JP2023510352A - Pcsk9の標的化のための組成物および方法 - Google Patents
Pcsk9の標的化のための組成物および方法 Download PDFInfo
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- JP2023510352A JP2023510352A JP2022542340A JP2022542340A JP2023510352A JP 2023510352 A JP2023510352 A JP 2023510352A JP 2022542340 A JP2022542340 A JP 2022542340A JP 2022542340 A JP2022542340 A JP 2022542340A JP 2023510352 A JP2023510352 A JP 2023510352A
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- gna
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- casx
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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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- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21111—Aqualysin 1 (3.4.21.111)
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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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
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70503—Immunoglobulin superfamily
- C07K14/70539—MHC-molecules, e.g. HLA-molecules
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- C12N15/09—Recombinant DNA-technology
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- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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- C12N15/86—Viral vectors
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- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
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- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Cell Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
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- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Crystallography & Structural Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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 true JP2023510352A (ja) | 2023-03-13 |
| JPWO2021142342A5 JPWO2021142342A5 (https=) | 2024-01-16 |
| JP2023510352A5 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=) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017077386A1 (en) * | 2015-11-06 | 2017-05-11 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1a |
| WO2018064371A1 (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 |
| WO2018119354A1 (en) * | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Gene editing of pcsk9 |
| WO2018154380A1 (en) * | 2017-02-22 | 2018-08-30 | Crispr Therapeutics Ag | Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders |
| WO2019126762A2 (en) * | 2017-12-22 | 2019-06-27 | The Broad Institute, Inc. | Cas12a systems, methods, and compositions for targeted rna base editing |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO2012068627A1 (en) | 2010-11-24 | 2012-05-31 | The University Of Western Australia | Peptides for the specific binding of rna targets |
| WO2017083722A1 (en) | 2015-11-11 | 2017-05-18 | Greenberg Kenneth P | Crispr compositions and methods of using the same for gene therapy |
| WO2018195555A1 (en) | 2017-04-21 | 2018-10-25 | 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 |
| US11578334B2 (en) * | 2017-10-25 | 2023-02-14 | Monsanto Technology Llc | Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes |
| PH12021553076A1 (en) * | 2019-06-07 | 2023-08-14 | Scribe Therapeutics Inc | Engineered casx systems |
| CA3153700A1 (en) * | 2019-09-09 | 2021-03-18 | Scribe Therapeutics Inc. | Compositions and methods for use in immunotherapy |
-
2021
- 2021-01-08 AU AU2021206270A patent/AU2021206270A1/en active Pending
- 2021-01-08 IL IL294620A patent/IL294620A/en unknown
- 2021-01-08 US US17/791,130 patent/US20230167424A1/en active Pending
- 2021-01-08 CN CN202180019446.7A patent/CN115427570A/zh active Pending
- 2021-01-08 EP EP21703125.1A patent/EP4087930A1/en active Pending
- 2021-01-08 KR KR1020227027481A patent/KR20220125332A/ko active Pending
- 2021-01-08 WO PCT/US2021/012804 patent/WO2021142342A1/en not_active Ceased
- 2021-01-08 CA CA3163714A patent/CA3163714A1/en active Pending
- 2021-01-08 JP JP2022542340A patent/JP2023510352A/ja active Pending
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017077386A1 (en) * | 2015-11-06 | 2017-05-11 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1a |
| WO2018064371A1 (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 |
| WO2018119354A1 (en) * | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Gene editing of pcsk9 |
| WO2018154380A1 (en) * | 2017-02-22 | 2018-08-30 | Crispr Therapeutics Ag | Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders |
| WO2019126762A2 (en) * | 2017-12-22 | 2019-06-27 | The Broad Institute, Inc. | Cas12a systems, methods, and compositions for targeted rna base editing |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3163714A1 (en) | 2021-07-15 |
| KR20220125332A (ko) | 2022-09-14 |
| AU2021206270A1 (en) | 2022-07-21 |
| CN115427570A (zh) | 2022-12-02 |
| EP4087930A1 (en) | 2022-11-16 |
| WO2021142342A1 (en) | 2021-07-15 |
| US20230167424A1 (en) | 2023-06-01 |
| IL294620A (en) | 2022-09-01 |
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