JPWO2020154342A5 - - Google Patents
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- Publication number
- JPWO2020154342A5 JPWO2020154342A5 JP2021541718A JP2021541718A JPWO2020154342A5 JP WO2020154342 A5 JPWO2020154342 A5 JP WO2020154342A5 JP 2021541718 A JP2021541718 A JP 2021541718A JP 2021541718 A JP2021541718 A JP 2021541718A JP WO2020154342 A5 JPWO2020154342 A5 JP WO2020154342A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide
- oligonucleotide
- formula
- nucleotides
- nucleobase
- 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.)
- Pending
Links
- 229920000272 Oligonucleotide Polymers 0.000 claims 76
- 239000002773 nucleotide Substances 0.000 claims 68
- 125000003729 nucleotide group Chemical group 0.000 claims 52
- 229910052739 hydrogen Inorganic materials 0.000 claims 22
- 239000001257 hydrogen Substances 0.000 claims 22
- 229920001914 Ribonucleotide Polymers 0.000 claims 20
- 239000002336 ribonucleotide Substances 0.000 claims 20
- 125000002652 ribonucleotide group Chemical group 0.000 claims 20
- 229920003013 deoxyribonucleic acid Chemical class 0.000 claims 16
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 15
- 125000004435 hydrogen atoms Chemical group [H]* 0.000 claims 13
- 108020004999 Messenger RNA Proteins 0.000 claims 9
- 229920002106 messenger RNA Polymers 0.000 claims 9
- 125000000217 alkyl group Chemical group 0.000 claims 8
- 125000002619 bicyclic group Chemical group 0.000 claims 8
- OIRDTQYFTABQOQ-GAWUUDPSSA-N 9-β-D-XYLOFURANOSYL-ADENINE Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@H](O)[C@H]1O OIRDTQYFTABQOQ-GAWUUDPSSA-N 0.000 claims 7
- OIRDTQYFTABQOQ-SXVXDFOESA-N Adenosine Natural products Nc1ncnc2c1ncn2[C@@H]3O[C@@H](CO)[C@H](O)[C@@H]3O OIRDTQYFTABQOQ-SXVXDFOESA-N 0.000 claims 7
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 7
- 229960005305 adenosine Drugs 0.000 claims 7
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 7
- 229910052736 halogen Inorganic materials 0.000 claims 7
- 150000002367 halogens Chemical class 0.000 claims 7
- 150000002431 hydrogen Chemical class 0.000 claims 7
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 7
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 7
- 239000002131 composite material Substances 0.000 claims 6
- 125000001153 fluoro group Chemical group F* 0.000 claims 6
- 125000003545 alkoxy group Chemical group 0.000 claims 5
- 150000001875 compounds Chemical class 0.000 claims 5
- 201000010099 disease Diseases 0.000 claims 5
- FDGQSTZJBFJUBT-UHFFFAOYSA-N Hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims 4
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 4
- 239000002253 acid Substances 0.000 claims 4
- 230000035772 mutation Effects 0.000 claims 4
- 108020004705 Codon Proteins 0.000 claims 3
- 229940104302 Cytosine Drugs 0.000 claims 3
- OPTASPLRGRRNAP-UHFFFAOYSA-N Cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 3
- 239000002214 arabinonucleotide Substances 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims 2
- NYHBQMYGNKIUIF-PXMDKTAGSA-N Guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1[C@H]1O[C@@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-PXMDKTAGSA-N 0.000 claims 2
- 229940029575 Guanosine Drugs 0.000 claims 2
- 101700080605 NUC1 Proteins 0.000 claims 2
- 208000002320 Spinal Muscular Atrophy Diseases 0.000 claims 2
- RWQNBRDOKXIBIV-UHFFFAOYSA-N Thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 2
- 229940035893 Uracil Drugs 0.000 claims 2
- 201000011510 cancer Diseases 0.000 claims 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 2
- 101700006494 nucA Proteins 0.000 claims 2
- 201000010874 syndrome Diseases 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-Methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- HQULDBYHEHYKRS-UHFFFAOYSA-N 6-amino-1H-pyrimidin-2-one Chemical compound NC1=CC=NC(O)=N1.NC=1C=CNC(=O)N=1 HQULDBYHEHYKRS-UHFFFAOYSA-N 0.000 claims 1
- 102100011470 ABCA4 Human genes 0.000 claims 1
- 229960000643 Adenine Drugs 0.000 claims 1
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Natural products NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 1
- 206010056981 Adenomatous polyposis coli Diseases 0.000 claims 1
- 102000009914 Adenosine deaminases Human genes 0.000 claims 1
- 108091022188 Adenosine deaminases Proteins 0.000 claims 1
- 206010001557 Albinism Diseases 0.000 claims 1
- 206010001897 Alzheimer's disease Diseases 0.000 claims 1
- 206010002026 Amyotrophic lateral sclerosis Diseases 0.000 claims 1
- 208000007502 Anemia Diseases 0.000 claims 1
- 208000006673 Asthma Diseases 0.000 claims 1
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims 1
- 208000010482 CADASIL Diseases 0.000 claims 1
- 102100001891 CTAG1A Human genes 0.000 claims 1
- 101710004449 CTAG1A Proteins 0.000 claims 1
- 206010065551 Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy Diseases 0.000 claims 1
- 201000011470 Charcot-Marie-Tooth disease Diseases 0.000 claims 1
- 206010010099 Combined immunodeficiency Diseases 0.000 claims 1
- 206010013801 Duchenne muscular dystrophy Diseases 0.000 claims 1
- 208000004298 Epidermolysis Bullosa Dystrophica Diseases 0.000 claims 1
- 206010014989 Epidermolysis bullosa Diseases 0.000 claims 1
- 102100015239 F2 Human genes 0.000 claims 1
- 102100009074 FBN1 Human genes 0.000 claims 1
- 101700001106 FBN1 Proteins 0.000 claims 1
- 201000005603 Fabry disease Diseases 0.000 claims 1
- 102100008255 GAA Human genes 0.000 claims 1
- 101710010383 GAA Proteins 0.000 claims 1
- 206010017604 Galactosaemia Diseases 0.000 claims 1
- 206010018048 Gaucher's disease Diseases 0.000 claims 1
- 206010018444 Glucose-6-phosphate dehydrogenase deficiency Diseases 0.000 claims 1
- 208000008605 Glucosephosphate Dehydrogenase Deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- UYTPUPDQBNUYGX-UHFFFAOYSA-N Guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 1
- 102100012716 HEXA Human genes 0.000 claims 1
- 101700075495 HEXA Proteins 0.000 claims 1
- 206010061992 Haemophilia Diseases 0.000 claims 1
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 206010057873 Hereditary haemochromatosis Diseases 0.000 claims 1
- 206010051922 Hereditary non-polyposis colorectal cancer syndrome Diseases 0.000 claims 1
- 201000001971 Huntington's disease Diseases 0.000 claims 1
- 206010061598 Immunodeficiency Diseases 0.000 claims 1
- 206010021972 Inflammatory bowel disease Diseases 0.000 claims 1
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 1
- 208000009856 Lung Disease Diseases 0.000 claims 1
- 201000005027 Lynch syndrome Diseases 0.000 claims 1
- 102100014726 MECP2 Human genes 0.000 claims 1
- 101700029603 MECP2 Proteins 0.000 claims 1
- 208000001826 Marfan Syndrome Diseases 0.000 claims 1
- 108010008148 Member 4 Subfamily A ATP Binding Cassette Transporter Proteins 0.000 claims 1
- 208000002678 Mucopolysaccharidosis Diseases 0.000 claims 1
- 206010028093 Mucopolysaccharidosis Diseases 0.000 claims 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
- 208000003627 Muscular Dystrophy Diseases 0.000 claims 1
- 206010068871 Myotonic dystrophy Diseases 0.000 claims 1
- 201000000794 Niemann-Pick disease type A Diseases 0.000 claims 1
- 201000000791 Niemann-Pick disease type B Diseases 0.000 claims 1
- 206010061536 Parkinson's disease Diseases 0.000 claims 1
- 206010034699 Peroneal muscular atrophy Diseases 0.000 claims 1
- 206010034764 Peutz-Jeghers syndrome Diseases 0.000 claims 1
- 229940039716 Prothrombin Drugs 0.000 claims 1
- 108010094028 Prothrombin Proteins 0.000 claims 1
- 208000002815 Pulmonary Hypertension Diseases 0.000 claims 1
- 208000007014 Retinitis Pigmentosa Diseases 0.000 claims 1
- 208000006289 Rett Syndrome Diseases 0.000 claims 1
- 201000008894 Sandhoff disease Diseases 0.000 claims 1
- 208000002491 Severe Combined Immunodeficiency Diseases 0.000 claims 1
- 206010062766 Stargardt's disease Diseases 0.000 claims 1
- 229920000978 Start codon Polymers 0.000 claims 1
- 206010042265 Sturge-Weber syndrome Diseases 0.000 claims 1
- 201000008902 Tay-Sachs disease Diseases 0.000 claims 1
- 208000002903 Thalassemia Diseases 0.000 claims 1
- 229940113082 Thymine Drugs 0.000 claims 1
- 208000007824 Type A Niemann-Pick Disease Diseases 0.000 claims 1
- 208000008291 Type B Niemann-Pick Disease Diseases 0.000 claims 1
- 208000007930 Type C Niemann-Pick Disease Diseases 0.000 claims 1
- 201000006051 Usher syndrome Diseases 0.000 claims 1
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 1
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 claims 1
- 150000001720 carbohydrates Chemical class 0.000 claims 1
- 235000014633 carbohydrates Nutrition 0.000 claims 1
- 230000001684 chronic Effects 0.000 claims 1
- 230000001886 ciliary Effects 0.000 claims 1
- 230000000875 corresponding Effects 0.000 claims 1
- 201000003883 cystic fibrosis Diseases 0.000 claims 1
- 238000006481 deamination reaction Methods 0.000 claims 1
- 108010091897 factor V Leiden Proteins 0.000 claims 1
- 201000006107 familial adenomatous polyposis Diseases 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 201000004404 neurofibroma Diseases 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 230000000414 obstructive Effects 0.000 claims 1
- 201000011252 phenylketonuria Diseases 0.000 claims 1
- 230000002028 premature Effects 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- 150000003432 sterols Chemical class 0.000 claims 1
- 235000003702 sterols Nutrition 0.000 claims 1
- 235000000346 sugar Nutrition 0.000 claims 1
- 150000008163 sugars Chemical class 0.000 claims 1
- 230000001225 therapeutic Effects 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962795357P | 2019-01-22 | 2019-01-22 | |
| US62/795,357 | 2019-01-22 | ||
| US201962822472P | 2019-03-22 | 2019-03-22 | |
| US62/822,472 | 2019-03-22 | ||
| US201962900019P | 2019-09-13 | 2019-09-13 | |
| US62/900,019 | 2019-09-13 | ||
| PCT/US2020/014510 WO2020154342A1 (en) | 2019-01-22 | 2020-01-22 | Rna-editing oligonucleotides and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022518477A JP2022518477A (ja) | 2022-03-15 |
| JPWO2020154342A5 true JPWO2020154342A5 (zh) | 2023-01-26 |
Family
ID=71736317
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021541718A Pending JP2022518477A (ja) | 2019-01-22 | 2020-01-22 | Ｒｎａ編集オリゴヌクレオチド及びその使用 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20200385713A1 (zh) |
| EP (1) | EP3914259A4 (zh) |
| JP (1) | JP2022518477A (zh) |
| KR (1) | KR20210132022A (zh) |
| CN (1) | CN113453694A (zh) |
| AU (1) | AU2020210900A1 (zh) |
| CA (1) | CA3126947A1 (zh) |
| TW (1) | TW202043469A (zh) |
| WO (1) | WO2020154342A1 (zh) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020508685A (ja) | 2017-03-03 | 2020-03-26 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
| TW202042822A (zh) | 2019-01-22 | 2020-12-01 | 美商科羅生物公司 | Rna編輯之寡核苷酸及其用途 |
| CA3127241A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| CN115038789A (zh) | 2019-12-02 | 2022-09-09 | 塑造治疗公司 | 治疗性编辑 |
| WO2021231691A1 (en) * | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi) |
| CA3162416C (en) * | 2020-05-15 | 2023-07-04 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1) |
| CN116157520A (zh) * | 2020-05-26 | 2023-05-23 | 塑造治疗公司 | 用于基因组编辑的组合物和方法 |
| US20230194709A9 (en) * | 2021-06-29 | 2023-06-22 | Seagate Technology Llc | Range information detection using coherent pulse sets with selected waveform characteristics |
| CN118019848A (zh) * | 2021-09-29 | 2024-05-10 | 豪夫迈·罗氏有限公司 | Rna编辑 |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| CN115304656A (zh) * | 2022-06-22 | 2022-11-08 | 中国人民解放军海军军医大学第一附属医院 | 一种特异性激活细胞泛凋亡途径的囊泡药物构建及应用 |
| WO2024013361A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE60110244T2 (de) * | 2000-09-06 | 2006-01-19 | Mcgill University, Montreal | Chimerische antisense-oligonukleotide aus arabinofuranose-analogen und deoxyribosenukleotide |
| US9943604B2 (en) * | 2013-09-20 | 2018-04-17 | Ionis Pharmaceuticals, Inc. | Targeted therapeutic nucleosides and their use |
| WO2017146186A1 (ja) * | 2016-02-26 | 2017-08-31 | 塩野義製薬株式会社 | Ａｍｐｋ活性化作用を有する５－フェニルアザインドール誘導体 |
| JP7074345B2 (ja) * | 2016-06-22 | 2022-05-24 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | 一本鎖ｒｎａ編集オリゴヌクレオチド |
| EP3507366B1 (en) * | 2016-09-01 | 2020-10-07 | ProQR Therapeutics II B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| CA3117163A1 (en) * | 2018-11-08 | 2020-05-14 | Aligos Therapeutics, Inc. | S-antigen transport inhibiting oligonucleotide polymers and methods |
-
2020
- 2020-01-22 EP EP20744578.4A patent/EP3914259A4/en active Pending
- 2020-01-22 US US16/749,608 patent/US20200385713A1/en not_active Abandoned
- 2020-01-22 JP JP2021541718A patent/JP2022518477A/ja active Pending
- 2020-01-22 AU AU2020210900A patent/AU2020210900A1/en active Pending
- 2020-01-22 CA CA3126947A patent/CA3126947A1/en active Pending
- 2020-01-22 TW TW109102639A patent/TW202043469A/zh unknown
- 2020-01-22 KR KR1020217024842A patent/KR20210132022A/ko unknown
- 2020-01-22 CN CN202080015037.5A patent/CN113453694A/zh active Pending
- 2020-01-22 WO PCT/US2020/014510 patent/WO2020154342A1/en unknown
-
2023
- 2023-05-09 US US18/314,258 patent/US20230332143A1/en active Pending
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