keywords
sequence
improved
protein
seq
nucleic acid
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
JP2022542340A
Other languages
English (en)
Japanese (ja)
Other versions
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 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 (2)

Publication Number Publication Date
JP2023510352A JP2023510352A (ja) 2023-03-13
JP2023510352A5 true JP2023510352A5 (enExample) 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 (enExample)
EP (1) EP4087930A1 (enExample)
JP (1) JP2023510352A (enExample)
KR (1) KR20220125332A (enExample)
CN (1) CN115427570A (enExample)
AU (1) AU2021206270A1 (enExample)
CA (1) CA3163714A1 (enExample)
IL (1) IL294620A (enExample)
WO (1) WO2021142342A1 (enExample)

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US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
SG11202113253SA (en) 2019-06-07 2021-12-30 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
WO2023173110A1 (en) * 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
CA3257824A1 (en) * 2022-06-02 2023-12-07 Scribe Therapeutics Inc. MODIFIED TYPE V, CLASS 2 CRISPR SYSTEMS
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
EP4536286A1 (en) 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
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
WO2024206555A1 (en) * 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
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
EP4735603A2 (en) * 2023-06-30 2026-05-06 nChroma Bio, 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
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
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
US9580714B2 (en) 2010-11-24 2017-02-28 The University Of Western Australia Peptides for the specific binding of RNA targets
US11866727B2 (en) * 2015-11-06 2024-01-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1A
EP3374494A4 (en) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE THEREOF FOR GENE THERAPY
JP2019532644A (ja) * 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Rna誘導型核酸修飾酵素及びその使用方法
KR102569848B1 (ko) * 2016-12-23 2023-08-25 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
JP7277052B2 (ja) * 2017-02-22 2023-05-18 クリスパー セラピューティクス アーゲー プロタンパク質転換酵素サブチリシン/ケキシン9型(pcsk9)関連障害の処置のための組成物および方法
US11773409B2 (en) 2017-04-21 2023-10-03 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors
WO2019084148A1 (en) * 2017-10-25 2019-05-02 Monsanto Technology Llc TARGETED RNA GUIDED ENDONUCLEASE ENDONUCLEASE ACTIVITY IN EUKARYOTES
WO2019126762A2 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
SG11202113253SA (en) * 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
US12551560B2 (en) * 2019-09-09 2026-02-17 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy

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