JP2020508659A5 - - Google Patents
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- Publication number
- JP2020508659A5 JP2020508659A5 JP2019545242A JP2019545242A JP2020508659A5 JP 2020508659 A5 JP2020508659 A5 JP 2020508659A5 JP 2019545242 A JP2019545242 A JP 2019545242A JP 2019545242 A JP2019545242 A JP 2019545242A JP 2020508659 A5 JP2020508659 A5 JP 2020508659A5
- Authority
- JP
- Japan
- Prior art keywords
- dna
- composition
- pcsk9
- endonucleases
- deoxyribonucleic 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.)
- Granted
Links
- 108020004414 DNA Proteins 0.000 claims description 66
- 102000053602 DNA Human genes 0.000 claims description 66
- 238000000034 method Methods 0.000 claims description 44
- 108010042407 Endonucleases Proteins 0.000 claims description 40
- 102000004533 Endonucleases Human genes 0.000 claims description 40
- 108020005004 Guide RNA Proteins 0.000 claims description 37
- 101150094724 PCSK9 gene Proteins 0.000 claims description 37
- 101710180553 Proprotein convertase subtilisin/kexin type 9 Proteins 0.000 claims description 31
- 102100038955 Proprotein convertase subtilisin/kexin type 9 Human genes 0.000 claims description 31
- 230000005782 double-strand break Effects 0.000 claims description 25
- 108091033409 CRISPR Proteins 0.000 claims description 20
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 19
- 229920002477 rna polymer Polymers 0.000 claims description 19
- 210000004027 cell Anatomy 0.000 claims description 15
- 230000001105 regulatory effect Effects 0.000 claims description 14
- 230000005783 single-strand break Effects 0.000 claims description 13
- 210000003494 hepatocyte Anatomy 0.000 claims description 12
- 102000040430 polynucleotide Human genes 0.000 claims description 12
- 108091033319 polynucleotide Proteins 0.000 claims description 12
- 239000002157 polynucleotide Substances 0.000 claims description 12
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 11
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 11
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 125000006850 spacer group Chemical group 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 9
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 8
- 230000037430 deletion Effects 0.000 claims description 8
- 238000012217 deletion Methods 0.000 claims description 8
- 230000037431 insertion Effects 0.000 claims description 8
- 238000003780 insertion Methods 0.000 claims description 8
- 230000035772 mutation Effects 0.000 claims description 8
- 239000002773 nucleotide Substances 0.000 claims description 8
- 125000003729 nucleotide group Chemical group 0.000 claims description 8
- 210000002901 mesenchymal stem cell Anatomy 0.000 claims description 6
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims description 5
- 239000002245 particle Substances 0.000 claims description 5
- 150000001413 amino acids Chemical class 0.000 claims description 4
- 210000004899 c-terminal region Anatomy 0.000 claims description 4
- 238000002347 injection Methods 0.000 claims description 4
- 239000007924 injection Substances 0.000 claims description 4
- 230000003834 intracellular effect Effects 0.000 claims description 4
- 150000002632 lipids Chemical class 0.000 claims description 4
- 239000002502 liposome Substances 0.000 claims description 4
- 239000002105 nanoparticle Substances 0.000 claims description 4
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 4
- 230000000295 complement effect Effects 0.000 claims description 3
- 108091028113 Trans-activating crRNA Proteins 0.000 claims description 2
- 238000010362 genome editing Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 229920001184 polypeptide Polymers 0.000 claims description 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 230000009885 systemic effect Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 27
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 241000282693 Cercopithecidae Species 0.000 claims 1
- 108020004705 Codon Proteins 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 230000007017 scission Effects 0.000 claims 1
- 238000002054 transplantation Methods 0.000 claims 1
- 208000009869 Neu-Laxova syndrome Diseases 0.000 description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 4
- 208000035475 disorder Diseases 0.000 description 4
- 239000013598 vector Substances 0.000 description 3
- 241001052819 Amylobacillus thermophilus Species 0.000 description 2
- 238000010354 CRISPR gene editing Methods 0.000 description 2
- 241000093740 Acidaminococcus sp. Species 0.000 description 1
- 241000894006 Bacteria Species 0.000 description 1
- 108091026890 Coding region Proteins 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 108700024394 Exon Proteins 0.000 description 1
- 201000009906 Meningitis Diseases 0.000 description 1
- 230000004075 alteration Effects 0.000 description 1
- 230000000536 complexating effect Effects 0.000 description 1
- 239000013612 plasmid Substances 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762461852P | 2017-02-22 | 2017-02-22 | |
| US62/461,852 | 2017-02-22 | ||
| US201762526646P | 2017-06-29 | 2017-06-29 | |
| US62/526,646 | 2017-06-29 | ||
| PCT/IB2018/000208 WO2018154380A1 (en) | 2017-02-22 | 2018-02-15 | Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020508659A JP2020508659A (ja) | 2020-03-26 |
| JP2020508659A5 true JP2020508659A5 (https=) | 2021-03-25 |
| JP7277052B2 JP7277052B2 (ja) | 2023-05-18 |
Family
ID=61972166
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019545242A Active JP7277052B2 (ja) | 2017-02-22 | 2018-02-15 | 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 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20200248168A1 (https=) |
| EP (1) | EP3585894A1 (https=) |
| JP (1) | JP7277052B2 (https=) |
| CN (1) | CN110582570A (https=) |
| AU (2) | AU2018224380A1 (https=) |
| CA (1) | CA3053709A1 (https=) |
| WO (1) | WO2018154380A1 (https=) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| JOP20190215A1 (ar) | 2017-03-24 | 2019-09-19 | Ionis Pharmaceuticals Inc | مُعدّلات التعبير الوراثي عن pcsk9 |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3592140A1 (en) | 2018-03-19 | 2020-01-15 | Regeneron Pharmaceuticals, Inc. | Transcription modulation in animals using crispr/cas systems |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| IL294620A (en) * | 2020-01-10 | 2022-09-01 | Scribe Therapeutics Inc | Compositions and methods for the targeting of pcsk9 |
| CA3175071A1 (en) | 2020-03-11 | 2021-09-16 | Bit Bio Limited | Method of generating hepatic cells |
| MX2022012683A (es) * | 2020-04-09 | 2023-01-11 | Verve Therapeutics Inc | Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades. |
| CN111378754B (zh) * | 2020-04-23 | 2020-11-17 | 嘉兴市第一医院 | 基于tcga数据库的乳腺癌甲基化生物标志物及其筛选方法 |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| CN114525258A (zh) * | 2020-10-30 | 2022-05-24 | 未来智人再生医学研究院(广州)有限公司 | 一种表达pcsk9阻断物的多能干细胞或其衍生物及应用 |
| US20240318172A1 (en) * | 2020-12-01 | 2024-09-26 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of peripheral myelin protein-22 |
| CA3204073A1 (en) * | 2020-12-10 | 2022-06-16 | Apellis Pharmaceuticals, Inc. | Genomic editing of complement |
| CN114657136A (zh) * | 2020-12-22 | 2022-06-24 | 未来智人再生医学研究院(广州)有限公司 | 一种表达靶向PCSK9的shRNA和/或shRNA-miR的多能干细胞或其衍生物 |
| WO2023069707A2 (en) * | 2021-10-22 | 2023-04-27 | Sirnaomics, Inc. | Products and compositions |
| WO2023093862A1 (en) * | 2021-11-26 | 2023-06-01 | Epigenic Therapeutics Inc. | Method of modulating pcsk9 and uses thereof |
| KR20240117571A (ko) | 2021-12-08 | 2024-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 돌연변이 마이오실린 질환 모델 및 이의 용도 |
| EP4479535A1 (en) | 2022-02-14 | 2024-12-25 | ProQR Therapeutics II B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| IL317874A (en) * | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| WO2024061296A2 (en) * | 2022-09-22 | 2024-03-28 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease |
| KR20250124819A (ko) * | 2022-12-21 | 2025-08-20 | 인텔리아 테라퓨틱스, 인크. | 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법 |
| WO2024238723A1 (en) * | 2023-05-16 | 2024-11-21 | Omega Therapeutics, Inc. | Methods and compositions for modulating pcsk9 expression |
| WO2025006563A1 (en) * | 2023-06-26 | 2025-01-02 | Arbor Biotechnologies, Inc. | Gene editing systems targeting pcsk9 and uses thereof |
| WO2025096355A1 (en) * | 2023-10-29 | 2025-05-08 | Corsera Llc | Improved sirna for silencing expression of pcsk9 and uses thereof |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20070173473A1 (en) * | 2001-05-18 | 2007-07-26 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA) |
| US9255250B2 (en) * | 2012-12-05 | 2016-02-09 | Sangamo Bioscience, Inc. | Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene |
| RU2716420C2 (ru) * | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
| EP3288594B1 (en) * | 2015-04-27 | 2022-06-29 | The Trustees of The University of Pennsylvania | Dual aav vector system for crispr/cas9 mediated correction of human disease |
| CN105886498A (zh) * | 2015-05-13 | 2016-08-24 | 沈志荣 | CRISPR-Cas9特异性敲除人PCSK9基因的方法以及用于特异性靶向PCSK9基因的sgRNA |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| CA2947904A1 (en) * | 2015-11-12 | 2017-05-12 | Pfizer Inc. | Tissue-specific genome engineering using crispr-cas9 |
| CN109715801B (zh) * | 2015-12-01 | 2022-11-01 | 克里斯普治疗股份公司 | 用于治疗α1抗胰蛋白酶缺乏的材料和方法 |
| KR20180118111A (ko) * | 2015-12-23 | 2018-10-30 | 크리스퍼 테라퓨틱스 아게 | 근위축성 측색 경화증 및/또는 전두측두엽 퇴행의 치료 물질 및 방법 |
| US20190127713A1 (en) * | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| US20190211317A1 (en) * | 2016-05-27 | 2019-07-11 | Aadigen, Llc | Peptides and nanoparticles for intracellular delivery of genome-editing molecules |
| US20180127786A1 (en) * | 2016-09-23 | 2018-05-10 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing |
| KR20230125856A (ko) * | 2016-12-23 | 2023-08-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Pcsk9의 유전자 편집 |
-
2018
- 2018-02-15 AU AU2018224380A patent/AU2018224380A1/en not_active Abandoned
- 2018-02-15 WO PCT/IB2018/000208 patent/WO2018154380A1/en not_active Ceased
- 2018-02-15 JP JP2019545242A patent/JP7277052B2/ja active Active
- 2018-02-15 US US16/488,149 patent/US20200248168A1/en active Pending
- 2018-02-15 CN CN201880026222.7A patent/CN110582570A/zh active Pending
- 2018-02-15 CA CA3053709A patent/CA3053709A1/en active Pending
- 2018-02-15 EP EP18717687.0A patent/EP3585894A1/en active Pending
-
2024
- 2024-08-01 AU AU2024205415A patent/AU2024205415A1/en active Pending
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