JPWO2020154343A5 - - Google Patents
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- Publication number
- JPWO2020154343A5 JPWO2020154343A5 JP2021541716A JP2021541716A JPWO2020154343A5 JP WO2020154343 A5 JPWO2020154343 A5 JP WO2020154343A5 JP 2021541716 A JP2021541716 A JP 2021541716A JP 2021541716 A JP2021541716 A JP 2021541716A JP WO2020154343 A5 JPWO2020154343 A5 JP WO2020154343A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide
- oligonucleotide
- nucleotides
- formulas
- ribonucleotide
- 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
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- 239000002773 nucleotide Substances 0.000 claims 66
- 229920000272 Oligonucleotide Polymers 0.000 claims 52
- 125000003729 nucleotide group Chemical group 0.000 claims 50
- 229920001914 Ribonucleotide Polymers 0.000 claims 19
- 239000002336 ribonucleotide Substances 0.000 claims 19
- 125000002652 ribonucleotide group Chemical group 0.000 claims 19
- 229920003013 deoxyribonucleic acid Chemical class 0.000 claims 16
- 229910052739 hydrogen Inorganic materials 0.000 claims 11
- 239000001257 hydrogen Substances 0.000 claims 11
- 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
- UFHFLCQGNIYNRP-UHFFFAOYSA-N hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 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
- 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
- 125000003545 alkoxy group Chemical group 0.000 claims 4
- 239000002214 arabinonucleotide Substances 0.000 claims 4
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 4
- 230000035772 mutation Effects 0.000 claims 4
- 108020004705 Codon Proteins 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 229940104302 Cytosine Drugs 0.000 claims 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N Cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 2
- 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
- 229940035893 Uracil Drugs 0.000 claims 2
- 201000011510 cancer Diseases 0.000 claims 2
- 150000002431 hydrogen Chemical class 0.000 claims 2
- 125000004435 hydrogen atoms Chemical group [H]* 0.000 claims 2
- 101700006494 nucA Proteins 0.000 claims 2
- 201000010874 syndrome Diseases 0.000 claims 2
- 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
- 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
- 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
- 125000001153 fluoro group Chemical group F* 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
- 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 |
|---|---|---|---|
| US201962795348P | 2019-01-22 | 2019-01-22 | |
| US62/795,348 | 2019-01-22 | ||
| US201962822586P | 2019-03-22 | 2019-03-22 | |
| US62/822,586 | 2019-03-22 | ||
| US201962900017P | 2019-09-13 | 2019-09-13 | |
| US62/900,017 | 2019-09-13 | ||
| PCT/US2020/014511 WO2020154343A1 (en) | 2019-01-22 | 2020-01-22 | Rna-editing oligonucleotides and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022519184A JP2022519184A (ja) | 2022-03-22 |
| JPWO2020154343A5 true JPWO2020154343A5 (zh) | 2023-01-26 |
Family
ID=71736903
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021541716A Pending JP2022519184A (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 (3) | US11453878B2 (zh) |
| EP (1) | EP3914260A4 (zh) |
| JP (1) | JP2022519184A (zh) |
| KR (1) | KR20210129646A (zh) |
| CN (1) | CN113518623A (zh) |
| AU (1) | AU2020210645A1 (zh) |
| CA (1) | CA3127241A1 (zh) |
| TW (1) | TW202043468A (zh) |
| WO (1) | WO2020154343A1 (zh) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11932856B2 (en) | 2017-03-03 | 2024-03-19 | The Regents Of The University Of California | RNA targeting of mutations via suppressor tRNAs and deaminases |
| US11479575B2 (en) | 2019-01-22 | 2022-10-25 | Korro Bio, Inc. | RNA-editing oligonucleotides and uses thereof |
| CN113518623A (zh) | 2019-01-22 | 2021-10-19 | 科罗生物公司 | Rna编辑的寡核苷酸及其用途 |
| EP4069842A1 (en) | 2019-12-02 | 2022-10-12 | Shape Therapeutics Inc. | Therapeutic editing |
| EP4150088A1 (en) * | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1) |
| EP4150089A1 (en) * | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1) |
| US20230183689A1 (en) * | 2020-05-26 | 2023-06-15 | Shape Therapeutics Inc. | Compositions and Methods for Genome Editing |
| EP4341405A1 (en) * | 2021-05-20 | 2024-03-27 | Korro Bio, Inc. | Methods and compositions for adar-mediated editing |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| 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 |
| WO2024110565A1 (en) | 2022-11-24 | 2024-05-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| WO2024121373A1 (en) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5399676A (en) | 1989-10-23 | 1995-03-21 | Gilead Sciences | Oligonucleotides with inverted polarity |
| US5314893A (en) | 1993-01-25 | 1994-05-24 | Bristol-Myers Squibb Co. | Antiviral tetrahydropyrans |
| US6878805B2 (en) * | 2002-08-16 | 2005-04-12 | Isis Pharmaceuticals, Inc. | Peptide-conjugated oligomeric compounds |
| US8192937B2 (en) | 2004-04-07 | 2012-06-05 | Exiqon A/S | Methods for quantification of microRNAs and small interfering RNAs |
| US20110065774A1 (en) * | 2008-01-31 | 2011-03-17 | Alnylam Pharmaceuticals | Chemically modified oligonucleotides and uses thereof |
| TWI595885B (zh) | 2012-05-02 | 2017-08-21 | 喜納製藥公司 | 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法 |
| CA2946003A1 (en) * | 2014-05-01 | 2015-11-05 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating angiopoietin-like 3 expression |
| EP3230445B1 (en) | 2014-12-12 | 2024-01-24 | Tod M. Woolf | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
| AU2015367378A1 (en) | 2014-12-17 | 2017-06-15 | Proqr Therapeutics Ii B.V. | Targeted RNA editing |
| US11390865B2 (en) | 2015-07-14 | 2022-07-19 | Fukuoka University | Method for introducing site-directed RNA mutation, target editing guide RNA used in the method and target RNA-target editing guide RNA complex |
| DE102015012522B3 (de) | 2015-09-26 | 2016-06-02 | Eberhard Karls Universität Tübingen | Verfahren und Substanzen zur gerichteten RNA-Editierung |
| EP3421467A4 (en) | 2016-02-26 | 2019-08-07 | Shionogi & Co., Ltd | 5-PHENYLAZAINDOLE DERIVATIVE HAVING AMPK ACTIVATION EFFECT |
| WO2017218813A1 (en) * | 2016-06-15 | 2017-12-21 | University Of Utah Research Foundation | Compositions and methods for using albumin-based nanomedicines |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| PL3507366T3 (pl) * | 2016-09-01 | 2021-05-04 | Proqr Therapeutics Ii B.V. | Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna |
| WO2018134301A1 (en) | 2017-01-19 | 2018-07-26 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in rna editing |
| WO2019111957A1 (ja) | 2017-12-06 | 2019-06-13 | 学校法人福岡大学 | オリゴヌクレオチド、その製造方法及び標的ｒｎａの部位特異的編集方法 |
| AU2019214858B2 (en) | 2018-01-30 | 2023-02-02 | Metro International Biotech, Llc | Nicotinamide riboside analogs, pharmaceutical compositions, and uses thereof |
| US20210079393A1 (en) | 2018-02-14 | 2021-03-18 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for rna editing |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| CA3108289A1 (en) * | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CN113453694A (zh) | 2019-01-22 | 2021-09-28 | 科罗生物公司 | Rna编辑的寡核苷酸及其用途 |
| US11479575B2 (en) | 2019-01-22 | 2022-10-25 | Korro Bio, Inc. | RNA-editing oligonucleotides and uses thereof |
| CN113518623A (zh) | 2019-01-22 | 2021-10-19 | 科罗生物公司 | Rna编辑的寡核苷酸及其用途 |
| JP2022523302A (ja) | 2019-01-28 | 2022-04-22 | 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 | アッシャー症候群の処置のためのｒｎａ編集オリゴヌクレオチド |
| GB201901873D0 (en) | 2019-02-11 | 2019-04-03 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for nucleic acid editing |
-
2020
- 2020-01-22 CN CN202080018215.XA patent/CN113518623A/zh active Pending
- 2020-01-22 KR KR1020217024844A patent/KR20210129646A/ko unknown
- 2020-01-22 AU AU2020210645A patent/AU2020210645A1/en active Pending
- 2020-01-22 US US16/749,601 patent/US11453878B2/en active Active
- 2020-01-22 EP EP20745581.7A patent/EP3914260A4/en active Pending
- 2020-01-22 JP JP2021541716A patent/JP2022519184A/ja active Pending
- 2020-01-22 CA CA3127241A patent/CA3127241A1/en active Pending
- 2020-01-22 WO PCT/US2020/014511 patent/WO2020154343A1/en unknown
- 2020-01-22 TW TW109102604A patent/TW202043468A/zh unknown
-
2022
- 2022-08-05 US US17/817,874 patent/US20230104978A1/en active Pending
-
2023
- 2023-05-03 US US18/311,416 patent/US20230340464A1/en active Pending
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