CL2020003367A1 - Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne - Google Patents
Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenneInfo
- Publication number
- CL2020003367A1 CL2020003367A1 CL2020003367A CL2020003367A CL2020003367A1 CL 2020003367 A1 CL2020003367 A1 CL 2020003367A1 CL 2020003367 A CL2020003367 A CL 2020003367A CL 2020003367 A CL2020003367 A CL 2020003367A CL 2020003367 A1 CL2020003367 A1 CL 2020003367A1
- Authority
- CL
- Chile
- Prior art keywords
- treatment
- muscular dystrophy
- duchenne muscular
- composition
- antisense oligonucleotide
- Prior art date
Links
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 title abstract 3
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 2
- 239000000074 antisense oligonucleotide Substances 0.000 title abstract 2
- 238000012230 antisense oligonucleotides Methods 0.000 title abstract 2
Classifications
-
- 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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862690270P | 2018-06-26 | 2018-06-26 | |
| US201862739386P | 2018-10-01 | 2018-10-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2020003367A1 true CL2020003367A1 (es) | 2021-05-24 |
Family
ID=68987235
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2020003367A CL2020003367A1 (es) | 2018-06-26 | 2020-12-23 | Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne |
Country Status (20)
| Country | Link |
|---|---|
| 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) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
-
2019
- 2019-06-26 MY MYPI2020006568A patent/MY209148A/en unknown
- 2019-06-26 BR BR112020026542-0A patent/BR112020026542A2/pt unknown
- 2019-06-26 MX MX2020013880A patent/MX2020013880A/es unknown
- 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
-
2020
- 2020-12-02 PH PH12020552078A patent/PH12020552078A1/en unknown
- 2020-12-09 ZA ZA2020/07682A patent/ZA202007682B/en unknown
- 2020-12-14 CO CONC2020/0015685A patent/CO2020015685A2/es unknown
- 2020-12-16 MX MX2025001486A patent/MX2025001486A/es unknown
- 2020-12-22 IL IL279692A patent/IL279692A/en unknown
- 2020-12-23 CL CL2020003367A patent/CL2020003367A1/es unknown
- 2020-12-23 EC ECSENADI202083454A patent/ECSP20083454A/es unknown
-
2023
- 2023-09-05 JP JP2023143451A patent/JP7595719B2/ja active Active
- 2023-11-08 US US18/504,781 patent/US20240158792A1/en active Pending
-
2024
- 2024-11-26 JP JP2024205093A patent/JP2025028961A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| 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 |
| CA3101321A1 (en) | 2020-01-02 |
| MY209148A (en) | 2025-06-24 |
| BR112020026542A2 (pt) | 2021-04-06 |
| 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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