JP2015505839A5 - - Google Patents
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- JP2015505839A5 JP2015505839A5 JP2014546150A JP2014546150A JP2015505839A5 JP 2015505839 A5 JP2015505839 A5 JP 2015505839A5 JP 2014546150 A JP2014546150 A JP 2014546150A JP 2014546150 A JP2014546150 A JP 2014546150A JP 2015505839 A5 JP2015505839 A5 JP 2015505839A5
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- JP
- Japan
- Prior art keywords
- seq
- targeting sequence
- pharmaceutically acceptable
- acceptable salt
- compound according
- 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.)
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- 230000008685 targeting Effects 0.000 claims description 93
- 150000003839 salts Chemical class 0.000 claims description 65
- 108091034117 Oligonucleotide Proteins 0.000 claims description 61
- 150000001875 compounds Chemical class 0.000 claims description 61
- 229910052739 hydrogen Inorganic materials 0.000 claims description 51
- 239000001257 hydrogen Substances 0.000 claims description 50
- 125000000217 alkyl group Chemical group 0.000 claims description 41
- 101001003584 Homo sapiens Prelamin-A/C Proteins 0.000 claims description 29
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 26
- 150000002431 hydrogen Chemical class 0.000 claims description 24
- 125000002091 cationic group Chemical group 0.000 claims description 20
- 108020004999 messenger RNA Proteins 0.000 claims description 20
- 230000000295 complement effect Effects 0.000 claims description 19
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 17
- 102000057754 human LMNA Human genes 0.000 claims description 16
- 239000000203 mixture Substances 0.000 claims description 16
- 102100026531 Prelamin-A/C Human genes 0.000 claims description 13
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 11
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 claims description 10
- 125000003118 aryl group Chemical group 0.000 claims description 9
- 229910052760 oxygen Inorganic materials 0.000 claims description 9
- 229910052717 sulfur Inorganic materials 0.000 claims description 9
- 125000003545 alkoxy group Chemical group 0.000 claims description 8
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 8
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 8
- 125000000623 heterocyclic group Chemical group 0.000 claims description 8
- 238000011144 upstream manufacturing Methods 0.000 claims description 8
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims description 7
- 229910052799 carbon Inorganic materials 0.000 claims description 7
- -1 4-amino-piperidinylmethyl Chemical group 0.000 claims description 6
- 125000003282 alkyl amino group Chemical group 0.000 claims description 6
- 150000001413 amino acids Chemical class 0.000 claims description 6
- 125000004429 atom Chemical group 0.000 claims description 6
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 6
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 5
- 125000002618 bicyclic heterocycle group Chemical group 0.000 claims description 5
- 125000002950 monocyclic group Chemical group 0.000 claims description 5
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 5
- 239000004475 Arginine Substances 0.000 claims description 4
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 4
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 4
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 claims description 4
- 230000001594 aberrant effect Effects 0.000 claims description 4
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 4
- 125000004103 aminoalkyl group Chemical group 0.000 claims description 4
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 4
- 125000001072 heteroaryl group Chemical group 0.000 claims description 4
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims description 3
- 230000008878 coupling Effects 0.000 claims description 3
- 238000010168 coupling process Methods 0.000 claims description 3
- 238000005859 coupling reaction Methods 0.000 claims description 3
- 229910052698 phosphorus Inorganic materials 0.000 claims description 3
- 239000011574 phosphorus Substances 0.000 claims description 3
- 125000004642 (C1-C12) alkoxy group Chemical group 0.000 claims description 2
- MHCMWGPPLOSCJY-UHFFFAOYSA-N 4-$l^{1}-azanylmorpholine Chemical compound [N]N1CCOCC1 MHCMWGPPLOSCJY-UHFFFAOYSA-N 0.000 claims description 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims description 2
- 239000004215 Carbon black (E152) Substances 0.000 claims description 2
- 108700024394 Exon Proteins 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 125000000747 amidyl group Chemical group [H][N-]* 0.000 claims description 2
- 229940099352 cholate Drugs 0.000 claims description 2
- BHQCQFFYRZLCQQ-OELDTZBJSA-N cholic acid Chemical compound C([C@H]1C[C@H]2O)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 BHQCQFFYRZLCQQ-OELDTZBJSA-N 0.000 claims description 2
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 2
- 229930195733 hydrocarbon Natural products 0.000 claims description 2
- 150000002430 hydrocarbons Chemical class 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 108020004707 nucleic acids Proteins 0.000 claims description 2
- 150000007523 nucleic acids Chemical class 0.000 claims description 2
- 102000039446 nucleic acids Human genes 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 125000004043 oxo group Chemical group O=* 0.000 claims description 2
- 125000002023 trifluoromethyl group Chemical group FC(F)(F)* 0.000 claims description 2
- KDCGOANMDULRCW-UHFFFAOYSA-N 7H-purine Chemical compound N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 0.000 claims 4
- CZPWVGJYEJSRLH-UHFFFAOYSA-N Pyrimidine Chemical compound C1=CN=CN=C1 CZPWVGJYEJSRLH-UHFFFAOYSA-N 0.000 claims 2
- 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 2
- 244000007835 Cyamopsis tetragonoloba Species 0.000 claims 1
- 102000002067 Protein Subunits Human genes 0.000 claims 1
- 108010001267 Protein Subunits Proteins 0.000 claims 1
- 125000004183 alkoxy alkyl group Chemical group 0.000 description 1
- 230000000692 anti-sense effect Effects 0.000 description 1
- 229940009976 deoxycholate Drugs 0.000 description 1
- KXGVEGMKQFWNSR-LLQZFEROSA-N deoxycholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 KXGVEGMKQFWNSR-LLQZFEROSA-N 0.000 description 1
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 description 1
- 238000000034 method Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161568590P | 2011-12-08 | 2011-12-08 | |
| US61/568,590 | 2011-12-08 | ||
| PCT/US2012/068606 WO2013086441A2 (en) | 2011-12-08 | 2012-12-07 | Oligonucleotide analogues targeting human lmna |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017041741A Division JP6596032B2 (ja) | 2011-12-08 | 2017-03-06 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015505839A JP2015505839A (ja) | 2015-02-26 |
| JP2015505839A5 true JP2015505839A5 (enExample) | 2016-01-28 |
| JP6132848B2 JP6132848B2 (ja) | 2017-05-31 |
Family
ID=47429038
Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014546152A Expired - Fee Related JP6132849B2 (ja) | 2011-12-08 | 2012-12-07 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体を使用する早老性ラミノパシーを処置するための方法 |
| JP2014546150A Active JP6132848B2 (ja) | 2011-12-08 | 2012-12-07 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
| JP2017041741A Active JP6596032B2 (ja) | 2011-12-08 | 2017-03-06 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
| JP2017041744A Expired - Fee Related JP6581132B2 (ja) | 2011-12-08 | 2017-03-06 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体を使用する早老性ラミノパシーを処置するための方法 |
| JP2019077698A Pending JP2019129848A (ja) | 2011-12-08 | 2019-04-16 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
| JP2019109410A Pending JP2019172691A (ja) | 2011-12-08 | 2019-06-12 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体を使用する早老性ラミノパシーを処置するための方法 |
| JP2021065398A Active JP7277503B2 (ja) | 2011-12-08 | 2021-04-07 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014546152A Expired - Fee Related JP6132849B2 (ja) | 2011-12-08 | 2012-12-07 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体を使用する早老性ラミノパシーを処置するための方法 |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017041741A Active JP6596032B2 (ja) | 2011-12-08 | 2017-03-06 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
| JP2017041744A Expired - Fee Related JP6581132B2 (ja) | 2011-12-08 | 2017-03-06 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体を使用する早老性ラミノパシーを処置するための方法 |
| JP2019077698A Pending JP2019129848A (ja) | 2011-12-08 | 2019-04-16 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
| JP2019109410A Pending JP2019172691A (ja) | 2011-12-08 | 2019-06-12 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体を使用する早老性ラミノパシーを処置するための方法 |
| JP2021065398A Active JP7277503B2 (ja) | 2011-12-08 | 2021-04-07 | ヒトｌｍｎａを標的とするオリゴヌクレオチド類似体 |
Country Status (16)
| Country | Link |
|---|---|
| US (7) | US9326992B2 (enExample) |
| EP (3) | EP2788487B1 (enExample) |
| JP (7) | JP6132849B2 (enExample) |
| CY (1) | CY1120495T1 (enExample) |
| DK (1) | DK2788487T3 (enExample) |
| ES (2) | ES2674929T3 (enExample) |
| HR (1) | HRP20181022T1 (enExample) |
| HU (1) | HUE038369T2 (enExample) |
| LT (1) | LT2788487T (enExample) |
| PL (1) | PL2788487T3 (enExample) |
| PT (1) | PT2788487T (enExample) |
| RS (1) | RS57467B1 (enExample) |
| SI (1) | SI2788487T1 (enExample) |
| SM (1) | SMT201800337T1 (enExample) |
| TR (1) | TR201809173T4 (enExample) |
| WO (2) | WO2013086441A2 (enExample) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| KR102182663B1 (ko) | 2010-05-28 | 2020-11-25 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US10017763B2 (en) | 2010-09-03 | 2018-07-10 | Sarepta Therapeutics, Inc. | dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups |
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| JP2015500204A (ja) | 2011-11-18 | 2015-01-05 | サレプタ セラピューティクス， インコーポレイテッド | 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット |
| WO2013086441A2 (en) | 2011-12-08 | 2013-06-13 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| KR102275504B1 (ko) * | 2013-09-05 | 2021-07-09 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
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| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CN114681621B (zh) * | 2015-05-19 | 2024-08-06 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
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| WO2017190041A1 (en) * | 2016-04-29 | 2017-11-02 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
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| CN110536964A (zh) * | 2017-03-10 | 2019-12-03 | 国立研究开发法人国立成育医疗研究中心 | 反义寡核苷酸和糖原贮积病Ia型预防或治疗用组合物 |
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| JP7246729B2 (ja) * | 2017-05-18 | 2023-03-28 | 国立大学法人京都大学 | 脊髄小脳変性症３６型の予防又は治療用組成物 |
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| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3763815A4 (en) | 2018-03-09 | 2021-12-15 | Daiichi Sankyo Company, Limited | THERAPEUTIC FOR GLYCOGEN STORAGE DISEASE OF TYPE IA |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
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| US20240173430A1 (en) * | 2018-09-05 | 2024-05-30 | The Broad Institute, Inc. | Base editing for treating hutchinson-gilford progeria syndrome |
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| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
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| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| JP7795914B2 (ja) * | 2019-06-03 | 2026-01-08 | クラリス コーポレーション | 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法 |
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