JP2017528441A5 - - Google Patents
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- Publication number
- JP2017528441A5 JP2017528441A5 JP2017506382A JP2017506382A JP2017528441A5 JP 2017528441 A5 JP2017528441 A5 JP 2017528441A5 JP 2017506382 A JP2017506382 A JP 2017506382A JP 2017506382 A JP2017506382 A JP 2017506382A JP 2017528441 A5 JP2017528441 A5 JP 2017528441A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligonucleotide
- inhibitor
- sequence
- nucleotides
- seq
- 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.)
- Granted
Links
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 112
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 112
- 108091034117 Oligonucleotide Proteins 0.000 claims description 110
- 239000003112 inhibitor Substances 0.000 claims description 74
- 125000003729 nucleotide group Chemical group 0.000 claims description 35
- 239000002773 nucleotide Substances 0.000 claims description 27
- 230000000295 complement effect Effects 0.000 claims description 13
- 101000958744 Homo sapiens Myosin-7B Proteins 0.000 claims description 12
- 102100038322 Myosin-7B Human genes 0.000 claims description 12
- 125000001921 locked nucleotide group Chemical group 0.000 claims description 12
- 208000006029 Cardiomegaly Diseases 0.000 claims description 10
- 238000012986 modification Methods 0.000 claims description 10
- 230000004048 modification Effects 0.000 claims description 10
- 230000001575 pathological effect Effects 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 10
- 206010007572 Cardiac hypertrophy Diseases 0.000 claims description 8
- 206010019280 Heart failures Diseases 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 6
- 208000010125 myocardial infarction Diseases 0.000 claims description 6
- 229940124597 therapeutic agent Drugs 0.000 claims description 6
- 108091028664 Ribonucleotide Proteins 0.000 claims description 4
- 239000005547 deoxyribonucleotide Substances 0.000 claims description 4
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 4
- 108091056170 miR-499 stem-loop Proteins 0.000 claims description 4
- 108091050885 miR-499-1 stem-loop Proteins 0.000 claims description 4
- 108091038523 miR-499-2 stem-loop Proteins 0.000 claims description 4
- 239000002336 ribonucleotide Substances 0.000 claims description 4
- 125000002652 ribonucleotide group Chemical group 0.000 claims description 4
- 108010051583 Ventricular Myosins Proteins 0.000 claims description 3
- -1 miR-15a Proteins 0.000 claims description 3
- 108091031326 miR-15b stem-loop Proteins 0.000 claims description 3
- 108091027943 miR-16 stem-loop Proteins 0.000 claims description 3
- 108091071651 miR-208 stem-loop Proteins 0.000 claims description 3
- 108091084446 miR-208a stem-loop Proteins 0.000 claims description 3
- 108091062547 miR-208a-1 stem-loop Proteins 0.000 claims description 3
- 108091055375 miR-208a-2 stem-loop Proteins 0.000 claims description 3
- 108091047268 miR-208b stem-loop Proteins 0.000 claims description 3
- 230000002265 prevention Effects 0.000 claims description 3
- 108090000623 proteins and genes Proteins 0.000 claims description 3
- KPPPLADORXGUFI-KCRXGDJASA-N 4-amino-1-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(1-hydroxyethyl)oxolan-2-yl]pyrimidin-2-one Chemical group O[C@@H]1[C@H](O)[C@@H](C(O)C)O[C@H]1N1C(=O)N=C(N)C=C1 KPPPLADORXGUFI-KCRXGDJASA-N 0.000 claims description 2
- 239000005977 Ethylene Substances 0.000 claims description 2
- 125000001475 halogen functional group Chemical group 0.000 claims description 2
- 206010020871 hypertrophic cardiomyopathy Diseases 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- DEFLNOSTNCSZRB-IDTAVKCVSA-N 9-[(2r,3r,4r,5r)-3,4-dimethoxy-5-(methoxymethyl)oxolan-2-yl]-n-methoxypurin-6-amine Chemical compound CO[C@@H]1[C@H](OC)[C@@H](COC)O[C@H]1N1C2=NC=NC(NOC)=C2N=C1 DEFLNOSTNCSZRB-IDTAVKCVSA-N 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 238000000034 method Methods 0.000 description 16
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 10
- 239000004055 small Interfering RNA Substances 0.000 description 6
- 108091027967 Small hairpin RNA Proteins 0.000 description 5
- 108020004459 Small interfering RNA Proteins 0.000 description 5
- 210000002064 heart cell Anatomy 0.000 description 3
- 108020004707 nucleic acids Proteins 0.000 description 3
- 102000039446 nucleic acids Human genes 0.000 description 3
- 150000007523 nucleic acids Chemical class 0.000 description 3
- 108020000948 Antisense Oligonucleotides Proteins 0.000 description 2
- 102000005604 Myosin Heavy Chains Human genes 0.000 description 2
- 108091093037 Peptide nucleic acid Proteins 0.000 description 2
- 230000000747 cardiac effect Effects 0.000 description 2
- 108091023037 Aptamer Proteins 0.000 description 1
- 108090000994 Catalytic RNA Proteins 0.000 description 1
- 102000053642 Catalytic RNA Human genes 0.000 description 1
- 101100025408 Mus musculus Myh7b gene Proteins 0.000 description 1
- 230000007423 decrease Effects 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 108091085564 miR-25 stem-loop Proteins 0.000 description 1
- 108091080167 miR-25-1 stem-loop Proteins 0.000 description 1
- 108091083056 miR-25-2 stem-loop Proteins 0.000 description 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 description 1
- ZJAOAACCNHFJAH-UHFFFAOYSA-N phosphonoformic acid Chemical compound OC(=O)P(O)(O)=O ZJAOAACCNHFJAH-UHFFFAOYSA-N 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 108091092562 ribozyme Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462033018P | 2014-08-04 | 2014-08-04 | |
| US62/033,018 | 2014-08-04 | ||
| PCT/US2015/043571 WO2016022536A2 (en) | 2014-08-04 | 2015-08-04 | Inhibitors of myh7b and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017528441A JP2017528441A (ja) | 2017-09-28 |
| JP2017528441A5 true JP2017528441A5 (enExample) | 2019-12-05 |
| JP6637961B2 JP6637961B2 (ja) | 2020-01-29 |
Family
ID=55179393
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017506382A Expired - Fee Related JP6637961B2 (ja) | 2014-08-04 | 2015-08-04 | Ｍｙｈ７ｂの阻害剤およびその使用 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US9752144B2 (enExample) |
| EP (1) | EP3177327A4 (enExample) |
| JP (1) | JP6637961B2 (enExample) |
| KR (1) | KR20170042625A (enExample) |
| CN (1) | CN108064175A (enExample) |
| AR (1) | AR101449A1 (enExample) |
| AU (1) | AU2015301221B2 (enExample) |
| CA (1) | CA2957618A1 (enExample) |
| MX (1) | MX2017001674A (enExample) |
| TW (1) | TW201614069A (enExample) |
| WO (1) | WO2016022536A2 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6637961B2 (ja) | 2014-08-04 | 2020-01-29 | ミラゲン セラピューティクス， インコーポレイテッド | Ｍｙｈ７ｂの阻害剤およびその使用 |
| US11198874B2 (en) * | 2014-08-20 | 2021-12-14 | Lifesplice Pharma Llc | SCN8A splice modulating oligonucleotides and methods of use thereof |
| WO2017201422A1 (en) * | 2016-05-20 | 2017-11-23 | The General Hospital Corporation | Using micrornas to control activation status of hepatic stellate cells and to prevent fibrosis in progressive liver diseases |
| JP2019535839A (ja) | 2016-11-29 | 2019-12-12 | ピュアテック ヘルス エルエルシー | 治療剤の送達のためのエクソソーム |
| EP3728562A4 (en) * | 2017-12-19 | 2021-09-29 | The Regents of The University of Michigan | HEART MICROTISSUE AND ASSOCIATED USES |
| WO2020002691A1 (en) * | 2018-06-28 | 2020-01-02 | United Kingdom Research And Innovation | TREATMENT OF CARDIOMYOPATHY THROUGH MODULATION OF HYPOXIA-INDUCED eRNA ACTIVITY |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| CN112912499A (zh) | 2018-08-02 | 2021-06-04 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 |
| WO2020028842A1 (en) * | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
Family Cites Families (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5270163A (en) | 1990-06-11 | 1993-12-14 | University Research Corporation | Methods for identifying nucleic acid ligands |
| US7517644B1 (en) * | 1991-08-23 | 2009-04-14 | Larry J. Smith | Method and compositions for cellular reprogramming |
| US5981505A (en) | 1993-01-26 | 1999-11-09 | The Trustees Of The University Of Pennsylvania | Compositions and methods for delivery of genetic material |
| AU7404994A (en) | 1993-07-30 | 1995-02-28 | Regents Of The University Of California, The | Endocardial infusion catheter |
| US5837533A (en) | 1994-09-28 | 1998-11-17 | American Home Products Corporation | Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent |
| US5840710A (en) | 1994-12-09 | 1998-11-24 | Genzyme Corporation | Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules |
| US6639062B2 (en) * | 1997-02-14 | 2003-10-28 | Isis Pharmaceuticals, Inc. | Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom |
| US6416510B1 (en) | 1997-03-13 | 2002-07-09 | Biocardia, Inc. | Drug delivery catheters that attach to tissue and methods for their use |
| US6217900B1 (en) | 1997-04-30 | 2001-04-17 | American Home Products Corporation | Vesicular complexes and methods of making and using the same |
| CA2294988C (en) | 1997-07-01 | 2015-11-24 | Isis Pharmaceuticals Inc. | Compositions and methods for the delivery of oligonucleotides via the alimentary canal |
| US6749617B1 (en) | 1997-11-04 | 2004-06-15 | Scimed Life Systems, Inc. | Catheter and implants for the delivery of therapeutic agents to tissues |
| JP2002516256A (ja) | 1998-05-26 | 2002-06-04 | アイ・シー・エヌ・フアーマシユーテイカルズ・インコーポレイテツド | 二環式糖成分を有する新規ヌクレオシド |
| US6838283B2 (en) | 1998-09-29 | 2005-01-04 | Isis Pharmaceuticals Inc. | Antisense modulation of survivin expression |
| US6716242B1 (en) | 1999-10-13 | 2004-04-06 | Peter A. Altman | Pulmonary vein stent and method for use |
| US6693187B1 (en) | 2000-10-17 | 2004-02-17 | Lievre Cornu Llc | Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge |
| US7083642B2 (en) | 2000-12-22 | 2006-08-01 | Avantec Vascular Corporation | Delivery of therapeutic capable agents |
| US7247313B2 (en) | 2001-06-27 | 2007-07-24 | Advanced Cardiovascular Systems, Inc. | Polyacrylates coatings for implantable medical devices |
| US6589286B1 (en) | 2001-09-12 | 2003-07-08 | Jason Litner | Eustachian tube stent |
| US7236821B2 (en) | 2002-02-19 | 2007-06-26 | Cardiac Pacemakers, Inc. | Chronically-implanted device for sensing and therapy |
| EP1549352A4 (en) | 2002-05-06 | 2005-07-27 | Nucleonics Inc | METHOD OF DISTRIBUTING NUCLEIC ACIDS |
| WO2004004602A1 (en) | 2002-07-08 | 2004-01-15 | Abbott Laboratories Vascular Enterprises Limited | Drug eluting stent and methods of manufacture |
| US7294329B1 (en) | 2002-07-18 | 2007-11-13 | Advanced Cardiovascular Systems, Inc. | Poly(vinyl acetal) coatings for implantable medical devices |
| US7232573B1 (en) | 2002-09-26 | 2007-06-19 | Advanced Cardiovascular Systems, Inc. | Stent coatings containing self-assembled monolayers |
| US7087263B2 (en) | 2002-10-09 | 2006-08-08 | Advanced Cardiovascular Systems, Inc. | Rare limiting barriers for implantable medical devices |
| US7144422B1 (en) | 2002-11-13 | 2006-12-05 | Advanced Cardiovascular Systems, Inc. | Drug-eluting stent and methods of making the same |
| EP2213738B1 (en) * | 2002-11-14 | 2012-10-10 | Dharmacon, Inc. | siRNA molecules targeting Bcl-2 |
| US7105018B1 (en) | 2002-12-30 | 2006-09-12 | Advanced Cardiovascular Systems, Inc. | Drug-eluting stent cover and method of use |
| US7156869B1 (en) | 2003-01-27 | 2007-01-02 | Advanced Cardiovascular Systems, Inc. | Drug-eluting stent and delivery system with tapered stent in shoulder region |
| US7041127B2 (en) | 2003-05-28 | 2006-05-09 | Ledergerber Walter J | Textured and drug eluting coronary artery stent |
| US7055237B2 (en) | 2003-09-29 | 2006-06-06 | Medtronic Vascular, Inc. | Method of forming a drug eluting stent |
| US20060148742A1 (en) | 2004-02-26 | 2006-07-06 | Kaye David M | Polynucleotide delivery to cardiac tissue |
| EP1735037B1 (en) | 2004-02-26 | 2015-05-20 | Osprey Medical Inc. | Isolating cardiac circulation |
| US20070203445A1 (en) | 2004-02-26 | 2007-08-30 | V-Kardia Pty Ltd | Isolating cardiac circulation |
| US7722596B2 (en) | 2004-02-26 | 2010-05-25 | Osprey Medical, Inc. | Regional cardiac tissue treatment |
| US7842800B2 (en) * | 2004-04-02 | 2010-11-30 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory bacterial and bacterial associated oligonucleotides and uses thereof |
| WO2007035532A2 (en) | 2005-09-15 | 2007-03-29 | Duke University | Focused libraries, functional profiling, laser selex and deselex |
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| MX2009006310A (es) * | 2006-12-14 | 2009-07-22 | Novartis Ag | Composiciones y metodos para tratar trastornos musculares y cardiovasculares. |
| AU2008283794B2 (en) * | 2007-07-31 | 2014-03-20 | Board Of Regents, The University Of Texas System | Micro-RNAs that control myosin expression and myofiber identity |
| MY156951A (en) * | 2007-10-04 | 2016-04-15 | Santaris Pharma As | Micromirs |
| WO2010091204A1 (en) * | 2009-02-04 | 2010-08-12 | Board Of Regents, The University Of Texas System | Dual targeting of mir-208 and mir-499 in the treatment of cardiac disorders |
| CA2760687A1 (en) * | 2009-05-01 | 2010-11-04 | Ophthotech Corporation | Methods for treating or preventing ophthalmological diseases |
| WO2011149354A1 (en) * | 2010-05-28 | 2011-12-01 | Vereniging Voor Christelijk Hoger Onderwijs, Wetenschappelijk Onderzoek En Patiëntenzorg | Mirnas involved in the blood brain barrier function |
| MX2013005875A (es) * | 2010-12-15 | 2014-04-30 | Miragen Therapeutics | Inhibidores de microarn que comprenden nucleotidos bloqueados. |
| WO2012140234A1 (en) | 2011-04-13 | 2012-10-18 | Vib Vzw | Modulation of mirna in diseases with aberrant angiogenesis |
| CN103814132B (zh) * | 2011-09-20 | 2018-06-05 | 苏州瑞博生物技术有限公司 | Gcgr表达的反义调节 |
| AU2013201303C1 (en) * | 2011-10-06 | 2016-06-23 | MiRagen Therapeutics, Inc. | Control of whole body energy homeostasis by microRNA regulation |
| US9034839B2 (en) | 2012-04-20 | 2015-05-19 | Aptamir Therapeutics, Inc. | miRNA modulators of thermogenesis |
| US20150211005A1 (en) * | 2012-04-20 | 2015-07-30 | Aptamir Therapeutics, Inc. | Mirna modulators of thermogenesis |
| CA2873772A1 (en) * | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating bdnf expression |
| KR101928418B1 (ko) | 2012-06-21 | 2018-12-12 | 미라젠 세러퓨틱스 인코포레이티드 | 잠금 핵산 모티프를 포함하는 올리고뉴클레오티드-기반 억제제 |
| US9163235B2 (en) | 2012-06-21 | 2015-10-20 | MiRagen Therapeutics, Inc. | Inhibitors of the miR-15 family of micro-RNAs |
| JP6637961B2 (ja) | 2014-08-04 | 2020-01-29 | ミラゲン セラピューティクス， インコーポレイテッド | Ｍｙｈ７ｂの阻害剤およびその使用 |
-
2015
- 2015-08-04 JP JP2017506382A patent/JP6637961B2/ja not_active Expired - Fee Related
- 2015-08-04 KR KR1020177005777A patent/KR20170042625A/ko not_active Withdrawn
- 2015-08-04 AU AU2015301221A patent/AU2015301221B2/en not_active Ceased
- 2015-08-04 TW TW104125326A patent/TW201614069A/zh unknown
- 2015-08-04 EP EP15829559.2A patent/EP3177327A4/en not_active Withdrawn
- 2015-08-04 CN CN201580052358.1A patent/CN108064175A/zh active Pending
- 2015-08-04 US US14/818,144 patent/US9752144B2/en not_active Expired - Fee Related
- 2015-08-04 MX MX2017001674A patent/MX2017001674A/es unknown
- 2015-08-04 WO PCT/US2015/043571 patent/WO2016022536A2/en not_active Ceased
- 2015-08-04 AR ARP150102501A patent/AR101449A1/es unknown
- 2015-08-04 CA CA2957618A patent/CA2957618A1/en not_active Abandoned
-
2017
- 2017-08-24 US US15/685,983 patent/US10144930B2/en not_active Expired - Fee Related
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