CL2016000301A1 - Compuestos y métodos para modular la expresión de la proteína cinasa de la distrofia miotónica (dmpk) - Google Patents
Compuestos y métodos para modular la expresión de la proteína cinasa de la distrofia miotónica (dmpk)Info
- Publication number
- CL2016000301A1 CL2016000301A1 CL2016000301A CL2016000301A CL2016000301A1 CL 2016000301 A1 CL2016000301 A1 CL 2016000301A1 CL 2016000301 A CL2016000301 A CL 2016000301A CL 2016000301 A CL2016000301 A CL 2016000301A CL 2016000301 A1 CL2016000301 A1 CL 2016000301A1
- Authority
- CL
- Chile
- Prior art keywords
- dmpk
- modulate
- expression
- compounds
- methods
- Prior art date
Links
- 101100063539 Mus musculus Dmpk gene Proteins 0.000 title 1
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 title 1
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 title 1
- 150000001875 compounds Chemical class 0.000 title 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Pain & Pain Management (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Epidemiology (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361864439P | 2013-08-09 | 2013-08-09 | |
| US201361889337P | 2013-10-10 | 2013-10-10 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2016000301A1 true CL2016000301A1 (es) | 2016-12-16 |
Family
ID=52462058
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2016000301A CL2016000301A1 (es) | 2013-08-09 | 2016-02-05 | Compuestos y métodos para modular la expresión de la proteína cinasa de la distrofia miotónica (dmpk) |
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 (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3031920B1 (en) | 2010-07-19 | 2019-08-21 | Ionis Pharmaceuticals, Inc. | Modulation of dystrophia myotonica-protein kinase (dmpk) expression |
| 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 |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| KR102539587B1 (ko) | 2015-04-03 | 2023-06-01 | 아이오니스 파마수티컬즈, 인코포레이티드 | Tmprss6 발현을 조절하기 위한 화합물 및 방법들 |
| US11116843B2 (en) | 2015-09-25 | 2021-09-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| SG11201804166YA (en) | 2015-11-16 | 2018-06-28 | Res Inst Nationwide Childrens Hospital | Materials and methods for treatment of titin-based myopathies and other titinopaties |
| KR20250044471A (ko) | 2015-11-30 | 2025-03-31 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| 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 |
| 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 |
| BR112019000061A2 (pt) * | 2016-07-05 | 2019-11-26 | Biomarin Technologies B.V. | comutação e modulação de splicing de pré-mrna compreendendo porções de arcabouço bicíclico, com características melhoradas para o tratamento de distúrbios genéticos |
| US12214056B2 (en) | 2016-07-19 | 2025-02-04 | Duke University | Therapeutic applications of CPF1-based genome editing |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| SG11201906200WA (en) | 2017-01-06 | 2019-08-27 | Avidity Biosciences Llc | 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 |
| AU2018311069A1 (en) | 2017-08-03 | 2020-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
| SG11202005022YA (en) | 2017-12-06 | 2020-06-29 | Avidity Biosciences Inc | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOMIC ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYOTES |
| SG11202006722RA (en) | 2018-01-31 | 2020-08-28 | Res Inst Nationwide Childrens Hospital | Gene therapy for limb-girdle muscular dystrophy type 2c |
| EP3752612A4 (en) | 2018-02-12 | 2021-11-10 | Ionis Pharmaceuticals, Inc. | MODIFIED COMPOUNDS AND USES THEREOF |
| EP3814514A1 (en) | 2018-06-29 | 2021-05-05 | 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 |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CA3108287A1 (en) * | 2018-08-02 | 2020-02-06 | 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 |
| 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 |
| AU2019326551A1 (en) | 2018-08-22 | 2021-03-18 | 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 |
| EP3959319A4 (en) | 2019-04-25 | 2023-06-07 | Avidity Biosciences, Inc. | NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING |
| DK4017871T5 (da) | 2019-08-21 | 2024-08-05 | Res Inst Nationwide Childrens Hospital | Adenoassocieret virusvektoradministration af alfa-sarcoglycan og behandling af muskeldystrofi |
| JP7788060B2 (ja) | 2020-03-19 | 2025-12-18 | アビディティー バイオサイエンシーズ，インク． | 顔面肩甲上腕型筋ジストロフィーを処置するための組成物および方法 |
| JP7748962B2 (ja) | 2020-03-27 | 2025-10-03 | アビディティー バイオサイエンシーズ，インク． | 筋ジストロフィーを処置するための組成物および方法 |
| 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 |
| JP2024503609A (ja) | 2020-12-31 | 2024-01-26 | ダイン セラピューティクス，インコーポレーテッド | 筋緊張性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の使用 |
| US11969475B2 (en) * | 2021-07-09 | 2024-04-30 | 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 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| EP4396351A4 (en) * | 2021-09-01 | 2025-12-24 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
| US11912779B2 (en) | 2021-09-16 | 2024-02-27 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| JP2025512464A (ja) | 2022-04-15 | 2025-04-17 | ダイン セラピューティクス，インコーポレーテッド | 筋緊張性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製剤 |
| 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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