AU2014236160A1 - Exon skipping compositions for treating muscular dystrophy - Google Patents
Exon skipping compositions for treating muscular dystrophy Download PDFInfo
- Publication number
- AU2014236160A1 AU2014236160A1 AU2014236160A AU2014236160A AU2014236160A1 AU 2014236160 A1 AU2014236160 A1 AU 2014236160A1 AU 2014236160 A AU2014236160 A AU 2014236160A AU 2014236160 A AU2014236160 A AU 2014236160A AU 2014236160 A1 AU2014236160 A1 AU 2014236160A1
- Authority
- AU
- Australia
- Prior art keywords
- antisense
- oligomer
- antisense oligomer
- pct
- dystrophin
- 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.)
- Abandoned
Links
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/351—Conjugate
-
- 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
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3535—Nitrogen
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2020203473A AU2020203473A1 (en) | 2013-03-14 | 2020-05-27 | Exon skipping compositions for treating muscular dystrophy |
| AU2022252769A AU2022252769A1 (en) | 2013-03-14 | 2022-10-13 | Exon skipping compositions for treating muscular dystrophy |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361782706P | 2013-03-14 | 2013-03-14 | |
| US61/782,706 | 2013-03-14 | ||
| PCT/US2014/029766 WO2014153240A2 (en) | 2013-03-14 | 2014-03-14 | Exon skipping compositions for treating muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2020203473A Division AU2020203473A1 (en) | 2013-03-14 | 2020-05-27 | Exon skipping compositions for treating muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2014236160A1 true AU2014236160A1 (en) | 2015-10-01 |
Family
ID=50694008
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2014236160A Abandoned AU2014236160A1 (en) | 2013-03-14 | 2014-03-14 | Exon skipping compositions for treating muscular dystrophy |
| AU2020203473A Abandoned AU2020203473A1 (en) | 2013-03-14 | 2020-05-27 | Exon skipping compositions for treating muscular dystrophy |
| AU2022252769A Abandoned AU2022252769A1 (en) | 2013-03-14 | 2022-10-13 | Exon skipping compositions for treating muscular dystrophy |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2020203473A Abandoned AU2020203473A1 (en) | 2013-03-14 | 2020-05-27 | Exon skipping compositions for treating muscular dystrophy |
| AU2022252769A Abandoned AU2022252769A1 (en) | 2013-03-14 | 2022-10-13 | Exon skipping compositions for treating muscular dystrophy |
Country Status (26)
| Country | Link |
|---|---|
| US (5) | US20140315977A1 (enExample) |
| EP (3) | EP3760720A1 (enExample) |
| JP (4) | JP2016521119A (enExample) |
| KR (5) | KR20240094032A (enExample) |
| CN (2) | CN110066793A (enExample) |
| AU (3) | AU2014236160A1 (enExample) |
| BR (1) | BR112015023038A2 (enExample) |
| CA (1) | CA2903872A1 (enExample) |
| CY (1) | CY1121876T1 (enExample) |
| DK (1) | DK2970964T3 (enExample) |
| EA (1) | EA201591694A1 (enExample) |
| ES (1) | ES2714290T3 (enExample) |
| HR (1) | HRP20190382T1 (enExample) |
| HU (1) | HUE042218T2 (enExample) |
| IL (2) | IL241047B (enExample) |
| LT (1) | LT2970964T (enExample) |
| MX (2) | MX366274B (enExample) |
| NZ (1) | NZ631132A (enExample) |
| PL (1) | PL2970964T3 (enExample) |
| PT (1) | PT2970964T (enExample) |
| RS (1) | RS58529B1 (enExample) |
| SA (1) | SA515361121B1 (enExample) |
| SI (1) | SI2970964T1 (enExample) |
| SM (1) | SMT201900139T1 (enExample) |
| TR (1) | TR201903009T4 (enExample) |
| WO (1) | WO2014153240A2 (enExample) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| PT2203173E (pt) * | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| TR201902952T4 (tr) | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
| CA2780563C (en) | 2009-11-12 | 2025-05-06 | Univ Western Australia | Antisense Molecules and Methods for Treating Pathologies |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| NZ775701A (en) | 2013-03-14 | 2022-08-26 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| NZ732507A (en) | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| JOP20200228A1 (ar) | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| IL263044B2 (en) * | 2016-05-24 | 2024-06-01 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45362A (fr) * | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| AU2017270598B2 (en) * | 2016-05-24 | 2022-12-01 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| HRP20240583T1 (hr) | 2016-05-24 | 2024-07-19 | Sarepta Therapeutics, Inc. | Postupci za pripremu fosforodiamidat morfolino oligomera |
| EP3464305B1 (en) * | 2016-05-24 | 2024-08-21 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| KR20190024977A (ko) | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| 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 |
| WO2018014042A1 (en) * | 2016-07-15 | 2018-01-18 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of dystrophin transcript |
| EP3515445A4 (en) * | 2016-09-20 | 2020-05-27 | The Regents of The University of Colorado, A Body Corporate | SYNTHESIS OF BACKBONE-MODIFIED MORPHOLINO-OLIGONUCLEOTIDES AND CHIMERES WITH THE AID OF PHOSPHORAMIDITE CHEMISTRY |
| WO2018118627A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| KR20240006057A (ko) * | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
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| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| US20200362339A1 (en) * | 2017-09-25 | 2020-11-19 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis |
| US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| JP7596148B2 (ja) * | 2018-03-16 | 2024-12-09 | サレプタ セラピューティクス， インコーポレイテッド | アンチセンス送達用キメラペプチド |
| WO2019209764A2 (en) * | 2018-04-26 | 2019-10-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| US10765760B2 (en) * | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| WO2019241470A2 (en) * | 2018-06-14 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| MX2020013880A (es) * | 2018-06-26 | 2021-03-09 | Nippon Shinyaku Co Ltd | Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne. |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| 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 |
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| GB201812980D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
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| CA3122200A1 (en) | 2018-12-07 | 2020-06-11 | Oxford University Innovation Limited | Linkers |
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| CA3153902A1 (en) | 2019-10-16 | 2021-04-22 | Pardis SABETI | Engineered muscle targeting compositions |
| JP7703579B2 (ja) * | 2020-07-10 | 2025-07-07 | チャンヂョウ シンザオール ファーマシューティカルズ カンパニー リミテッド | オリゴヌクレオチドを調製するための方法 |
| CA3211038A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| MX2023011612A (es) | 2021-03-31 | 2023-12-15 | Entrada Therapeutics Inc | Peptidos de penetracion celular ciclicos. |
| WO2022241408A1 (en) | 2021-05-10 | 2022-11-17 | Entrada Therapeutics, Inc. | Compositions and methods for modulating tissue distribution of intracellular therapeutics |
| WO2022217366A1 (en) * | 2021-04-14 | 2022-10-20 | The Royal Institution For The Advancement Of Learning/Mcgill University | Modified oligonucleotides for the treatment of duchenne muscular dystrophy |
| EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
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| JPWO2023127918A1 (enExample) | 2021-12-27 | 2023-07-06 | ||
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