JPWO2021217002A5 - - Google Patents
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- JPWO2021217002A5 JPWO2021217002A5 JP2022564464A JP2022564464A JPWO2021217002A5 JP WO2021217002 A5 JPWO2021217002 A5 JP WO2021217002A5 JP 2022564464 A JP2022564464 A JP 2022564464A JP 2022564464 A JP2022564464 A JP 2022564464A JP WO2021217002 A5 JPWO2021217002 A5 JP WO2021217002A5
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- 229920001184 polypeptide Polymers 0.000 claims 102
- 108090000765 processed proteins & peptides Proteins 0.000 claims 102
- 102000004196 processed proteins & peptides Human genes 0.000 claims 102
- 150000001413 amino acids Chemical group 0.000 claims 62
- 108020005004 Guide RNA Proteins 0.000 claims 43
- 108091033409 CRISPR Proteins 0.000 claims 41
- 238000010354 CRISPR gene editing Methods 0.000 claims 41
- 108091028113 Trans-activating crRNA Proteins 0.000 claims 40
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 32
- 108091079001 CRISPR RNA Proteins 0.000 claims 20
- 102000039446 nucleic acids Human genes 0.000 claims 15
- 108020004707 nucleic acids Proteins 0.000 claims 15
- 150000007523 nucleic acids Chemical class 0.000 claims 15
- 210000004027 cell Anatomy 0.000 claims 12
- 101710163270 Nuclease Proteins 0.000 claims 11
- 102000040430 polynucleotide Human genes 0.000 claims 11
- 108091033319 polynucleotide Proteins 0.000 claims 11
- 239000002157 polynucleotide Substances 0.000 claims 11
- 238000000034 method Methods 0.000 claims 10
- 239000002773 nucleotide Substances 0.000 claims 10
- 125000003729 nucleotide group Chemical group 0.000 claims 10
- 108020004414 DNA Proteins 0.000 claims 7
- 102000053602 DNA Human genes 0.000 claims 6
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 claims 6
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Natural products CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 claims 6
- 239000012636 effector Substances 0.000 claims 6
- 239000004474 valine Substances 0.000 claims 6
- 238000003776 cleavage reaction Methods 0.000 claims 5
- 230000007017 scission Effects 0.000 claims 5
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims 4
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- AGPKZVBTJJNPAG-WHFBIAKZSA-N L-isoleucine Chemical compound CC[C@H](C)[C@H](N)C(O)=O AGPKZVBTJJNPAG-WHFBIAKZSA-N 0.000 claims 3
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims 3
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Natural products CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 claims 3
- 208000009869 Neu-Laxova syndrome Diseases 0.000 claims 3
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims 3
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims 3
- 239000004473 Threonine Substances 0.000 claims 3
- 238000012258 culturing Methods 0.000 claims 3
- 230000004049 epigenetic modification Effects 0.000 claims 3
- 229960000310 isoleucine Drugs 0.000 claims 3
- AGPKZVBTJJNPAG-UHFFFAOYSA-N isoleucine Natural products CCC(C)C(N)C(O)=O AGPKZVBTJJNPAG-UHFFFAOYSA-N 0.000 claims 3
- 230000035772 mutation Effects 0.000 claims 3
- 230000029279 positive regulation of transcription, DNA-dependent Effects 0.000 claims 3
- 230000037426 transcriptional repression Effects 0.000 claims 3
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 2
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 2
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 210000003527 eukaryotic cell Anatomy 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 230000009977 dual effect Effects 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 230000003834 intracellular effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 210000001236 prokaryotic cell Anatomy 0.000 claims 1
- 125000002652 ribonucleotide group Chemical group 0.000 claims 1
- 125000006850 spacer group Chemical group 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063014970P | 2020-04-24 | 2020-04-24 | |
| US63/014,970 | 2020-04-24 | ||
| US202063077211P | 2020-09-11 | 2020-09-11 | |
| US63/077,211 | 2020-09-11 | ||
| PCT/US2021/028843 WO2021217002A1 (en) | 2020-04-24 | 2021-04-23 | Rna-guided nucleases and active fragments and variants thereof and methods of use |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023516226A JP2023516226A (ja) | 2023-04-18 |
| JPWO2021217002A5 true JPWO2021217002A5 (https=) | 2024-05-02 |
| JP2023516226A5 JP2023516226A5 (https=) | 2024-05-02 |
Family
ID=75919401
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022564464A Pending JP2023516226A (ja) | 2020-04-24 | 2021-04-23 | Ｒｎａ誘導型ヌクレアーゼとその活性な断片およびバリアント、ならびに利用の方法 |
Country Status (17)
| Country | Link |
|---|---|
| US (6) | US20230175018A1 (https=) |
| EP (2) | EP4242306A3 (https=) |
| JP (1) | JP2023516226A (https=) |
| KR (1) | KR20230014700A (https=) |
| CN (1) | CN116096879A (https=) |
| AU (1) | AU2021258273A1 (https=) |
| BR (1) | BR112022021459A2 (https=) |
| CA (1) | CA3174124A1 (https=) |
| DK (1) | DK4118198T3 (https=) |
| ES (1) | ES3029182T3 (https=) |
| FI (1) | FI4118198T3 (https=) |
| IL (1) | IL297500A (https=) |
| MX (1) | MX2022013187A (https=) |
| PL (1) | PL4118198T3 (https=) |
| PT (1) | PT4118198T (https=) |
| TW (1) | TW202208626A (https=) |
| WO (1) | WO2021217002A1 (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| TW202208626A (zh) * | 2020-04-24 | 2022-03-01 | 美商生命編輯公司 | Rna引導核酸酶及其活性片段與變體，以及使用方法 |
| EP4288541A1 (en) | 2021-02-05 | 2023-12-13 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| CN117295817A (zh) | 2021-03-22 | 2023-12-26 | 生命编辑制药股份有限公司 | Dna修饰酶及其活性片段和变体以及使用方法 |
| AU2022290278A1 (en) | 2021-06-11 | 2024-01-04 | LifeEDIT Therapeutics, Inc. | Rna polymerase iii promoters and methods of use |
| CA3264752A1 (en) | 2022-08-12 | 2024-02-15 | Life Edit Therapeutics, Inc. | RNA-GUIDED NUCLEASE AND ACTIVE FRAGMENTS, ASSOCIATED VARIANTS AND METHODS OF USE |
| TW202424186A (zh) | 2022-08-25 | 2024-06-16 | 美商生命編輯治療學公司 | Rna引導核酸酶中介基因編輯用之具鎖核酸引導 rna之化學修飾 |
| EP4612284A2 (en) | 2022-11-04 | 2025-09-10 | Life Edit Therapeutics, Inc. | Evolved adenine deaminases and rna-guided nuclease fusion proteins with internal insertion sites and methods of use |
| CA3277009A1 (en) | 2022-12-16 | 2024-06-20 | Life Edit Therapeutics, Inc. | RNA GUIDELINES TARGETING THE TRAC GENE AND METHODS OF USE |
| EP4634383A1 (en) | 2022-12-16 | 2025-10-22 | Life Edit Therapeutics, Inc. | Guide rnas that target foxp3 gene and methods of use |
| WO2024178397A2 (en) | 2023-02-24 | 2024-08-29 | Elevatebio Technologies, Inc. | Modified immune effector cells and methods of use |
| TW202507006A (zh) * | 2023-04-12 | 2025-02-16 | 美商生命編輯治療學公司 | 藉由編輯突變亨廷頓基因治療亨廷頓氏病之組合物及方法 |
| WO2025083619A1 (en) | 2023-10-18 | 2025-04-24 | Life Edit Therapeutics, Inc. | Rna-guided nucleases and acive fragments and variants thereof and methods of use |
| TW202546224A (zh) | 2024-02-12 | 2025-12-01 | 美商生命編輯治療學公司 | 新穎的ｒｎａ引導之核酸酶及用於聚合酶編輯之蛋白質 |
| WO2026003754A1 (en) | 2024-06-25 | 2026-01-02 | Life Edit Therapeutics, Inc. | Novel reverse transcriptases and uses thereof |
| WO2026044087A1 (en) | 2024-08-21 | 2026-02-26 | Shape Therapeutics Inc. | Increased cellular stability for aav production |
| WO2026073032A1 (en) | 2024-09-27 | 2026-04-02 | Shape Therapeutics Inc. | Constructs for improved aav production |
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-
2021
- 2021-04-19 TW TW110113939A patent/TW202208626A/zh unknown
- 2021-04-23 PT PT217258805T patent/PT4118198T/pt unknown
- 2021-04-23 CA CA3174124A patent/CA3174124A1/en active Pending
- 2021-04-23 JP JP2022564464A patent/JP2023516226A/ja active Pending
- 2021-04-23 BR BR112022021459A patent/BR112022021459A2/pt unknown
- 2021-04-23 CN CN202180044771.9A patent/CN116096879A/zh active Pending
- 2021-04-23 PL PL21725880.5T patent/PL4118198T3/pl unknown
- 2021-04-23 EP EP23152451.3A patent/EP4242306A3/en active Pending
- 2021-04-23 EP EP21725880.5A patent/EP4118198B1/en active Active
- 2021-04-23 WO PCT/US2021/028843 patent/WO2021217002A1/en not_active Ceased
- 2021-04-23 KR KR1020227040633A patent/KR20230014700A/ko active Pending
- 2021-04-23 AU AU2021258273A patent/AU2021258273A1/en active Pending
- 2021-04-23 IL IL297500A patent/IL297500A/en unknown
- 2021-04-23 DK DK21725880.5T patent/DK4118198T3/da active
- 2021-04-23 FI FIEP21725880.5T patent/FI4118198T3/fi active
- 2021-04-23 MX MX2022013187A patent/MX2022013187A/es unknown
- 2021-04-23 ES ES21725880T patent/ES3029182T3/es active Active
-
2022
- 2022-06-28 US US17/851,887 patent/US20230175018A1/en active Pending
- 2022-08-31 US US17/823,897 patent/US11859181B2/en active Active
- 2022-09-01 US US17/929,165 patent/US11981916B2/en active Active
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2023
- 2023-08-10 US US18/447,947 patent/US20240026384A1/en active Pending
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2024
- 2024-04-29 US US18/649,317 patent/US12252706B2/en active Active
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2025
- 2025-02-13 US US19/052,482 patent/US20250179529A1/en active Pending
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