JP2016530882A5 - - Google Patents
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- JP2016530882A5 JP2016530882A5 JP2016533487A JP2016533487A JP2016530882A5 JP 2016530882 A5 JP2016530882 A5 JP 2016530882A5 JP 2016533487 A JP2016533487 A JP 2016533487A JP 2016533487 A JP2016533487 A JP 2016533487A JP 2016530882 A5 JP2016530882 A5 JP 2016530882A5
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- Prior art keywords
- wing segment
- linked nucleosides
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- compound according
- segment
- 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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- 150000001875 compounds Chemical class 0.000 claims description 198
- 239000002777 nucleoside Substances 0.000 claims description 145
- 125000003835 nucleoside group Chemical group 0.000 claims description 118
- 235000000346 sugar Nutrition 0.000 claims description 82
- 108091034117 Oligonucleotide Proteins 0.000 claims description 80
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 claims description 35
- 241001465754 Metazoa Species 0.000 claims description 27
- 230000000295 complement effect Effects 0.000 claims description 27
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 27
- 125000002619 bicyclic group Chemical group 0.000 claims description 26
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 19
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 17
- 239000000203 mixture Substances 0.000 claims description 17
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 15
- 150000007523 nucleic acids Chemical class 0.000 claims description 13
- 230000004048 modification Effects 0.000 claims description 12
- 238000012986 modification Methods 0.000 claims description 12
- 108020004707 nucleic acids Proteins 0.000 claims description 12
- 102000039446 nucleic acids Human genes 0.000 claims description 12
- 239000008194 pharmaceutical composition Substances 0.000 claims description 9
- 150000008163 sugars Chemical class 0.000 claims description 6
- 102100022437 Myotonin-protein kinase Human genes 0.000 claims description 4
- 229940104302 cytosine Drugs 0.000 claims description 3
- ZIEXYIQTFZVRBI-UHFFFAOYSA-N 2-[(4-bromophenyl)methoxy]acetic acid Chemical compound OC(=O)COCC1=CC=C(Br)C=C1 ZIEXYIQTFZVRBI-UHFFFAOYSA-N 0.000 claims description 2
- 239000003085 diluting agent Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 description 34
- 238000000034 method Methods 0.000 description 32
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 15
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 9
- ABEXEQSGABRUHS-UHFFFAOYSA-N 16-methylheptadecyl 16-methylheptadecanoate Chemical compound CC(C)CCCCCCCCCCCCCCCOC(=O)CCCCCCCCCCCCCCC(C)C ABEXEQSGABRUHS-UHFFFAOYSA-N 0.000 description 8
- 241000764238 Isis Species 0.000 description 8
- 238000005417 image-selected in vivo spectroscopy Methods 0.000 description 8
- 238000012739 integrated shape imaging system Methods 0.000 description 8
- 206010068871 Myotonic dystrophy Diseases 0.000 description 7
- 108020004999 messenger RNA Proteins 0.000 description 7
- 101150086792 CLCN1 gene Proteins 0.000 description 3
- 101100107064 Drosophila melanogaster Zasp52 gene Proteins 0.000 description 3
- 102000006382 Ribonucleases Human genes 0.000 description 2
- 108010083644 Ribonucleases Proteins 0.000 description 2
- 230000001419 dependent effect Effects 0.000 description 2
- 230000009885 systemic effect Effects 0.000 description 2
- -1 RNA bases Chemical class 0.000 description 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 1
- 238000012230 antisense oligonucleotides Methods 0.000 description 1
- 230000001413 cellular effect Effects 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 238000007914 intraventricular administration Methods 0.000 description 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 1
- 210000003205 muscle Anatomy 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 208000024891 symptom Diseases 0.000 description 1
- 238000007910 systemic administration Methods 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361864439P | 2013-08-09 | 2013-08-09 | |
| US61/864,439 | 2013-08-09 | ||
| US201361889337P | 2013-10-10 | 2013-10-10 | |
| US61/889,337 | 2013-10-10 | ||
| PCT/US2014/050481 WO2015021457A2 (en) | 2013-08-09 | 2014-08-11 | Compounds and methods for modulation of dystrophia myotonica-protein kinase (dmpk) expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019212072A Division JP7059242B2 (ja) | 2013-08-09 | 2019-11-25 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016530882A JP2016530882A (ja) | 2016-10-06 |
| JP2016530882A5 true JP2016530882A5 (enExample) | 2017-09-21 |
Family
ID=52462058
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016533487A Withdrawn JP2016530882A (ja) | 2013-08-09 | 2014-08-11 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2019212072A Active JP7059242B2 (ja) | 2013-08-09 | 2019-11-25 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2022029819A Pending JP2022071053A (ja) | 2013-08-09 | 2022-02-28 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2024019301A Pending JP2024056842A (ja) | 2013-08-09 | 2024-02-13 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019212072A Active JP7059242B2 (ja) | 2013-08-09 | 2019-11-25 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2022029819A Pending JP2022071053A (ja) | 2013-08-09 | 2022-02-28 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
| JP2024019301A Pending JP2024056842A (ja) | 2013-08-09 | 2024-02-13 | 筋強直性ジストロフィープロテインキナーゼ（ｄｍｐｋ）の発現を調節するための化合物及び方法 |
Country Status (27)
| Country | Link |
|---|---|
| US (4) | US20160304877A1 (enExample) |
| EP (2) | EP3030658B1 (enExample) |
| JP (4) | JP2016530882A (enExample) |
| KR (1) | KR102589140B1 (enExample) |
| CN (1) | CN105452464A (enExample) |
| AU (2) | AU2014306284B2 (enExample) |
| BR (1) | BR112016002399A2 (enExample) |
| CA (1) | CA2920776A1 (enExample) |
| CL (1) | CL2016000301A1 (enExample) |
| CY (1) | CY1124522T1 (enExample) |
| DK (1) | DK3030658T3 (enExample) |
| ES (1) | ES2895099T3 (enExample) |
| HR (1) | HRP20211365T1 (enExample) |
| HU (1) | HUE055867T2 (enExample) |
| IL (3) | IL243917B (enExample) |
| LT (1) | LT3030658T (enExample) |
| MX (2) | MX380778B (enExample) |
| PH (1) | PH12016500263A1 (enExample) |
| PL (1) | PL3030658T3 (enExample) |
| PT (1) | PT3030658T (enExample) |
| RS (1) | RS62403B1 (enExample) |
| RU (1) | RU2690333C2 (enExample) |
| SG (1) | SG11201600941YA (enExample) |
| SI (1) | SI3030658T1 (enExample) |
| SM (1) | SMT202100554T1 (enExample) |
| TW (1) | TW201536329A (enExample) |
| WO (1) | WO2015021457A2 (enExample) |
Families Citing this family (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103167883B (zh) | 2010-07-19 | 2016-08-03 | F·C·贝内特 | 肌强直性营养障碍蛋白激酶(dmpk)表达的调节 |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| MX2017012426A (es) * | 2015-04-03 | 2018-01-26 | Ionis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion de tmprss6. |
| WO2017053999A1 (en) | 2015-09-25 | 2017-03-30 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| EP3384055B1 (en) | 2015-11-30 | 2025-07-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP3478829A1 (en) * | 2016-06-29 | 2019-05-08 | Crispr Therapeutics AG | Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders |
| MX2018016253A (es) * | 2016-07-05 | 2019-09-09 | Biomarin Tech Bv | Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos. |
| EP3487523B1 (en) | 2016-07-19 | 2023-09-06 | Duke University | Therapeutic applications of cpf1-based genome editing |
| EP3548005A4 (en) | 2016-11-29 | 2020-06-17 | Puretech Health LLC | Exosomes for delivery of therapeutic agents |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| WO2018226788A1 (en) * | 2017-06-07 | 2018-12-13 | University Of Massachusetts | Anti-adam33 oligonucleotides and related methods |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| KR102850887B1 (ko) | 2017-08-03 | 2025-08-28 | 보이저 테라퓨틱스, 인크. | Aav의 전달을 위한 조성물 및 방법 |
| WO2019113393A1 (en) | 2017-12-06 | 2019-06-13 | Avidity Biosciences Llc | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
| AU2019216257B2 (en) | 2018-01-31 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2C |
| WO2019157531A1 (en) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| WO2020006458A1 (en) | 2018-06-29 | 2020-01-02 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| JP7705793B2 (ja) * | 2018-08-02 | 2025-07-10 | ダイン セラピューティクス，インコーポレーテッド | 筋強直症ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| 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 |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| CA3110466A1 (en) * | 2018-08-22 | 2020-02-27 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene |
| IL319265A (en) | 2018-12-21 | 2025-04-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and their uses |
| US12377170B2 (en) | 2019-02-26 | 2025-08-05 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of β-sarcoglycan and the treatment of muscular dystrophy |
| AU2020263487A1 (en) | 2019-04-25 | 2021-12-16 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| EP4017871B1 (en) | 2019-08-21 | 2024-01-17 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| TW202144572A (zh) | 2020-03-19 | 2021-12-01 | 美商亞維代堤生物科學公司 | 治療臉肩胛肱骨肌肉失養症之組合物及方法 |
| WO2021195469A1 (en) | 2020-03-27 | 2021-09-30 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle dystrophy |
| JP2023130528A (ja) * | 2020-06-24 | 2023-09-21 | 田辺三菱製薬株式会社 | Ｐｌｐ１発現を調節するための化合物、方法及び医薬組成物 |
| CA3202832A1 (en) | 2020-12-31 | 2022-07-07 | Romesh R. Subramanian | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11633498B2 (en) * | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11969475B2 (en) * | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
| CN118265544A (zh) | 2021-09-16 | 2024-06-28 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
| JP2023059858A (ja) | 2021-10-15 | 2023-04-27 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用 |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| WO2025206092A1 (ja) * | 2024-03-27 | 2025-10-02 | 田辺三菱製薬株式会社 | Ｍｙｏｒｅｇｕｌｉｎ発現を調節するための化合物及び医薬組成物 |
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