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)
- 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 (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 (21)
| Country | Link |
|---|---|
| US (2) | US20210261963A1 (https=) |
| EP (1) | EP3815696A4 (https=) |
| JP (3) | JP7345466B2 (https=) |
| KR (1) | KR20210023988A (https=) |
| CN (1) | CN112399849A (https=) |
| AU (2) | AU2019293687B2 (https=) |
| BR (1) | BR112020026542A2 (https=) |
| CA (1) | CA3101321A1 (https=) |
| CL (1) | CL2020003367A1 (https=) |
| CO (1) | CO2020015685A2 (https=) |
| EC (1) | ECSP20083454A (https=) |
| IL (1) | IL279692A (https=) |
| MX (2) | MX2020013880A (https=) |
| MY (1) | MY209148A (https=) |
| NZ (1) | NZ770540A (https=) |
| PE (1) | PE20210630A1 (https=) |
| PH (1) | PH12020552078A1 (https=) |
| SG (1) | SG11202011554PA (https=) |
| TW (1) | TW202035692A (https=) |
| WO (1) | WO2020004675A1 (https=) |
| ZA (1) | ZA202007682B (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| BR112022022889A2 (pt) | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| MX2023012559A (es) | 2021-04-30 | 2023-12-15 | Sarepta Therapeutics Inc | Métodos de tratamiento para la distrofia muscular. |
| JP2025509438A (ja) | 2022-03-17 | 2025-04-11 | サレプタ セラピューティクス， インコーポレイテッド | ホスホロジアミデートモルホリノオリゴマーコンジュゲート |
| WO2023215781A1 (en) * | 2022-05-05 | 2023-11-09 | Biomarin Pharmaceutical Inc. | Method of treating duchenne muscular dystrophy |
| 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 |
|---|---|---|---|---|
| JP3398378B2 (ja) | 1989-12-20 | 2003-04-21 | アンチビラルス・インコーポレイテツド | リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー |
| JP4777777B2 (ja) | 2002-11-25 | 2011-09-21 | 雅文 松尾 | ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬 |
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| EP1811024A1 (en) | 2004-10-05 | 2007-07-25 | Nippon Shinyaku Co., Ltd. | Oligo double-stranded rna and medicinal composition |
| 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 |
| CN112574988A (zh) | 2008-10-24 | 2021-03-30 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CN112251436A (zh) * | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| RS58529B1 (sr) * | 2013-03-14 | 2019-04-30 | Sarepta Therapeutics Inc | Sastavi koji preskaču egzon za tretiranje mišićne distrofije |
| CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| WO2014189142A1 (ja) * | 2013-05-24 | 2014-11-27 | 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 |
| EA201890811A1 (ru) * | 2015-09-30 | 2018-09-28 | Сарепта Терапьютикс, Инк. | Способы лечения мышечной дистрофии |
| 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 EP EP19826094.5A patent/EP3815696A4/en active Pending
- 2019-06-26 MY MYPI2020006568A patent/MY209148A/en unknown
- 2019-06-26 PE PE2020002157A patent/PE20210630A1/es unknown
- 2019-06-26 TW TW108122381A patent/TW202035692A/zh unknown
- 2019-06-26 US US17/253,760 patent/US20210261963A1/en not_active Abandoned
- 2019-06-26 BR BR112020026542-0A patent/BR112020026542A2/pt unknown
- 2019-06-26 WO PCT/JP2019/026393 patent/WO2020004675A1/ja not_active Ceased
- 2019-06-26 CA CA3101321A patent/CA3101321A1/en active Pending
- 2019-06-26 MX MX2020013880A patent/MX2020013880A/es unknown
- 2019-06-26 JP JP2020527712A patent/JP7345466B2/ja active Active
- 2019-06-26 KR KR1020217001037A patent/KR20210023988A/ko not_active Ceased
- 2019-06-26 SG SG11202011554PA patent/SG11202011554PA/en unknown
- 2019-06-26 AU AU2019293687A patent/AU2019293687B2/en active Active
- 2019-06-26 NZ NZ770540A patent/NZ770540A/en unknown
- 2019-06-26 CN CN201980043482.XA patent/CN112399849A/zh 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
-
2025
- 2025-12-19 AU AU2025283628A patent/AU2025283628A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| ZA202007682B (en) | 2024-04-24 |
| JP7595719B2 (ja) | 2024-12-06 |
| JP2023171731A (ja) | 2023-12-05 |
| MY209148A (en) | 2025-06-24 |
| ECSP20083454A (es) | 2021-01-29 |
| WO2020004675A1 (ja) | 2020-01-02 |
| EP3815696A4 (en) | 2022-11-30 |
| JP7345466B2 (ja) | 2023-09-15 |
| AU2025283628A1 (en) | 2026-01-22 |
| JP2025028961A (ja) | 2025-03-05 |
| IL279692A (en) | 2021-03-01 |
| SG11202011554PA (en) | 2020-12-30 |
| CO2020015685A2 (es) | 2021-04-30 |
| MX2025001486A (es) | 2025-03-07 |
| EP3815696A1 (en) | 2021-05-05 |
| AU2019293687B2 (en) | 2025-10-02 |
| JPWO2020004675A1 (ja) | 2021-07-15 |
| CL2020003367A1 (es) | 2021-05-24 |
| CA3101321A1 (en) | 2020-01-02 |
| CN112399849A (zh) | 2021-02-23 |
| MX2020013880A (es) | 2021-03-09 |
| AU2019293687A1 (en) | 2021-01-07 |
| US20210261963A1 (en) | 2021-08-26 |
| PH12020552078A1 (en) | 2021-05-31 |
| TW202035692A (zh) | 2020-10-01 |
| BR112020026542A2 (pt) | 2021-04-06 |
| NZ770540A (en) | 2025-12-19 |
| KR20210023988A (ko) | 2021-03-04 |
| US20240158792A1 (en) | 2024-05-16 |
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