CA3101321A1 - Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy - Google Patents
Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy Download PDFInfo
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- CA3101321A1 CA3101321A1 CA3101321A CA3101321A CA3101321A1 CA 3101321 A1 CA3101321 A1 CA 3101321A1 CA 3101321 A CA3101321 A CA 3101321A CA 3101321 A CA3101321 A CA 3101321A CA 3101321 A1 CA3101321 A1 CA 3101321A1
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- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 title claims abstract description 137
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- 239000000074 antisense oligonucleotide Substances 0.000 title abstract description 4
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- QEMXHQIAXOOASZ-UHFFFAOYSA-N tetramethylammonium Chemical class C[N+](C)(C)C QEMXHQIAXOOASZ-UHFFFAOYSA-N 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
- JOXIMZWYDAKGHI-UHFFFAOYSA-N toluene-4-sulfonic acid Chemical compound CC1=CC=C(S(O)(=O)=O)C=C1 JOXIMZWYDAKGHI-UHFFFAOYSA-N 0.000 description 1
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Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
-
- 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
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- 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
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862690270P | 2018-06-26 | 2018-06-26 | |
| US62/690,270 | 2018-06-26 | ||
| US201862739386P | 2018-10-01 | 2018-10-01 | |
| US62/739,386 | 2018-10-01 | ||
| PCT/JP2019/026393 WO2020004675A1 (ja) | 2018-06-26 | 2019-06-26 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3101321A1 true CA3101321A1 (en) | 2020-01-02 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3101321A Pending CA3101321A1 (en) | 2018-06-26 | 2019-06-26 | Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy |
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| US (2) | US20210261963A1 (OSRAM) |
| EP (1) | EP3815696A4 (OSRAM) |
| JP (3) | JP7345466B2 (OSRAM) |
| KR (1) | KR20210023988A (OSRAM) |
| CN (1) | CN112399849A (OSRAM) |
| AU (1) | AU2019293687B2 (OSRAM) |
| BR (1) | BR112020026542A2 (OSRAM) |
| CA (1) | CA3101321A1 (OSRAM) |
| CL (1) | CL2020003367A1 (OSRAM) |
| CO (1) | CO2020015685A2 (OSRAM) |
| EC (1) | ECSP20083454A (OSRAM) |
| IL (1) | IL279692A (OSRAM) |
| MX (2) | MX2020013880A (OSRAM) |
| MY (1) | MY209148A (OSRAM) |
| PE (1) | PE20210630A1 (OSRAM) |
| PH (1) | PH12020552078A1 (OSRAM) |
| SG (1) | SG11202011554PA (OSRAM) |
| TW (1) | TW202035692A (OSRAM) |
| WO (1) | WO2020004675A1 (OSRAM) |
| ZA (1) | ZA202007682B (OSRAM) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| CA3173647A1 (en) | 2020-05-11 | 2021-11-18 | Isabel AZNAREZ | Opa1 antisense oligomers for treatment of conditions and diseases |
| KR20240004609A (ko) | 2021-04-30 | 2024-01-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근이영양증에 대한 치료 방법 |
| EP4493693A1 (en) | 2022-03-17 | 2025-01-22 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
| AU2024222908A1 (en) * | 2023-02-14 | 2025-09-04 | Stoke Therapeutics, Inc. | Antisense oligomer formulations |
| US20240368596A1 (en) * | 2023-05-05 | 2024-11-07 | Biomarin Pharmaceutical Inc. | Dystrophin exon skipping oligonucleotides |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| EP0506830B1 (en) | 1989-12-20 | 2005-12-14 | Avi Biopharma, Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| CA3001404C (en) | 2002-11-25 | 2020-07-28 | Masafumi Matsuo | Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors |
| PL1766010T3 (pl) | 2004-06-28 | 2011-07-29 | Univ Western Australia | Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania |
| EP1811024A1 (en) | 2004-10-05 | 2007-07-25 | Nippon Shinyaku Co., Ltd. | Oligo double-stranded rna and medicinal composition |
| US20090069260A1 (en) | 2005-05-30 | 2009-03-12 | Nippon Shinyaku Co., Ltd | Method for producing a nucleic-acid-containing complex preparation |
| CA2704261C (en) | 2007-11-15 | 2015-05-26 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| SMT201900159T1 (it) | 2008-10-24 | 2019-05-10 | Sarepta Therapeutics Inc | Composizioni di salto di esone per dmd |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| BR112014018427B1 (pt) * | 2012-01-27 | 2021-11-03 | Biomarin Technologies B.V. | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker |
| KR20240094032A (ko) * | 2013-03-14 | 2024-06-24 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| HK1220154A1 (zh) | 2013-03-15 | 2017-04-28 | Sarepta Therapeutics, Inc. | 改进的用於治疗肌营养不良的组合物 |
| JP6477464B2 (ja) * | 2013-05-24 | 2019-03-06 | 味の素株式会社 | モルフォリノオリゴヌクレオチドの製造方法 |
| JP2018529715A (ja) * | 2015-09-30 | 2018-10-11 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための方法 |
| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
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- 2019-06-26 WO PCT/JP2019/026393 patent/WO2020004675A1/ja not_active Ceased
- 2019-06-26 CN CN201980043482.XA patent/CN112399849A/zh active Pending
- 2019-06-26 US US17/253,760 patent/US20210261963A1/en not_active Abandoned
- 2019-06-26 AU AU2019293687A patent/AU2019293687B2/en active Active
- 2019-06-26 JP JP2020527712A patent/JP7345466B2/ja active Active
- 2019-06-26 PE PE2020002157A patent/PE20210630A1/es unknown
- 2019-06-26 CA CA3101321A patent/CA3101321A1/en active Pending
- 2019-06-26 SG SG11202011554PA patent/SG11202011554PA/en unknown
- 2019-06-26 TW TW108122381A patent/TW202035692A/zh unknown
- 2019-06-26 EP EP19826094.5A patent/EP3815696A4/en active Pending
- 2019-06-26 KR KR1020217001037A patent/KR20210023988A/ko not_active Ceased
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2020
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2023
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- 2023-11-08 US US18/504,781 patent/US20240158792A1/en active Pending
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2024
- 2024-11-26 JP JP2024205093A patent/JP2025028961A/ja active Pending
Also Published As
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| PH12020552078A1 (en) | 2021-05-31 |
| TW202035692A (zh) | 2020-10-01 |
| SG11202011554PA (en) | 2020-12-30 |
| CN112399849A (zh) | 2021-02-23 |
| JP7345466B2 (ja) | 2023-09-15 |
| EP3815696A4 (en) | 2022-11-30 |
| CO2020015685A2 (es) | 2021-04-30 |
| AU2019293687B2 (en) | 2025-10-02 |
| ECSP20083454A (es) | 2021-01-29 |
| KR20210023988A (ko) | 2021-03-04 |
| WO2020004675A1 (ja) | 2020-01-02 |
| JP2023171731A (ja) | 2023-12-05 |
| AU2019293687A1 (en) | 2021-01-07 |
| US20210261963A1 (en) | 2021-08-26 |
| EP3815696A1 (en) | 2021-05-05 |
| MY209148A (en) | 2025-06-24 |
| BR112020026542A2 (pt) | 2021-04-06 |
| CL2020003367A1 (es) | 2021-05-24 |
| JPWO2020004675A1 (ja) | 2021-07-15 |
| JP7595719B2 (ja) | 2024-12-06 |
| MX2025001486A (es) | 2025-03-07 |
| ZA202007682B (en) | 2024-04-24 |
| JP2025028961A (ja) | 2025-03-05 |
| US20240158792A1 (en) | 2024-05-16 |
| IL279692A (en) | 2021-03-01 |
| PE20210630A1 (es) | 2021-03-23 |
| MX2020013880A (es) | 2021-03-09 |
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