JPWO2019200185A5 - - Google Patents
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- JPWO2019200185A5 JPWO2019200185A5 JP2020555234A JP2020555234A JPWO2019200185A5 JP WO2019200185 A5 JPWO2019200185 A5 JP WO2019200185A5 JP 2020555234 A JP2020555234 A JP 2020555234A JP 2020555234 A JP2020555234 A JP 2020555234A JP WO2019200185 A5 JPWO2019200185 A5 JP WO2019200185A5
- Authority
- JP
- Japan
- Prior art keywords
- independently
- heteroatoms
- ring
- optionally
- oligonucleotide
- 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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- 125000005842 heteroatoms Chemical group 0.000 claims 88
- 229920000272 Oligonucleotide Polymers 0.000 claims 54
- 125000004429 atoms Chemical group 0.000 claims 36
- 239000000203 mixture Substances 0.000 claims 32
- 125000003118 aryl group Chemical group 0.000 claims 23
- 125000000623 heterocyclic group Chemical group 0.000 claims 19
- 125000001072 heteroaryl group Chemical group 0.000 claims 17
- 125000002723 alicyclic group Chemical group 0.000 claims 13
- 125000001931 aliphatic group Chemical group 0.000 claims 13
- 125000002950 monocyclic group Chemical group 0.000 claims 13
- 229910052698 phosphorus Inorganic materials 0.000 claims 13
- 125000003367 polycyclic group Chemical group 0.000 claims 13
- 125000002619 bicyclic group Chemical group 0.000 claims 9
- 229910052736 halogen Inorganic materials 0.000 claims 7
- 150000002367 halogens Chemical class 0.000 claims 7
- 230000004048 modification Effects 0.000 claims 7
- 238000006011 modification reaction Methods 0.000 claims 7
- 229910052757 nitrogen Inorganic materials 0.000 claims 7
- 125000004450 alkenylene group Chemical group 0.000 claims 6
- 229910052799 carbon Inorganic materials 0.000 claims 6
- 125000004432 carbon atoms Chemical group C* 0.000 claims 6
- 229910052739 hydrogen Inorganic materials 0.000 claims 6
- 125000000325 methylidene group Chemical group [H]C([H])=* 0.000 claims 6
- 229910052760 oxygen Inorganic materials 0.000 claims 6
- 229910052717 sulfur Inorganic materials 0.000 claims 6
- 125000003161 (C1-C6) alkylene group Chemical group 0.000 claims 5
- 230000027455 binding Effects 0.000 claims 5
- 125000004122 cyclic group Chemical group 0.000 claims 5
- IJGRMHOSHXDMSA-UHFFFAOYSA-N nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims 5
- OAICVXFJPJFONN-UHFFFAOYSA-N phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims 5
- 239000011574 phosphorus Substances 0.000 claims 5
- 150000003839 salts Chemical group 0.000 claims 5
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 4
- 229920000023 polynucleotide Polymers 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 229910052711 selenium Inorganic materials 0.000 claims 4
- 206010013801 Duchenne muscular dystrophy Diseases 0.000 claims 3
- 125000003835 nucleoside group Chemical group 0.000 claims 3
- 239000011780 sodium chloride Substances 0.000 claims 3
- HVYWMOMLDIMFJA-DPAQBDIFSA-N (3β)-Cholest-5-en-3-ol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims 2
- JCAIWDXKLCEQEO-LXOWHHAPSA-N Copalyl diphosphate Natural products [P@@](=O)(OP(=O)(O)O)(OC/C=C(\CC[C@H]1C(=C)CC[C@H]2C(C)(C)CCC[C@@]12C)/C)O JCAIWDXKLCEQEO-LXOWHHAPSA-N 0.000 claims 2
- 229920000665 Exon Polymers 0.000 claims 2
- 150000001875 compounds Chemical class 0.000 claims 2
- 238000010511 deprotection reaction Methods 0.000 claims 2
- OVBPIULPVIDEAO-LBPRGKRZSA-N folic acid Chemical compound C=1N=C2NC(N)=NC(=O)C2=NC=1CNC1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 OVBPIULPVIDEAO-LBPRGKRZSA-N 0.000 claims 2
- MYMOFIZGZYHOMD-UHFFFAOYSA-N oxygen Chemical compound O=O MYMOFIZGZYHOMD-UHFFFAOYSA-N 0.000 claims 2
- 239000001301 oxygen Substances 0.000 claims 2
- -1 phosphoramidite compound Chemical class 0.000 claims 2
- 229910052710 silicon Inorganic materials 0.000 claims 2
- 239000010703 silicon Substances 0.000 claims 2
- XUIMIQQOPSSXEZ-UHFFFAOYSA-N silicon Chemical compound [Si] XUIMIQQOPSSXEZ-UHFFFAOYSA-N 0.000 claims 2
- 239000007787 solid Substances 0.000 claims 2
- NINIDFKCEFEMDL-UHFFFAOYSA-N sulfur Chemical compound [S] NINIDFKCEFEMDL-UHFFFAOYSA-N 0.000 claims 2
- 239000011593 sulfur Substances 0.000 claims 2
- OQQOAWVKVDAJOI-UHFFFAOYSA-N (2-dodecanoyloxy-3-hydroxypropyl) dodecanoate Chemical compound CCCCCCCCCCCC(=O)OCC(CO)OC(=O)CCCCCCCCCCC OQQOAWVKVDAJOI-UHFFFAOYSA-N 0.000 claims 1
- PHIQHXFUZVPYII-ZCFIWIBFSA-N (R)-carnitine Chemical compound C[N+](C)(C)C[C@H](O)CC([O-])=O PHIQHXFUZVPYII-ZCFIWIBFSA-N 0.000 claims 1
- 229940107161 Cholesterol Drugs 0.000 claims 1
- WQZGKKKJIJFFOK-GASJEMHNSA-N D-Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-QTVWNMPRSA-N D-mannopyranose Chemical compound OC[C@H]1OC(O)[C@@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-QTVWNMPRSA-N 0.000 claims 1
- HPNMFZURTQLUMO-UHFFFAOYSA-N Diethylamine Chemical compound CCNCC HPNMFZURTQLUMO-UHFFFAOYSA-N 0.000 claims 1
- 229960000304 Folic Acid Drugs 0.000 claims 1
- 102000003746 Insulin Receptor Human genes 0.000 claims 1
- 108010001127 Insulin Receptor Proteins 0.000 claims 1
- 208000003627 Muscular Dystrophy Diseases 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 125000002947 alkylene group Chemical group 0.000 claims 1
- 150000001408 amides Chemical class 0.000 claims 1
- 150000001412 amines Chemical class 0.000 claims 1
- 150000001450 anions Chemical class 0.000 claims 1
- ZOXJGFHDIHLPTG-UHFFFAOYSA-N boron Chemical compound [B] ZOXJGFHDIHLPTG-UHFFFAOYSA-N 0.000 claims 1
- 229910052796 boron Inorganic materials 0.000 claims 1
- KXDHJXZQYSOELW-UHFFFAOYSA-M carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims 1
- NHYCGSASNAIGLD-UHFFFAOYSA-N chlorine monoxide Inorganic materials Cl[O] NHYCGSASNAIGLD-UHFFFAOYSA-N 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 230000001808 coupling Effects 0.000 claims 1
- 238000010168 coupling process Methods 0.000 claims 1
- 238000005859 coupling reaction Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000006575 electron-withdrawing group Chemical group 0.000 claims 1
- 150000002148 esters Chemical class 0.000 claims 1
- 235000019152 folic acid Nutrition 0.000 claims 1
- 239000011724 folic acid Substances 0.000 claims 1
- 239000008103 glucose Substances 0.000 claims 1
- 150000002357 guanidines Chemical group 0.000 claims 1
- 239000003446 ligand Substances 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023110962A JP2023139036A (ja) | 2018-04-12 | 2023-07-05 | オリゴヌクレオチド組成物及びその使用方法 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862656949P | 2018-04-12 | 2018-04-12 | |
| US62/656,949 | 2018-04-12 | ||
| US201862670709P | 2018-05-11 | 2018-05-11 | |
| US62/670,709 | 2018-05-11 | ||
| US201862715684P | 2018-08-07 | 2018-08-07 | |
| US62/715,684 | 2018-08-07 | ||
| US201862723375P | 2018-08-27 | 2018-08-27 | |
| US62/723,375 | 2018-08-27 | ||
| US201862776432P | 2018-12-06 | 2018-12-06 | |
| US62/776,432 | 2018-12-06 | ||
| PCT/US2019/027109 WO2019200185A1 (en) | 2018-04-12 | 2019-04-11 | Oligonucleotide compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023110962A Division JP2023139036A (ja) | 2018-04-12 | 2023-07-05 | オリゴヌクレオチド組成物及びその使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021521140A JP2021521140A (ja) | 2021-08-26 |
| JPWO2019200185A5 true JPWO2019200185A5 (pt) | 2022-04-19 |
Family
ID=68163789
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020555234A Pending JP2021521140A (ja) | 2018-04-12 | 2019-04-11 | オリゴヌクレオチド組成物及びその使用方法 |
| JP2023110962A Pending JP2023139036A (ja) | 2018-04-12 | 2023-07-05 | オリゴヌクレオチド組成物及びその使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023110962A Pending JP2023139036A (ja) | 2018-04-12 | 2023-07-05 | オリゴヌクレオチド組成物及びその使用方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220306573A1 (pt) |
| EP (1) | EP3775203A4 (pt) |
| JP (2) | JP2021521140A (pt) |
| KR (1) | KR20210005057A (pt) |
| CN (1) | CN112004928A (pt) |
| AU (1) | AU2019252680A1 (pt) |
| BR (1) | BR112020020670A2 (pt) |
| CA (1) | CA3096667A1 (pt) |
| IL (1) | IL277889A (pt) |
| MX (1) | MX2020010687A (pt) |
| SG (1) | SG11202009877XA (pt) |
| WO (1) | WO2019200185A1 (pt) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4219516A3 (en) | 2012-07-13 | 2024-01-10 | Wave Life Sciences Ltd. | Chiral control |
| US9598458B2 (en) | 2012-07-13 | 2017-03-21 | Wave Life Sciences Japan, Inc. | Asymmetric auxiliary group |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| CN114685589A (zh) | 2016-03-13 | 2022-07-01 | 波涛生命科学有限公司 | 用于亚磷酰胺和寡核苷酸合成的组合物和方法 |
| EP3463386A4 (en) | 2016-06-03 | 2020-03-04 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDES, COMPOSITIONS AND METHOD THEREFOR |
| MA46905A (fr) | 2016-11-23 | 2019-10-02 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides |
| CN110997692A (zh) | 2017-06-02 | 2020-04-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| JP2020522510A (ja) | 2017-06-02 | 2020-07-30 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物及びその使用方法 |
| JP7402696B2 (ja) | 2017-06-21 | 2023-12-21 | ウェイブ ライフ サイエンシズ リミテッド | 合成のための化合物、組成物、及び方法 |
| AR112779A1 (es) | 2017-08-08 | 2019-12-11 | Wave Life Sciences Ltd | Composiciones de nucleótidos y métodos relacionados |
| EP3684755A4 (en) | 2017-09-18 | 2022-03-02 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE PREPARATION TECHNOLOGIES |
| US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| CN112955153A (zh) | 2018-08-02 | 2021-06-11 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗肌养蛋白病的用途 |
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| AU2020241559A1 (en) * | 2019-03-20 | 2021-11-11 | Wave Life Sciences Ltd. | Technologies useful for oligonucleotide preparation |
| KR20220062517A (ko) | 2019-08-15 | 2022-05-17 | 아이오니스 파마수티컬즈, 인코포레이티드 | 결합 변형된 올리고머 화합물 및 이의 용도 |
| AU2020408773A1 (en) * | 2019-12-19 | 2022-07-14 | National Center Of Neurology And Psychiatry | Antisense nucleic acid enabling exon skipping |
| US20230203484A1 (en) | 2020-05-22 | 2023-06-29 | Wave Life Sciences Ltd. | Double stranded oligonucleotide compositions and methods relating thereto |
| CN112430645A (zh) * | 2020-12-09 | 2021-03-02 | 北京华瑞康源生物科技发展有限公司 | 一种多重实时荧光pcr法检测人dmd基因拷贝数的相对定量方法及试剂盒 |
| WO2022270585A1 (ja) | 2021-06-23 | 2022-12-29 | 日本新薬株式会社 | アンチセンスオリゴマーの組み合わせ |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CA3226457A1 (en) * | 2021-07-30 | 2023-02-02 | Amy WALKER | Nanoparticles and peptides for the delivery of cargos to muscle cells |
| EP4124345A1 (en) * | 2021-07-30 | 2023-02-01 | 4basebio UK Ltd | Nanoparticles for muscle delivery |
| WO2023014938A1 (en) * | 2021-08-05 | 2023-02-09 | Sanegene Bio Usa Inc. | 1'-alkyl modified ribose derivatives and methods of use |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2023168014A2 (en) * | 2022-03-02 | 2023-09-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof for exon skipping |
| CN114600803B (zh) * | 2022-03-15 | 2023-05-09 | 北京协和建昊医药技术开发有限责任公司 | 一种斑马鱼cyp3a4代谢酶抑制模型、建立方法及应用 |
| 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 (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5852188A (en) * | 1990-01-11 | 1998-12-22 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
| ES2099718T3 (es) * | 1990-07-02 | 1997-06-01 | Hoechst Ag | Analogos de oligonucleotidos con uniones internucleotidicas de 3'-3' o 5'-5' terminales. |
| US5512668A (en) * | 1991-03-06 | 1996-04-30 | Polish Academy Of Sciences | Solid phase oligonucleotide synthesis using phospholane intermediates |
| US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
| AR040996A1 (es) * | 2002-08-19 | 2005-04-27 | Coley Pharm Group Inc | Acidos nucleicos inmunoestimuladores |
| EP2206781B1 (en) * | 2004-06-28 | 2015-12-02 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| JP2006248949A (ja) * | 2005-03-09 | 2006-09-21 | Univ Nagoya | ヌクレオシド誘導体、ヌクレオチド誘導体及びそれらの製造方法 |
| ES2639852T3 (es) * | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| KR101881596B1 (ko) * | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| CN110257379B (zh) * | 2012-07-03 | 2023-08-11 | 马林生物科技有限公司 | 用于治疗肌肉萎缩症患者的寡核苷酸 |
| EP4219516A3 (en) * | 2012-07-13 | 2024-01-10 | Wave Life Sciences Ltd. | Chiral control |
| BR112015000723A2 (pt) * | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
| BR112016016400A2 (pt) * | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| RU2708237C2 (ru) * | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| MA43072A (fr) * | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| TW201722439A (zh) * | 2015-10-09 | 2017-07-01 | 波濤生命科學有限公司 | 寡核苷酸組合物及其方法 |
| EP3463386A4 (en) * | 2016-06-03 | 2020-03-04 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDES, COMPOSITIONS AND METHOD THEREFOR |
-
2019
- 2019-04-11 AU AU2019252680A patent/AU2019252680A1/en active Pending
- 2019-04-11 US US17/046,752 patent/US20220306573A1/en active Pending
- 2019-04-11 KR KR1020207032342A patent/KR20210005057A/ko unknown
- 2019-04-11 EP EP19784832.8A patent/EP3775203A4/en active Pending
- 2019-04-11 CN CN201980025941.1A patent/CN112004928A/zh active Pending
- 2019-04-11 SG SG11202009877XA patent/SG11202009877XA/en unknown
- 2019-04-11 BR BR112020020670-9A patent/BR112020020670A2/pt unknown
- 2019-04-11 MX MX2020010687A patent/MX2020010687A/es unknown
- 2019-04-11 CA CA3096667A patent/CA3096667A1/en active Pending
- 2019-04-11 JP JP2020555234A patent/JP2021521140A/ja active Pending
- 2019-04-11 WO PCT/US2019/027109 patent/WO2019200185A1/en active Application Filing
-
2020
- 2020-10-08 IL IL277889A patent/IL277889A/en unknown
-
2023
- 2023-07-05 JP JP2023110962A patent/JP2023139036A/ja active Pending
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