PE20210630A1 - Composicion que comprende el oligonucleotido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne - Google Patents
Composicion que comprende el oligonucleotido antisentido y su uso para el tratamiento de la distrofia muscular de duchenneInfo
- Publication number
- PE20210630A1 PE20210630A1 PE2020002157A PE2020002157A PE20210630A1 PE 20210630 A1 PE20210630 A1 PE 20210630A1 PE 2020002157 A PE2020002157 A PE 2020002157A PE 2020002157 A PE2020002157 A PE 2020002157A PE 20210630 A1 PE20210630 A1 PE 20210630A1
- Authority
- PE
- Peru
- Prior art keywords
- treatment
- muscular dystrophy
- duchenne muscular
- composition including
- including anti
- Prior art date
Links
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 108091034117 Oligonucleotide Proteins 0.000 title 1
- 239000000074 antisense oligonucleotide Substances 0.000 title 1
- 238000012230 antisense oligonucleotides Methods 0.000 title 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
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)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (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)
Abstract
La presente invencion se refiere a una composicion farmaceutica que contiene un oligomero antisentido que consiste en una secuencia de bases (SEQ ID NO: 3) complementaria a una secuencia que consiste en nucleotidos en las posiciones 36 a 56 de la terminal 5' del exon 53 de un gen de distrofina humana, o una sal farmaceuticamente aceptable del mismo, o un hidrato de este. Dicha composicion es util en el tratamiento de la distrofia muscular de Duchenne
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862690270P | 2018-06-26 | 2018-06-26 | |
| US201862739386P | 2018-10-01 | 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 |
|---|---|
| PE20210630A1 true PE20210630A1 (es) | 2021-03-23 |
Family
ID=68987235
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2020002157A PE20210630A1 (es) | 2018-06-26 | 2019-06-26 | Composicion que comprende el oligonucleotido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne |
Country Status (20)
| Country | Link |
|---|---|
| US (2) | US20210261963A1 (es) |
| EP (1) | EP3815696A4 (es) |
| JP (3) | JP7345466B2 (es) |
| KR (1) | KR20210023988A (es) |
| CN (1) | CN112399849A (es) |
| AU (1) | AU2019293687B2 (es) |
| BR (1) | BR112020026542A2 (es) |
| CA (1) | CA3101321A1 (es) |
| CL (1) | CL2020003367A1 (es) |
| CO (1) | CO2020015685A2 (es) |
| EC (1) | ECSP20083454A (es) |
| IL (1) | IL279692A (es) |
| MX (2) | MX2020013880A (es) |
| MY (1) | MY209148A (es) |
| PE (1) | PE20210630A1 (es) |
| PH (1) | PH12020552078A1 (es) |
| SG (1) | SG11202011554PA (es) |
| TW (1) | TW202035692A (es) |
| WO (1) | WO2020004675A1 (es) |
| ZA (1) | ZA202007682B (es) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4150092A4 (en) | 2020-05-11 | 2024-11-06 | Stoke Therapeutics, Inc. | OPA1 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASES AND CONDITIONS |
| EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| CA3252177A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | MORPHOLINO PHOSPHORODIAMIDATE OLIGOMER CONJUGATE |
| EP4665407A2 (en) * | 2023-02-14 | 2025-12-24 | 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 |
|---|---|---|---|---|
| CA2069869C (en) | 1989-12-20 | 1995-12-19 | James E. Summerton | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| CA2796924C (en) | 2002-11-25 | 2016-12-13 | Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy | Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors |
| EP1766010B1 (en) | 2004-06-28 | 2011-02-16 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2006038608A1 (ja) | 2004-10-05 | 2006-04-13 | Nippon Shinyaku Co., Ltd. | オリゴ二本鎖ｒｎａ及び医薬組成物 |
| WO2006129594A1 (ja) | 2005-05-30 | 2006-12-07 | Nippon Shinyaku Co., Ltd. | 核酸含有複合体製剤の製造方法 |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| US8084601B2 (en) * | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| KR20180118828A (ko) | 2008-10-24 | 2018-10-31 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Dmd를 위한 다중 엑손 스키핑 조성물 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| JP2015509922A (ja) * | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． | デュシェンヌ型及びベッカー型筋ジストロフィー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 |
| CN110066793A (zh) * | 2013-03-14 | 2019-07-30 | 萨勒普塔医疗公司 | 用于治疗肌肉萎缩的外显子跳跃组合物 |
| BR112015022998A2 (pt) | 2013-03-15 | 2017-11-14 | Sarepta Therapeutics Inc | composições melhoradas para o tratamento de distrofia muscular |
| EP3015467B1 (en) * | 2013-05-24 | 2024-12-04 | Ajinomoto Co., Inc. | Morpholino oligonucleotide manufacturing method |
| EP3653216A1 (en) * | 2015-09-30 | 2020-05-20 | Sarepta Therapeutics, Inc. | Methods 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 |
-
2019
- 2019-06-26 AU AU2019293687A patent/AU2019293687B2/en active Active
- 2019-06-26 CN CN201980043482.XA patent/CN112399849A/zh active Pending
- 2019-06-26 MX MX2020013880A patent/MX2020013880A/es unknown
- 2019-06-26 JP JP2020527712A patent/JP7345466B2/ja active Active
- 2019-06-26 EP EP19826094.5A patent/EP3815696A4/en active Pending
- 2019-06-26 KR KR1020217001037A patent/KR20210023988A/ko not_active Ceased
- 2019-06-26 BR BR112020026542-0A patent/BR112020026542A2/pt unknown
- 2019-06-26 PE PE2020002157A patent/PE20210630A1/es unknown
- 2019-06-26 US US17/253,760 patent/US20210261963A1/en not_active Abandoned
- 2019-06-26 SG SG11202011554PA patent/SG11202011554PA/en unknown
- 2019-06-26 WO PCT/JP2019/026393 patent/WO2020004675A1/ja not_active Ceased
- 2019-06-26 MY MYPI2020006568A patent/MY209148A/en unknown
- 2019-06-26 TW TW108122381A patent/TW202035692A/zh unknown
- 2019-06-26 CA CA3101321A patent/CA3101321A1/en active Pending
-
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 EC ECSENADI202083454A patent/ECSP20083454A/es unknown
- 2020-12-23 CL CL2020003367A patent/CL2020003367A1/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 |
|---|---|
| BR112020026542A2 (pt) | 2021-04-06 |
| MY209148A (en) | 2025-06-24 |
| MX2025001486A (es) | 2025-03-07 |
| ZA202007682B (en) | 2024-04-24 |
| MX2020013880A (es) | 2021-03-09 |
| EP3815696A1 (en) | 2021-05-05 |
| JP7595719B2 (ja) | 2024-12-06 |
| CN112399849A (zh) | 2021-02-23 |
| CL2020003367A1 (es) | 2021-05-24 |
| JP2025028961A (ja) | 2025-03-05 |
| WO2020004675A1 (ja) | 2020-01-02 |
| IL279692A (en) | 2021-03-01 |
| EP3815696A4 (en) | 2022-11-30 |
| ECSP20083454A (es) | 2021-01-29 |
| KR20210023988A (ko) | 2021-03-04 |
| PH12020552078A1 (en) | 2021-05-31 |
| TW202035692A (zh) | 2020-10-01 |
| JP2023171731A (ja) | 2023-12-05 |
| SG11202011554PA (en) | 2020-12-30 |
| CO2020015685A2 (es) | 2021-04-30 |
| US20210261963A1 (en) | 2021-08-26 |
| AU2019293687A1 (en) | 2021-01-07 |
| AU2019293687B2 (en) | 2025-10-02 |
| JP7345466B2 (ja) | 2023-09-15 |
| CA3101321A1 (en) | 2020-01-02 |
| JPWO2020004675A1 (ja) | 2021-07-15 |
| US20240158792A1 (en) | 2024-05-16 |
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