JP6174678B2 - 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド - Google Patents
神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド Download PDFInfo
- Publication number
- JP6174678B2 JP6174678B2 JP2015506930A JP2015506930A JP6174678B2 JP 6174678 B2 JP6174678 B2 JP 6174678B2 JP 2015506930 A JP2015506930 A JP 2015506930A JP 2015506930 A JP2015506930 A JP 2015506930A JP 6174678 B2 JP6174678 B2 JP 6174678B2
- Authority
- JP
- Japan
- Prior art keywords
- nucleotides
- oligonucleotide
- seq
- rna
- length
- 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.)
- Active
Links
Images
Classifications
-
- 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
-
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2310/333—Modified A
-
- 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
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- 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
- C12N2310/336—Modified G
-
- 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
-
- 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
- C12N2310/3521—Methyl
-
- 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
-
- 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
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Neurosurgery (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Pain & Pain Management (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261636914P | 2012-04-23 | 2012-04-23 | |
| EP12165139.2 | 2012-04-23 | ||
| US61/636,914 | 2012-04-23 | ||
| EP12165139 | 2012-04-23 | ||
| PCT/NL2013/050306 WO2013162363A1 (en) | 2012-04-23 | 2013-04-23 | RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017133005A Division JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015514418A JP2015514418A (ja) | 2015-05-21 |
| JP2015514418A5 JP2015514418A5 (enExample) | 2016-06-16 |
| JP6174678B2 true JP6174678B2 (ja) | 2017-08-02 |
Family
ID=49483552
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015506930A Active JP6174678B2 (ja) | 2012-04-23 | 2013-04-23 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2017133005A Pending JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2020115868A Active JP7459691B2 (ja) | 2012-04-23 | 2020-07-03 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2022093254A Pending JP2022126711A (ja) | 2012-04-23 | 2022-06-08 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2025061303A Pending JP2025111478A (ja) | 2012-04-23 | 2025-04-02 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017133005A Pending JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2020115868A Active JP7459691B2 (ja) | 2012-04-23 | 2020-07-03 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2022093254A Pending JP2022126711A (ja) | 2012-04-23 | 2022-06-08 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2025061303A Pending JP2025111478A (ja) | 2012-04-23 | 2025-04-02 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Country Status (19)
| Country | Link |
|---|---|
| US (5) | US9745576B2 (enExample) |
| EP (4) | EP4400169A3 (enExample) |
| JP (5) | JP6174678B2 (enExample) |
| CN (2) | CN104271741A (enExample) |
| AU (1) | AU2013253074C1 (enExample) |
| BR (1) | BR112014026285B1 (enExample) |
| CA (2) | CA2870697C (enExample) |
| DK (1) | DK2841578T3 (enExample) |
| ES (2) | ES2983421T3 (enExample) |
| HR (1) | HRP20171254T1 (enExample) |
| HU (1) | HUE033431T2 (enExample) |
| LT (1) | LT2841578T (enExample) |
| NZ (1) | NZ700561A (enExample) |
| PL (2) | PL3604536T3 (enExample) |
| PT (1) | PT2841578T (enExample) |
| RS (1) | RS56319B1 (enExample) |
| SI (1) | SI2841578T1 (enExample) |
| SM (1) | SMT201700339T1 (enExample) |
| WO (1) | WO2013162363A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020182472A (ja) * | 2012-04-23 | 2020-11-12 | バイオマリン テクノロジーズ ベー.フェー. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008018795A1 (en) | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| RU2015151857A (ru) | 2008-12-02 | 2019-01-15 | Уэйв Лайф Сайенсес Джапан, Инк. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| US9476043B2 (en) * | 2011-04-08 | 2016-10-25 | Rula Zain-Luqman | Diagnosis and treatment of friedreich's ataxia |
| EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| BR112015008399A8 (pt) * | 2012-10-15 | 2017-10-03 | Ionis Pharmaceuticals Inc | Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição |
| FI2906696T4 (fi) | 2012-10-15 | 2023-03-18 | Menetelmiä c9orf72:n ilmentymisen moduloimiseksi | |
| US10577604B2 (en) * | 2012-10-15 | 2020-03-03 | Ionis Pharmaceuticals, Inc. | Methods for monitoring C9ORF72 expression |
| MY192689A (en) | 2013-10-11 | 2022-09-01 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| WO2015143246A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| WO2016060919A1 (en) | 2014-10-14 | 2016-04-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant c9orf72 foci expression by duplex rnas targeting the expanded hexanucleotide repeat |
| JP6606557B2 (ja) | 2015-04-16 | 2019-11-13 | アイオーニス ファーマシューティカルズ, インコーポレーテッド | C9orf72発現を調節するための組成物 |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| EP3397288A4 (en) | 2015-12-31 | 2019-09-11 | Ionis Pharmaceuticals, Inc. | PROCESS FOR REDUCING ATAXIN-2 EXPRESSION |
| KR20230175312A (ko) * | 2016-07-05 | 2023-12-29 | 바이오마린 테크놀로지스 비.브이. | 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드 |
| JP2020508685A (ja) | 2017-03-03 | 2020-03-26 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | サプレッサーtRNA及びデアミナーゼによる変異のRNAターゲティング |
| BR112020008033A2 (pt) * | 2017-10-23 | 2020-10-27 | Prevail Therapeutics, Inc. | terapias gênicas para doença neurodegenerativa |
| MX2021000922A (es) | 2018-07-25 | 2021-03-31 | Ionis Pharmaceuticals Inc | Compuestos y metodos para reducir la expresion de la atxn2. |
| 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 |
| JP2021533200A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 顔面・肩甲・上腕筋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 |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2021087036A1 (en) * | 2019-11-01 | 2021-05-06 | Alnylam Pharmaceuticals, Inc. | HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| CA3159944A1 (en) | 2019-12-02 | 2021-06-10 | David HUSS | Therapeutic editing |
| JP7738573B2 (ja) * | 2020-05-01 | 2025-09-12 | アイオーニス ファーマシューティカルズ, インコーポレーテッド | Atxn1を調節するための化合物及び方法 |
| CA3195722A1 (en) | 2020-09-17 | 2022-03-24 | Q-State Biosciences, Inc. | Therapeutic compositions for treating pain via multiple targets |
| WO2022232395A2 (en) * | 2021-04-28 | 2022-11-03 | Q-State Biosciences, Inc. | Therapeutic compositions for treating pain via multiple targets |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| AR127158A1 (es) * | 2021-09-29 | 2023-12-27 | Hoffmann La Roche | Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina |
| WO2023164656A2 (en) * | 2022-02-25 | 2023-08-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating atn1 expression |
| KR20250004770A (ko) | 2022-04-15 | 2025-01-08 | 다인 세라퓨틱스, 인크. | 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제 |
| WO2025068368A1 (en) * | 2023-09-26 | 2025-04-03 | Katholieke Universiteit Leuven | Inhibitors for treating cancer |
Family Cites Families (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5695933A (en) | 1993-05-28 | 1997-12-09 | Massachusetts Institute Of Technology | Direct detection of expanded nucleotide repeats in the human genome |
| DE69503126T2 (de) | 1994-05-05 | 1998-11-12 | Beckman Instruments Inc | Repetitive oligonukleotide matrix |
| US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
| US5945290A (en) * | 1998-09-18 | 1999-08-31 | Isis Pharmaceuticals, Inc. | Antisense modulation of RhoA expression |
| US6900187B2 (en) * | 1999-02-26 | 2005-05-31 | The University Of British Columbia | TRPM-2 antisense therapy using an oligonucleotide having 2′-O-(2-methoxy)ethyl modifications |
| US20050277133A1 (en) | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| AU2002319033B2 (en) * | 2001-07-06 | 2008-01-10 | Pharmaxis Ltd | Methods for increasing in vivo efficacy of oligonucleotides and inhibiting inflammation in mammals |
| US20030109476A1 (en) | 2001-08-07 | 2003-06-12 | Kmiec Eric B. | Compositions and methods for the prevention and treatment of Huntington's disease |
| US20060074034A1 (en) | 2001-09-17 | 2006-04-06 | Collins Douglas A | Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof |
| US20050255086A1 (en) | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
| US7655785B1 (en) | 2002-11-14 | 2010-02-02 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof |
| US7514551B2 (en) | 2003-04-03 | 2009-04-07 | Enzo Life Sciences, Inc. | Multisignal labeling reagents, and processes and uses therefor |
| AU2004239114B2 (en) | 2003-05-14 | 2008-03-13 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| WO2006083800A2 (en) | 2005-01-31 | 2006-08-10 | University Of Iowa Research Foundation | Nucleic acid silencing of huntington's disease gene |
| EP2161038B1 (en) | 2006-01-26 | 2013-12-25 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to Huntingtin |
| WO2008018795A1 (en) * | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| CN101501193B (zh) * | 2006-08-11 | 2013-07-03 | 普罗森那技术公司 | 用于治疗与dna重复不稳定性相关的遗传病的方法和手段 |
| AU2008273096B2 (en) | 2007-07-12 | 2013-05-02 | Academisch Ziekenhuis Leiden | Molecules for targeting compounds to various selected organs or tissues |
| EP2249874A1 (en) * | 2008-02-08 | 2010-11-17 | ProSensa Holding BV | Methods and means for treating dna repeat instability associated genetic disorders |
| SI2320925T1 (sl) | 2008-07-10 | 2016-05-31 | Regenesance B.V. | Komplement antagonisti in njih uporaba |
| US20110218334A1 (en) | 2008-07-11 | 2011-09-08 | Alnylam Pharmaceuticals, Inc. | PHOSPHOROTHIOATE OLIGONUCLEOTIDES AND NON-NUCLEOSIDIC PHOSPHOROTHIOATES AS DELIVERY AGENTS FOR iRNA AGENTS |
| CA2732343C (en) | 2008-07-29 | 2017-05-09 | The Board Of Regents Of The University Of Texas System | Selective inhibition of polyglutamine protein expression |
| EP2370582B1 (en) * | 2008-12-04 | 2017-05-10 | CuRNA, Inc. | Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene |
| SG176716A1 (en) | 2009-06-08 | 2012-01-30 | Miragen Therapeutics | CHEMICAL MODIFICATION MOTIFS FOR miRNA INHIBITORS AND MIMETICS |
| WO2011097614A1 (en) * | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Mehods and compositions useful in diseases or conditions related to repeat expansion |
| WO2011097641A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
| EP2576574A2 (en) | 2010-05-28 | 2013-04-10 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| KR20180105730A (ko) | 2010-07-19 | 2018-09-28 | 아이오니스 파마수티컬즈, 인코포레이티드 | 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법 |
| SI2605794T1 (sl) | 2010-08-20 | 2017-01-31 | Replicor Inc. | Oligonukleotidni kelatni kompleksi |
| EP2673361B1 (en) | 2011-02-08 | 2016-04-13 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| WO2012144906A1 (en) * | 2011-04-22 | 2012-10-26 | Prosensa Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1) |
| KR102183273B1 (ko) | 2011-05-05 | 2020-11-27 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| NZ627896A (en) * | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| US20140378533A1 (en) | 2012-02-08 | 2014-12-25 | Isis Pharmaceuticals, Inc. | Modulation of rna by repeat targeting |
| BR112014026285B1 (pt) | 2012-04-23 | 2021-08-31 | Biomarin Technologies B.V | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares |
-
2013
- 2013-04-23 BR BR112014026285-3A patent/BR112014026285B1/pt active IP Right Grant
- 2013-04-23 CN CN201380021505.XA patent/CN104271741A/zh active Pending
- 2013-04-23 EP EP24173123.1A patent/EP4400169A3/en active Pending
- 2013-04-23 EP EP19194127.7A patent/EP3604536B1/en active Active
- 2013-04-23 SM SM20170339T patent/SMT201700339T1/it unknown
- 2013-04-23 HU HUE13720617A patent/HUE033431T2/en unknown
- 2013-04-23 ES ES19194127T patent/ES2983421T3/es active Active
- 2013-04-23 DK DK13720617.3T patent/DK2841578T3/en active
- 2013-04-23 JP JP2015506930A patent/JP6174678B2/ja active Active
- 2013-04-23 AU AU2013253074A patent/AU2013253074C1/en active Active
- 2013-04-23 NZ NZ700561A patent/NZ700561A/en unknown
- 2013-04-23 WO PCT/NL2013/050306 patent/WO2013162363A1/en not_active Ceased
- 2013-04-23 EP EP13720617.3A patent/EP2841578B1/en active Active
- 2013-04-23 CA CA2870697A patent/CA2870697C/en active Active
- 2013-04-23 SI SI201330728T patent/SI2841578T1/sl unknown
- 2013-04-23 HR HRP20171254TT patent/HRP20171254T1/hr unknown
- 2013-04-23 PT PT137206173T patent/PT2841578T/pt unknown
- 2013-04-23 EP EP17170330.9A patent/EP3228712A1/en not_active Ceased
- 2013-04-23 ES ES13720617.3T patent/ES2639066T3/es active Active
- 2013-04-23 CA CA3103101A patent/CA3103101C/en active Active
- 2013-04-23 RS RS20170765A patent/RS56319B1/sr unknown
- 2013-04-23 PL PL19194127.7T patent/PL3604536T3/pl unknown
- 2013-04-23 LT LTEP13720617.3T patent/LT2841578T/lt unknown
- 2013-04-23 CN CN201811488693.8A patent/CN110025628B/zh active Active
- 2013-04-23 PL PL13720617T patent/PL2841578T3/pl unknown
-
2014
- 2014-10-23 US US14/522,002 patent/US9745576B2/en active Active
-
2017
- 2017-07-06 JP JP2017133005A patent/JP2018027080A/ja active Pending
- 2017-08-14 US US15/676,569 patent/US20180066256A1/en not_active Abandoned
-
2019
- 2019-10-22 US US16/660,416 patent/US11345915B2/en active Active
-
2020
- 2020-07-03 JP JP2020115868A patent/JP7459691B2/ja active Active
-
2022
- 2022-04-25 US US17/728,336 patent/US20220389421A1/en active Pending
- 2022-06-08 JP JP2022093254A patent/JP2022126711A/ja active Pending
-
2024
- 2024-06-24 US US18/752,689 patent/US20250011784A1/en active Pending
-
2025
- 2025-04-02 JP JP2025061303A patent/JP2025111478A/ja active Pending
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020182472A (ja) * | 2012-04-23 | 2020-11-12 | バイオマリン テクノロジーズ ベー.フェー. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2022126711A (ja) * | 2012-04-23 | 2022-08-30 | ヴィコ セラピューティクス ビー.ヴイ. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP7459691B2 (ja) | 2012-04-23 | 2024-04-02 | ヴィコ セラピューティクス ビー.ヴイ. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7459691B2 (ja) | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド | |
| AU2020257111B2 (en) | RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy | |
| HK40114855A (en) | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders | |
| HK40020154A (en) | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders | |
| HK1243733A1 (en) | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders | |
| HK40020154B (en) | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders | |
| HK40078359A (en) | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy | |
| HK1207118B (en) | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20160422 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20160422 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20170207 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20170508 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20170606 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20170706 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 6174678 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| S531 | Written request for registration of change of domicile |
Free format text: JAPANESE INTERMEDIATE CODE: R313531 |
|
| R350 | Written notification of registration of transfer |
Free format text: JAPANESE INTERMEDIATE CODE: R350 |
|
| S111 | Request for change of ownership or part of ownership |
Free format text: JAPANESE INTERMEDIATE CODE: R313113 |
|
| R350 | Written notification of registration of transfer |
Free format text: JAPANESE INTERMEDIATE CODE: R350 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |
|
| R250 | Receipt of annual fees |
Free format text: JAPANESE INTERMEDIATE CODE: R250 |