JP2020503049A5 - - Google Patents
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- JP2020503049A5 JP2020503049A5 JP2019535879A JP2019535879A JP2020503049A5 JP 2020503049 A5 JP2020503049 A5 JP 2020503049A5 JP 2019535879 A JP2019535879 A JP 2019535879A JP 2019535879 A JP2019535879 A JP 2019535879A JP 2020503049 A5 JP2020503049 A5 JP 2020503049A5
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- 125000003729 nucleotide group Chemical group 0.000 claims description 200
- 239000002773 nucleotide Substances 0.000 claims description 151
- 239000000203 mixture Substances 0.000 claims description 113
- 150000003839 salts Chemical class 0.000 claims description 85
- 230000000295 complement effect Effects 0.000 claims description 76
- 108091034117 Oligonucleotide Proteins 0.000 claims description 65
- 238000000034 method Methods 0.000 claims description 59
- 108091033409 CRISPR Proteins 0.000 claims description 57
- 238000010354 CRISPR gene editing Methods 0.000 claims description 39
- 125000000217 alkyl group Chemical group 0.000 claims description 34
- 108020004707 nucleic acids Proteins 0.000 claims description 28
- 102000039446 nucleic acids Human genes 0.000 claims description 28
- 150000007523 nucleic acids Chemical class 0.000 claims description 28
- 125000001153 fluoro group Chemical group F* 0.000 claims description 19
- 229910052739 hydrogen Inorganic materials 0.000 claims description 19
- 125000006239 protecting group Chemical group 0.000 claims description 19
- 125000001246 bromo group Chemical group Br* 0.000 claims description 16
- 125000001309 chloro group Chemical group Cl* 0.000 claims description 16
- 239000001257 hydrogen Substances 0.000 claims description 16
- 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 description 15
- 230000002194 synthesizing effect Effects 0.000 claims description 11
- -1 bromoacetyl moiety Chemical group 0.000 claims description 9
- 229910052760 oxygen Inorganic materials 0.000 claims description 7
- 229910052717 sulfur Inorganic materials 0.000 claims description 7
- XSQUKJJJFZCRTK-UHFFFAOYSA-N Urea Chemical compound NC(N)=O XSQUKJJJFZCRTK-UHFFFAOYSA-N 0.000 claims description 5
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 5
- 125000003396 thiol group Chemical group [H]S* 0.000 claims description 5
- 150000001412 amines Chemical group 0.000 claims description 4
- 238000000137 annealing Methods 0.000 claims description 4
- 239000004202 carbamide Substances 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims description 3
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical group NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 16
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical group O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 description 16
- 230000001268 conjugating effect Effects 0.000 description 14
- 0 CC(C(CC1)OC1(C1*)I)[C@@]1OP(*)(OC)=* Chemical compound CC(C(CC1)OC1(C1*)I)[C@@]1OP(*)(OC)=* 0.000 description 12
- 239000000543 intermediate Substances 0.000 description 12
- 230000015572 biosynthetic process Effects 0.000 description 11
- RAXXELZNTBOGNW-UHFFFAOYSA-N imidazole Natural products C1=CNC=N1 RAXXELZNTBOGNW-UHFFFAOYSA-N 0.000 description 11
- 239000012634 fragment Substances 0.000 description 10
- LMDZBCPBFSXMTL-UHFFFAOYSA-N 1-ethyl-3-(3-dimethylaminopropyl)carbodiimide Chemical group CCN=C=NCCCN(C)C LMDZBCPBFSXMTL-UHFFFAOYSA-N 0.000 description 8
- QOSSAOTZNIDXMA-UHFFFAOYSA-N Dicylcohexylcarbodiimide Chemical compound C1CCCCC1N=C=NC1CCCCC1 QOSSAOTZNIDXMA-UHFFFAOYSA-N 0.000 description 8
- JUJWROOIHBZHMG-UHFFFAOYSA-N Pyridine Chemical compound C1=CC=NC=C1 JUJWROOIHBZHMG-UHFFFAOYSA-N 0.000 description 8
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 8
- GFFGJBXGBJISGV-UHFFFAOYSA-N adenyl group Chemical group N1=CN=C2N=CNC2=C1N GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 description 8
- 239000000126 substance Substances 0.000 description 8
- 150000001718 carbodiimides Chemical class 0.000 description 7
- BVKZGUZCCUSVTD-UHFFFAOYSA-L Carbonate Chemical compound [O-]C([O-])=O BVKZGUZCCUSVTD-UHFFFAOYSA-L 0.000 description 6
- 239000012190 activator Substances 0.000 description 6
- 125000002652 ribonucleotide group Chemical group 0.000 description 6
- 238000010362 genome editing Methods 0.000 description 5
- 230000001588 bifunctional effect Effects 0.000 description 4
- 238000003776 cleavage reaction Methods 0.000 description 4
- 102000004169 proteins and genes Human genes 0.000 description 4
- 108090000623 proteins and genes Proteins 0.000 description 4
- UMJSCPRVCHMLSP-UHFFFAOYSA-N pyridine Natural products COC1=CC=CN=C1 UMJSCPRVCHMLSP-UHFFFAOYSA-N 0.000 description 4
- 230000007017 scission Effects 0.000 description 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 3
- QMQZIXCNLUPEIN-UHFFFAOYSA-N 1h-imidazole-2-carbonitrile Chemical compound N#CC1=NC=CN1 QMQZIXCNLUPEIN-UHFFFAOYSA-N 0.000 description 3
- IAZDPXIOMUYVGZ-UHFFFAOYSA-N Dimethylsulphoxide Chemical compound CS(C)=O IAZDPXIOMUYVGZ-UHFFFAOYSA-N 0.000 description 3
- 150000001875 compounds Chemical class 0.000 description 3
- 239000003431 cross linking reagent Substances 0.000 description 3
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 3
- PSHKMPUSSFXUIA-UHFFFAOYSA-N n,n-dimethylpyridin-2-amine Chemical compound CN(C)C1=CC=CC=N1 PSHKMPUSSFXUIA-UHFFFAOYSA-N 0.000 description 3
- 238000000746 purification Methods 0.000 description 3
- 125000000547 substituted alkyl group Chemical group 0.000 description 3
- FDKXTQMXEQVLRF-ZHACJKMWSA-N (E)-dacarbazine Chemical compound CN(C)\N=N\c1[nH]cnc1C(N)=O FDKXTQMXEQVLRF-ZHACJKMWSA-N 0.000 description 2
- XKRFYHLGVUSROY-UHFFFAOYSA-N Argon Chemical compound [Ar] XKRFYHLGVUSROY-UHFFFAOYSA-N 0.000 description 2
- KCXVZYZYPLLWCC-UHFFFAOYSA-N EDTA Chemical compound OC(=O)CN(CC(O)=O)CCN(CC(O)=O)CC(O)=O KCXVZYZYPLLWCC-UHFFFAOYSA-N 0.000 description 2
- 102000004190 Enzymes Human genes 0.000 description 2
- 108090000790 Enzymes Proteins 0.000 description 2
- YNAVUWVOSKDBBP-UHFFFAOYSA-N Morpholine Chemical compound C1COCCN1 YNAVUWVOSKDBBP-UHFFFAOYSA-N 0.000 description 2
- ZMXDDKWLCZADIW-UHFFFAOYSA-N N,N-Dimethylformamide Chemical compound CN(C)C=O ZMXDDKWLCZADIW-UHFFFAOYSA-N 0.000 description 2
- 101710163270 Nuclease Proteins 0.000 description 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-N Phosphoric acid Chemical group OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 description 2
- 239000004698 Polyethylene Substances 0.000 description 2
- 239000002202 Polyethylene glycol Substances 0.000 description 2
- 239000004743 Polypropylene Substances 0.000 description 2
- 125000004452 carbocyclyl group Chemical group 0.000 description 2
- 150000001768 cations Chemical class 0.000 description 2
- 238000006243 chemical reaction Methods 0.000 description 2
- 239000002184 metal Substances 0.000 description 2
- 229920000573 polyethylene Polymers 0.000 description 2
- 229920001223 polyethylene glycol Polymers 0.000 description 2
- 229920001155 polypropylene Polymers 0.000 description 2
- 229920001451 polypropylene glycol Polymers 0.000 description 2
- 239000003381 stabilizer Substances 0.000 description 2
- WEVYAHXRMPXWCK-UHFFFAOYSA-N Acetonitrile Chemical compound CC#N WEVYAHXRMPXWCK-UHFFFAOYSA-N 0.000 description 1
- KPDBJZUAABGPIN-UHFFFAOYSA-N CC(C1O)OC(CO)C1O Chemical compound CC(C1O)OC(CO)C1O KPDBJZUAABGPIN-UHFFFAOYSA-N 0.000 description 1
- FREIHJREAFWAML-CLFYSBASSA-N CCCCCOC(C(COP(O)(O)=O)OC1BN(C)N[I-]C(C(C2O)O)O/C2=C\O)C1O Chemical compound CCCCCOC(C(COP(O)(O)=O)OC1BN(C)N[I-]C(C(C2O)O)O/C2=C\O)C1O FREIHJREAFWAML-CLFYSBASSA-N 0.000 description 1
- GHCAMHZMPOXCGS-UHFFFAOYSA-N CN(BC(C1[O]=C)OC(COP(O)(O)=O)C1O)N1N(C)N(C)N1 Chemical compound CN(BC(C1[O]=C)OC(COP(O)(O)=O)C1O)N1N(C)N(C)N1 GHCAMHZMPOXCGS-UHFFFAOYSA-N 0.000 description 1
- XDZFOYOPRMXWOT-UHFFFAOYSA-N CNBC(C1O)OC(CO)C1O Chemical compound CNBC(C1O)OC(CO)C1O XDZFOYOPRMXWOT-UHFFFAOYSA-N 0.000 description 1
- 239000004971 Cross linker Substances 0.000 description 1
- GSNUFIFRDBKVIE-UHFFFAOYSA-N DMF Natural products CC1=CC=C(C)O1 GSNUFIFRDBKVIE-UHFFFAOYSA-N 0.000 description 1
- 102100034343 Integrase Human genes 0.000 description 1
- FXHOOIRPVKKKFG-UHFFFAOYSA-N N,N-Dimethylacetamide Chemical compound CN(C)C(C)=O FXHOOIRPVKKKFG-UHFFFAOYSA-N 0.000 description 1
- SECXISVLQFMRJM-UHFFFAOYSA-N N-Methylpyrrolidone Chemical compound CN1CCCC1=O SECXISVLQFMRJM-UHFFFAOYSA-N 0.000 description 1
- AHVYPIQETPWLSZ-UHFFFAOYSA-N N-methyl-pyrrolidine Natural products CN1CC=CC1 AHVYPIQETPWLSZ-UHFFFAOYSA-N 0.000 description 1
- 108010092799 RNA-directed DNA polymerase Proteins 0.000 description 1
- SPEUIVXLLWOEMJ-UHFFFAOYSA-N acetaldehyde dimethyl acetal Natural products COC(C)OC SPEUIVXLLWOEMJ-UHFFFAOYSA-N 0.000 description 1
- 229910052786 argon Inorganic materials 0.000 description 1
- ACBQROXDOHKANW-UHFFFAOYSA-N bis(4-nitrophenyl) carbonate Chemical compound C1=CC([N+](=O)[O-])=CC=C1OC(=O)OC1=CC=C([N+]([O-])=O)C=C1 ACBQROXDOHKANW-UHFFFAOYSA-N 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 230000021615 conjugation Effects 0.000 description 1
- 239000006184 cosolvent Substances 0.000 description 1
- 238000004132 cross linking Methods 0.000 description 1
- 238000005520 cutting process Methods 0.000 description 1
- 230000005782 double-strand break Effects 0.000 description 1
- 125000002883 imidazolyl group Chemical group 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 230000005783 single-strand break Effects 0.000 description 1
- 239000000758 substrate Substances 0.000 description 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022152461A 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 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 (5)
| 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 | ||
| PCT/US2017/069019 WO2018126176A1 (en) | 2016-12-30 | 2017-12-29 | Synthetic guide molecules, compositions and methods relating thereto |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022152461A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020503049A JP2020503049A (ja) | 2020-01-30 |
| JP2020503049A5 true JP2020503049A5 (enExample) | 2021-02-12 |
| JP7167029B2 JP7167029B2 (ja) | 2022-11-08 |
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 After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230111575A1 (enExample) |
| EP (1) | EP3565895A1 (enExample) |
| JP (3) | JP7167029B2 (enExample) |
| KR (2) | KR102618864B1 (enExample) |
| CN (2) | CN110249052B (enExample) |
| AU (2) | AU2017388753A1 (enExample) |
| CA (1) | CA3048434A1 (enExample) |
| MX (1) | MX420023B (enExample) |
| WO (1) | WO2018126176A1 (enExample) |
Families Citing this family (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11028388B2 (en) | 2014-03-05 | 2021-06-08 | Editas Medicine, Inc. | CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US9938521B2 (en) | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| EP3981876A1 (en) | 2014-03-26 | 2022-04-13 | 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 |
| AU2015367378A1 (en) | 2014-12-17 | 2017-06-15 | Proqr Therapeutics Ii B.V. | Targeted RNA editing |
| EP4019975A1 (en) | 2015-04-24 | 2022-06-29 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| EP3433364A1 (en) | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| US11566263B2 (en) | 2016-08-02 | 2023-01-31 | Editas Medicine, Inc. | Compositions and methods for treating CEP290 associated disease |
| WO2018041973A1 (en) | 2016-09-01 | 2018-03-08 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| KR102709603B1 (ko) | 2017-02-28 | 2024-09-27 | 보르 바이오파마 인크. | 계통 특이적 단백질의 억제를 위한 조성물 및 방법 |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| US11499151B2 (en) | 2017-04-28 | 2022-11-15 | Editas Medicine, Inc. | Methods and systems for analyzing guide RNA molecules |
| EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | 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 |
| EP3681637A4 (en) | 2017-09-11 | 2021-06-02 | Synthego Corporation | SYSTEM AND PROCEDURE FOR BIOPOLYMER SYNTHESIS |
| IL277234B2 (en) | 2018-03-14 | 2026-03-01 | Editas Medicine Inc | Systems and methods for treating hemoglobin diseases |
| CN112654710A (zh) | 2018-05-16 | 2021-04-13 | 辛瑟高公司 | 用于指导rna设计和使用的方法和系统 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| AU2019291918B2 (en) | 2018-06-29 | 2025-06-12 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| CA3110837A1 (en) | 2018-08-28 | 2020-03-05 | 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 |
| AU2020338011A1 (en) | 2019-08-28 | 2022-03-10 | Vor Biopharma Inc. | Compositions and methods for CLL1 modification |
| WO2021041977A1 (en) | 2019-08-28 | 2021-03-04 | Vor Biopharma, Inc. | Compositions and methods for cd123 modification |
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| CA3199623A1 (en) | 2020-10-27 | 2022-05-05 | Vor Biopharma Inc. | Compositions and methods for treating hematopoietic malignancy |
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| AU2021413252A1 (en) | 2020-12-31 | 2023-06-08 | Vor Biopharma Inc. | Compositions and methods for cd34 gene modification |
| WO2022217086A1 (en) | 2021-04-09 | 2022-10-13 | 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 |
| EP4381062A1 (en) | 2021-08-02 | 2024-06-12 | Vor Biopharma Inc. | Compositions and methods for gene modification |
| EP4408991A2 (en) | 2021-09-27 | 2024-08-07 | Vor Biopharma Inc. | Fusion polypeptides for genetic editing and methods of use thereof |
| EP4430186A1 (en) | 2021-11-09 | 2024-09-18 | 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 |
| WO2024015925A2 (en) | 2022-07-13 | 2024-01-18 | 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 |
| WO2024112876A2 (en) * | 2022-11-23 | 2024-05-30 | Prime Medicine, Inc. | Split synthesis of long rnas |
| WO2024168312A1 (en) | 2023-02-09 | 2024-08-15 | Vor Biopharma Inc. | Methods for treating hematopoietic malignancy |
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| 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 |
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| EP2766642B1 (en) | 2011-10-10 | 2019-07-03 | KMT Waterjet Systems, Inc. | Gasketless high pressure connection |
| CN111218447B (zh) * | 2013-11-07 | 2024-10-11 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| US9938521B2 (en) | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| EP3981876A1 (en) * | 2014-03-26 | 2022-04-13 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| CN113930455A (zh) * | 2014-10-09 | 2022-01-14 | 生命技术公司 | Crispr寡核苷酸和基因剪辑 |
| WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | 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 |
| CN107787367B (zh) * | 2015-04-06 | 2021-10-26 | 里兰斯坦福初级大学理事会 | 用于crispr/cas介导的基因调控的化学修饰的引导rna |
| EP4019975A1 (en) * | 2015-04-24 | 2022-06-29 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| EP3763814A1 (en) * | 2015-05-08 | 2021-01-13 | The Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
| 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 |
| WO2017131237A1 (ja) * | 2016-01-30 | 2017-08-03 | 株式会社ボナック | 人工単一ガイドｒｎａ及びその用途 |
| WO2017180711A1 (en) | 2016-04-13 | 2017-10-19 | Editas Medicine, Inc. | Grna fusion molecules, gene editing systems, and methods of use thereof |
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- 2017-12-29 KR KR1020197022278A patent/KR102618864B1/ko active Active
- 2017-12-29 WO PCT/US2017/069019 patent/WO2018126176A1/en not_active Ceased
- 2017-12-29 KR KR1020237043357A patent/KR102758180B1/ko active Active
- 2017-12-29 US US16/474,198 patent/US20230111575A1/en active Pending
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- 2017-12-29 CN CN202410342599.0A patent/CN118345072A/zh active Pending
- 2017-12-29 CA CA3048434A patent/CA3048434A1/en active Pending
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- 2017-12-29 MX MX2019007750A patent/MX420023B/es unknown
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2022
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