CA2920776A1 - Compounds and methods for modulation of dystrophia myotonica-protein kinase (dmpk) expression - Google Patents
Compounds and methods for modulation of dystrophia myotonica-protein kinase (dmpk) expression Download PDFInfo
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Classifications
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- C—CHEMISTRY; METALLURGY
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/35—Nature of the modification
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- C12N2310/3525—MOE, methoxyethoxy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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Applications Claiming Priority (5)
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| 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 |
Publications (1)
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| CA2920776A1 true CA2920776A1 (en) | 2015-02-12 |
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| CA2920776A Abandoned CA2920776A1 (en) | 2013-08-09 | 2014-08-11 | Compounds and methods for modulation of dystrophia myotonica-protein kinase (dmpk) expression |
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| US20130225659A1 (en) | 2010-07-19 | 2013-08-29 | Isis Pharmaceuticals, Inc. | Modulation of nuclear-retained rna |
| 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 |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| WO2016161429A1 (en) | 2015-04-03 | 2016-10-06 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating tmprss6 expression |
| US11116843B2 (en) | 2015-09-25 | 2021-09-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| US11617783B2 (en) | 2015-11-16 | 2023-04-04 | Research Institute At Nationwide Children's Hospital | Repairing a mutant human titin gene using CRISPR technology |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| KR102775461B1 (ko) | 2016-04-01 | 2025-02-28 | 어비디티 바이오사이언시스 인크. | 핵산-폴리펩타이드 조성물 및 이의 용도 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| US11427838B2 (en) | 2016-06-29 | 2022-08-30 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders |
| CN109757108A (zh) * | 2016-07-05 | 2019-05-14 | 比奥马林技术公司 | 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸 |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| WO2018102397A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| SG10202107429WA (en) | 2017-01-06 | 2021-08-30 | Avidity Biosciences Inc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
| 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 |
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| CN118638787A (zh) | 2017-12-06 | 2024-09-13 | 艾维迪提生物科学公司 | 治疗肌萎缩和强直性肌营养不良的组合物和方法 |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| AU2019216257B2 (en) | 2018-01-31 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2C |
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| US12391928B2 (en) | 2018-06-29 | 2025-08-19 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A |
| 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 |
| 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 |
| US20210308272A1 (en) * | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| AU2019316103B2 (en) | 2018-08-02 | 2026-02-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| WO2020041634A1 (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 |
| CN113766935A (zh) | 2019-02-26 | 2021-12-07 | 全国儿童医院研究所 | β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗 |
| EP3959319A4 (en) | 2019-04-25 | 2023-06-07 | Avidity Biosciences, Inc. | NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING |
| SI4017871T1 (sl) | 2019-08-21 | 2024-06-28 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije |
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| 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 |
| KR20230128050A (ko) | 2020-12-31 | 2023-09-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 근긴장성 이영양증을 치료하기위한 그의 용도 |
| 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 |
| US11633498B2 (en) * | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US20250177433A1 (en) * | 2021-09-01 | 2025-06-05 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
| EP4396352A4 (en) * | 2021-09-01 | 2025-10-01 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS 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 |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| TW202340468A (zh) | 2022-01-10 | 2023-10-16 | 中國大陸商杭州浩博醫藥有限公司 | B型肝炎病毒(hbv)表現之調節 |
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