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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- Prior art keywords
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- item
- seq
- guide molecule
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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 (https=) | 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 (https=) |
| EP (1) | EP3565895A1 (https=) |
| JP (3) | JP7167029B2 (https=) |
| KR (2) | KR102758180B1 (https=) |
| CN (2) | CN118345072A (https=) |
| AU (2) | AU2017388753A1 (https=) |
| CA (1) | CA3048434A1 (https=) |
| MX (1) | MX420023B (https=) |
| WO (1) | WO2018126176A1 (https=) |
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 |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| US11242525B2 (en) | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| US12460231B2 (en) | 2014-04-02 | 2025-11-04 | Editas Medicine, Inc. | Crispr/CAS-related methods and compositions for treating primary open angle glaucoma |
| CA2982966C (en) | 2015-04-24 | 2024-02-20 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | 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 |
| WO2018026976A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| US10941402B2 (en) | 2016-09-01 | 2021-03-09 | 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 |
| SG11201907870VA (en) | 2017-02-28 | 2019-09-27 | Vor Biopharma Inc | Compositions and methods for inhibition of lineage specific proteins |
| 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 |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | 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 |
| WO2019178426A1 (en) | 2018-03-14 | 2019-09-19 | 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 |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED 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 |
| CN114423865A (zh) | 2019-05-23 | 2022-04-29 | Vor生物制药股份有限公司 | Cd33修饰的组合物和方法 |
| AU2020336211A1 (en) | 2019-08-28 | 2022-03-10 | Vor Biopharma Inc. | Compositions and methods for CD123 modification |
| US20220290160A1 (en) | 2019-08-28 | 2022-09-15 | Vor Biopharma Inc. | Compositions and methods for cll1 modification |
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| US20240238344A1 (en) | 2020-08-28 | 2024-07-18 | Vor Biopharma Inc. | Compositions and methods for cd123 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 |
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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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| JP2023550355A (ja) | 2020-11-13 | 2023-12-01 | ブイオーアール バイオファーマ インコーポレーテッド | キメラ抗原受容体を発現する遺伝子操作された細胞に関連する方法および組成物 |
| WO2022147347A1 (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 |
| US20240384304A1 (en) | 2021-07-06 | 2024-11-21 | Vor Biopharma Inc. | Inhibitor oligonucleotides and methods of use thereof |
| AU2022324093A1 (en) | 2021-08-02 | 2024-02-08 | 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 |
| CA3236152A1 (en) | 2021-11-09 | 2023-05-19 | 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 |
| 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 |
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| MX357240B (es) * | 2011-10-10 | 2018-06-29 | Kmt Waterjet Systems Inc | Conexión de alta presion sin juntas. |
| AU2014346559B2 (en) * | 2013-11-07 | 2020-07-09 | Editas Medicine,Inc. | CRISPR-related methods and compositions with governing gRNAs |
| WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| US11242525B2 (en) * | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| WO2016057951A2 (en) * | 2014-10-09 | 2016-04-14 | Life Technologies Corporation | Crispr oligonucleotides and gene editing |
| CA2963820A1 (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 |
| EP3280803B1 (en) * | 2015-04-06 | 2021-05-26 | The Board of Trustees of the Leland Stanford Junior University | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
| CA2982966C (en) * | 2015-04-24 | 2024-02-20 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| ES2835861T5 (en) * | 2015-05-08 | 2025-02-18 | Childrens Medical Ct Corp | 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 |
| US11518994B2 (en) * | 2016-01-30 | 2022-12-06 | Bonac Corporation | Artificial single guide RNA and use thereof |
| EP3443088B1 (en) | 2016-04-13 | 2024-09-18 | Editas Medicine, Inc. | Grna fusion molecules, gene editing systems, and methods of use thereof |
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2017
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- 2017-12-29 MX MX2019007750A patent/MX420023B/es unknown
- 2017-12-29 CN CN202410342599.0A patent/CN118345072A/zh active Pending
- 2017-12-29 JP JP2019535879A patent/JP7167029B2/ja active Active
- 2017-12-29 KR KR1020237043357A patent/KR102758180B1/ko active Active
- 2017-12-29 WO PCT/US2017/069019 patent/WO2018126176A1/en not_active Ceased
- 2017-12-29 KR KR1020197022278A patent/KR102618864B1/ko active Active
- 2017-12-29 US US16/474,198 patent/US20230111575A1/en active Pending
- 2017-12-29 CA CA3048434A patent/CA3048434A1/en active Pending
- 2017-12-29 EP EP17840468.7A patent/EP3565895A1/en active Pending
- 2017-12-29 CN CN201780085248.4A patent/CN110249052B/zh active Active
-
2022
- 2022-09-26 JP JP2022152461A patent/JP7610559B2/ja active Active
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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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