JP2022173349A5 - - Google Patents
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- JP2022173349A5 JP2022173349A5 JP2022152461A JP2022152461A JP2022173349A5 JP 2022173349 A5 JP2022173349 A5 JP 2022173349A5 JP 2022152461 A JP2022152461 A JP 2022152461A JP 2022152461 A JP2022152461 A JP 2022152461A JP 2022173349 A5 JP2022173349 A5 JP 2022173349A5
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- Prior art keywords
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- 150000003839 salts Chemical class 0.000 claims 9
- 108091034117 Oligonucleotide Proteins 0.000 claims 8
- 239000002773 nucleotide Substances 0.000 claims 7
- 125000003729 nucleotide group Chemical group 0.000 claims 6
- 108091033409 CRISPR Proteins 0.000 claims 4
- 230000000295 complement effect Effects 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 2
- 125000000217 alkyl group Chemical group 0.000 claims 2
- 150000001412 amines Chemical group 0.000 claims 2
- 150000003568 thioethers Chemical class 0.000 claims 2
- 238000010354 CRISPR gene editing Methods 0.000 claims 1
- 238000000137 annealing Methods 0.000 claims 1
- 125000001246 bromo group Chemical group Br* 0.000 claims 1
- 125000001309 chloro group Chemical group Cl* 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000001153 fluoro group Chemical group F* 0.000 claims 1
- 229910052739 hydrogen Inorganic materials 0.000 claims 1
- 239000001257 hydrogen Substances 0.000 claims 1
- 125000006239 protecting group Chemical group 0.000 claims 1
- 230000002194 synthesizing effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024195755A JP2025016776A (ja) | 2016-12-30 | 2024-11-08 | 合成ガイド分子、それに関連する組成物および方法 |
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662441046P | 2016-12-30 | 2016-12-30 | |
| US62/441,046 | 2016-12-30 | ||
| US201762492001P | 2017-04-28 | 2017-04-28 | |
| US62/492,001 | 2017-04-28 | ||
| JP2019535879A JP7167029B2 (ja) | 2016-12-30 | 2017-12-29 | 合成ガイド分子、それに関連する組成物および方法 |
| PCT/US2017/069019 WO2018126176A1 (en) | 2016-12-30 | 2017-12-29 | Synthetic guide molecules, compositions and methods relating thereto |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019535879A Division JP7167029B2 (ja) | 2016-12-30 | 2017-12-29 | 合成ガイド分子、それに関連する組成物および方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024195755A Division JP2025016776A (ja) | 2016-12-30 | 2024-11-08 | 合成ガイド分子、それに関連する組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022173349A JP2022173349A (ja) | 2022-11-18 |
| JP2022173349A5 true JP2022173349A5 (enExample) | 2023-01-04 |
| JP7610559B2 JP7610559B2 (ja) | 2025-01-08 |
Family
ID=61187807
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019535879A Active JP7167029B2 (ja) | 2016-12-30 | 2017-12-29 | 合成ガイド分子、それに関連する組成物および方法 |
| JP2022152461A Active JP7610559B2 (ja) | 2016-12-30 | 2022-09-26 | 合成ガイド分子、それに関連する組成物および方法 |
| JP2024195755A Withdrawn JP2025016776A (ja) | 2016-12-30 | 2024-11-08 | 合成ガイド分子、それに関連する組成物および方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019535879A Active JP7167029B2 (ja) | 2016-12-30 | 2017-12-29 | 合成ガイド分子、それに関連する組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024195755A Withdrawn JP2025016776A (ja) | 2016-12-30 | 2024-11-08 | 合成ガイド分子、それに関連する組成物および方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230111575A1 (enExample) |
| EP (1) | EP3565895A1 (enExample) |
| JP (3) | JP7167029B2 (enExample) |
| KR (2) | KR102758180B1 (enExample) |
| CN (2) | CN110249052B (enExample) |
| AU (2) | AU2017388753A1 (enExample) |
| CA (1) | CA3048434A1 (enExample) |
| MX (1) | MX2019007750A (enExample) |
| WO (1) | WO2018126176A1 (enExample) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015134812A1 (en) | 2014-03-05 | 2015-09-11 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| EP3553176A1 (en) | 2014-03-10 | 2019-10-16 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| EP3122880B1 (en) | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| JP2018522249A (ja) | 2015-04-24 | 2018-08-09 | エディタス・メディシン、インコーポレイテッド | Ｃａｓ９分子／ガイドｒｎａ分子複合体の評価 |
| EP3433364A1 (en) | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| WO2018026976A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| BR112019017751A2 (pt) | 2017-02-28 | 2020-04-07 | Vor Biopharma, Inc. | composições e métodos de inibição de proteínas de linhagem específica |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| EP3615672A1 (en) | 2017-04-28 | 2020-03-04 | Editas Medicine, Inc. | Methods and systems for analyzing guide rna molecules |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| US20190076814A1 (en) | 2017-09-11 | 2019-03-14 | Synthego Corporation | Biopolymer synthesis system and method |
| SG11202008956XA (en) | 2018-03-14 | 2020-10-29 | Editas Medicine Inc | Systems and methods for the treatment of hemoglobinopathies |
| KR20210045360A (ko) | 2018-05-16 | 2021-04-26 | 신테고 코포레이션 | 가이드 rna 설계 및 사용을 위한 방법 및 시스템 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| CA3104856A1 (en) | 2018-06-29 | 2020-01-02 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| SG11202101994XA (en) | 2018-08-28 | 2021-03-30 | Vor Biopharma Inc | Genetically engineered hematopoietic stem cells and uses thereof |
| GB201901873D0 (en) * | 2019-02-11 | 2019-04-03 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for nucleic acid editing |
| AU2020280103A1 (en) | 2019-05-23 | 2021-12-23 | Vor Biopharma Inc. | Compositions and methods for CD33 modification |
| US20220290160A1 (en) | 2019-08-28 | 2022-09-15 | Vor Biopharma Inc. | Compositions and methods for cll1 modification |
| CA3151669A1 (en) | 2019-08-28 | 2021-03-04 | Vor Biopharma Inc. | Compositions and methods for cd123 modification |
| US20240110189A1 (en) | 2020-08-28 | 2024-04-04 | Vor Biopharma Inc. | Compositions and methods for cll1 modification |
| EP4204564A1 (en) | 2020-08-28 | 2023-07-05 | Vor Biopharma Inc. | Compositions and methods for cd123 modification |
| WO2022056459A1 (en) | 2020-09-14 | 2022-03-17 | Vor Biopharma, Inc. | Compositions and methods for cd5 modification |
| JP2023541457A (ja) | 2020-09-14 | 2023-10-02 | ブイオーアール バイオファーマ インコーポレーテッド | Ｃｄ３８修飾のための化合物および方法 |
| EP4214318A1 (en) | 2020-09-18 | 2023-07-26 | Vor Biopharma Inc. | Compositions and methods for cd7 modification |
| US20230364233A1 (en) | 2020-09-28 | 2023-11-16 | Vor Biopharma Inc. | Compositions and methods for cd6 modification |
| US20230364146A1 (en) | 2020-09-30 | 2023-11-16 | Vor Biopharma Inc. | Compositions and methods for cd30 gene modification |
| KR20230097089A (ko) | 2020-10-27 | 2023-06-30 | 보르 바이오파마 인크. | 조혈 악성종양을 치료하기 위한 조성물 및 방법 |
| WO2022094245A1 (en) | 2020-10-30 | 2022-05-05 | Vor Biopharma, Inc. | Compositions and methods for bcma modification |
| US20230414755A1 (en) | 2020-11-13 | 2023-12-28 | Vor Biopharma Inc. | Methods and compositions relating to genetically engineered cells expressing chimeric antigen receptors |
| CA3202219A1 (en) | 2020-12-31 | 2022-07-07 | Vor Biopharma Inc. | Compositions and methods for cd34 gene modification |
| US20240200059A1 (en) | 2021-04-09 | 2024-06-20 | Vor Biopharma Inc. | Photocleavable guide rnas and methods of use thereof |
| WO2023283585A2 (en) | 2021-07-06 | 2023-01-12 | Vor Biopharma Inc. | Inhibitor oligonucleotides and methods of use thereof |
| US20250122534A1 (en) | 2021-08-02 | 2025-04-17 | Vor Biopharma Inc. | Compositions and methods for gene modification |
| US20240417755A1 (en) | 2021-09-27 | 2024-12-19 | Vor Biopharma Inc. | Fusion polypeptides for genetic editing and methods of use thereof |
| AU2022387087A1 (en) | 2021-11-09 | 2024-05-02 | Vor Biopharma Inc. | Compositions and methods for erm2 modification |
| US20250179531A1 (en) | 2022-02-25 | 2025-06-05 | Vor Biopharma Inc. | Compositions and methods for homology-directed repair gene modification |
| US20250295695A1 (en) | 2022-04-04 | 2025-09-25 | Vor Biopharma Inc. | Compositions and methods for mediating epitope engineering |
| EP4555091A2 (en) | 2022-07-13 | 2025-05-21 | Vor Biopharma Inc. | Compositions and methods for artificial protospacer adjacent motif (pam) generation |
| WO2024073751A1 (en) | 2022-09-29 | 2024-04-04 | Vor Biopharma Inc. | Methods and compositions for gene modification and enrichment |
| EP4623081A2 (en) * | 2022-11-23 | 2025-10-01 | Prime Medicine, Inc. | Split synthesis of long rnas |
| WO2024168312A1 (en) | 2023-02-09 | 2024-08-15 | Vor Biopharma Inc. | Methods for treating hematopoietic malignancy |
| US20250006305A1 (en) * | 2023-06-29 | 2025-01-02 | Synthego Corporation | Methods and systems for automated fraction selection in nucleic acid manufacturing |
| WO2025030010A1 (en) | 2023-08-01 | 2025-02-06 | Vor Biopharma Inc. | Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof |
| WO2025194138A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | St1cas9 compositions and methods for modulating a genome |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX357240B (es) | 2011-10-10 | 2018-06-29 | Kmt Waterjet Systems Inc | Conexión de alta presion sin juntas. |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| EP3553176A1 (en) | 2014-03-10 | 2019-10-16 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| EP3122880B1 (en) * | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP3204513A2 (en) * | 2014-10-09 | 2017-08-16 | Life Technologies Corporation | Crispr oligonucleotides and gene editing |
| AU2015342749B2 (en) | 2014-11-07 | 2022-01-27 | Editas Medicine, Inc. | Methods for improving CRISPR/Cas-mediated genome-editing |
| US10059940B2 (en) * | 2015-01-27 | 2018-08-28 | Minghong Zhong | Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents |
| ES2884838T3 (es) | 2015-04-06 | 2021-12-13 | Univ Leland Stanford Junior | ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS |
| JP2018522249A (ja) * | 2015-04-24 | 2018-08-09 | エディタス・メディシン、インコーポレイテッド | Ｃａｓ９分子／ガイドｒｎａ分子複合体の評価 |
| JP7288302B2 (ja) | 2015-05-08 | 2023-06-07 | ザ チルドレンズ メディカル センター コーポレーション | 胎児型ヘモグロビン再誘導のための、ｂｃｌ１１ａエンハンサー機能性領域を標的とする方法 |
| CN107709555A (zh) * | 2015-05-15 | 2018-02-16 | 达尔马科恩有限公司 | 用于Cas9介导的基因编辑的合成的单向导RNA |
| CA3013179A1 (en) * | 2016-01-30 | 2017-08-03 | Bonac Corporation | Artificial single guide rna and use thereof |
| WO2017180711A1 (en) | 2016-04-13 | 2017-10-19 | Editas Medicine, Inc. | Grna fusion molecules, gene editing systems, and methods of use thereof |
-
2017
- 2017-12-29 KR KR1020237043357A patent/KR102758180B1/ko active Active
- 2017-12-29 JP JP2019535879A patent/JP7167029B2/ja active Active
- 2017-12-29 KR KR1020197022278A patent/KR102618864B1/ko active Active
- 2017-12-29 CA CA3048434A patent/CA3048434A1/en active Pending
- 2017-12-29 EP EP17840468.7A patent/EP3565895A1/en active Pending
- 2017-12-29 MX MX2019007750A patent/MX2019007750A/es unknown
- 2017-12-29 CN CN201780085248.4A patent/CN110249052B/zh active Active
- 2017-12-29 AU AU2017388753A patent/AU2017388753A1/en not_active Abandoned
- 2017-12-29 CN CN202410342599.0A patent/CN118345072A/zh active Pending
- 2017-12-29 WO PCT/US2017/069019 patent/WO2018126176A1/en not_active Ceased
- 2017-12-29 US US16/474,198 patent/US20230111575A1/en active Pending
-
2022
- 2022-09-26 JP JP2022152461A patent/JP7610559B2/ja active Active
-
2024
- 2024-07-30 AU AU2024205204A patent/AU2024205204A1/en active Pending
- 2024-11-08 JP JP2024195755A patent/JP2025016776A/ja not_active Withdrawn
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