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 (2) |
| EP (1) | EP3815696A4 (2) |
| JP (3) | JP7345466B2 (2) |
| KR (1) | KR20210023988A (2) |
| CN (1) | CN112399849A (2) |
| AU (2) | AU2019293687B2 (2) |
| BR (1) | BR112020026542A2 (2) |
| CA (1) | CA3101321A1 (2) |
| CL (1) | CL2020003367A1 (2) |
| CO (1) | CO2020015685A2 (2) |
| EC (1) | ECSP20083454A (2) |
| IL (1) | IL279692A (2) |
| MX (2) | MX2020013880A (2) |
| MY (1) | MY209148A (2) |
| NZ (1) | NZ770540A (2) |
| PE (1) | PE20210630A1 (2) |
| PH (1) | PH12020552078A1 (2) |
| SG (1) | SG11202011554PA (2) |
| TW (1) | TW202035692A (2) |
| WO (1) | WO2020004675A1 (2) |
| ZA (1) | ZA202007682B (2) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| IL307937A (en) | 2021-04-30 | 2023-12-01 | Sarepta Therapeutics Inc | Treatment methods for muscular dystrophy |
| CN119013401A (zh) | 2022-03-17 | 2024-11-22 | 萨勒普塔医疗公司 | 二氨基磷酸酯吗啉代寡聚物缀合物 |
| KR20250006069A (ko) * | 2022-05-05 | 2025-01-10 | 바이오마린 파머수티컬 인크. | 뒤시엔느 근이영양증을 치료하는 방법 |
| CN121194802A (zh) * | 2023-02-14 | 2025-12-23 | 斯托克制药公司 | 反义寡聚物调配物 |
| WO2024233303A1 (en) * | 2023-05-05 | 2024-11-14 | Biomarin Pharmaceutical Inc. | Dystrophin exon skipping oligonucleotides |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| EP2392660A3 (en) | 2002-11-25 | 2012-03-28 | Masafumi Matsuo | ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | 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 |
| EP1886688A4 (en) | 2005-05-30 | 2013-01-09 | Nippon Shinyaku Co Ltd | METHOD FOR PRODUCING PREPARATION OF A NUCLEIC ACID-CONTAINING COMPLEX |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| NZ627896A (en) * | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| US20140315977A1 (en) * | 2013-03-14 | 2014-10-23 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| DK3015467T3 (da) * | 2013-05-24 | 2025-02-10 | Ajinomoto Kk | Morpholino-oligonukleotidfremstillingsfremgangsmåde |
| WO2017059131A1 (en) * | 2015-09-30 | 2017-04-06 | 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 CN CN201980043482.XA patent/CN112399849A/zh active Pending
- 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 NZ NZ770540A patent/NZ770540A/en unknown
- 2019-06-26 MX MX2020013880A patent/MX2020013880A/es unknown
- 2019-06-26 PE PE2020002157A patent/PE20210630A1/es unknown
- 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 KR KR1020217001037A patent/KR20210023988A/ko not_active Ceased
- 2019-06-26 CA CA3101321A patent/CA3101321A1/en active Pending
- 2019-06-26 JP JP2020527712A patent/JP7345466B2/ja active Active
- 2019-06-26 TW TW108122381A patent/TW202035692A/zh unknown
- 2019-06-26 EP EP19826094.5A patent/EP3815696A4/en active Pending
- 2019-06-26 AU AU2019293687A patent/AU2019293687B2/en active Active
- 2019-06-26 MY MYPI2020006568A patent/MY209148A/en unknown
-
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
-
2025
- 2025-12-19 AU AU2025283628A patent/AU2025283628A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CL2020003367A1 (es) | 2021-05-24 |
| BR112020026542A2 (pt) | 2021-04-06 |
| MX2025001486A (es) | 2025-03-07 |
| JP7595719B2 (ja) | 2024-12-06 |
| PH12020552078A1 (en) | 2021-05-31 |
| TW202035692A (zh) | 2020-10-01 |
| WO2020004675A1 (ja) | 2020-01-02 |
| CN112399849A (zh) | 2021-02-23 |
| SG11202011554PA (en) | 2020-12-30 |
| ECSP20083454A (es) | 2021-01-29 |
| AU2019293687B2 (en) | 2025-10-02 |
| EP3815696A1 (en) | 2021-05-05 |
| CO2020015685A2 (es) | 2021-04-30 |
| ZA202007682B (en) | 2024-04-24 |
| AU2019293687A1 (en) | 2021-01-07 |
| CA3101321A1 (en) | 2020-01-02 |
| US20240158792A1 (en) | 2024-05-16 |
| JP2025028961A (ja) | 2025-03-05 |
| MX2020013880A (es) | 2021-03-09 |
| JP7345466B2 (ja) | 2023-09-15 |
| MY209148A (en) | 2025-06-24 |
| EP3815696A4 (en) | 2022-11-30 |
| NZ770540A (en) | 2025-12-19 |
| JP2023171731A (ja) | 2023-12-05 |
| IL279692A (en) | 2021-03-01 |
| KR20210023988A (ko) | 2021-03-04 |
| AU2025283628A1 (en) | 2026-01-22 |
| JPWO2020004675A1 (ja) | 2021-07-15 |
| US20210261963A1 (en) | 2021-08-26 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| PE20210630A1 (es) | Composicion que comprende el oligonucleotido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne | |
| US9981020B2 (en) | Methods and compositions for RNA-guided treatment of HIV infection | |
| CY1124197T1 (el) | Antinohmatika πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων | |
| JP2016522674A5 (2) | ||
| ES2886480T3 (es) | Métodos y composiciones para el tratamiento guiado por ARN de una infección por VIH | |
| JP2018512876A5 (2) | ||
| JP2015523853A5 (2) | ||
| JP2015518712A5 (2) | ||
| JP2015518713A5 (2) | ||
| CY1120685T1 (el) | Ρετινοειδες-λιποσωματα για ενισχυση ρυθμισης εκφρασης hsp47 | |
| JP2016521556A5 (2) | ||
| US20130245096A1 (en) | COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA | |
| JP2015519057A5 (2) | ||
| JP2019506156A (ja) | Ｈｉｖ感染症のｒｎａ誘導型治療のための方法及び組成物 | |
| JP2016195616A5 (2) | ||
| MX2013009191A (es) | Oligonucleotidos antisentido. | |
| MX2024005652A (es) | Agentes de acido ribonucleico (arn) de cadena doble modificados. | |
| JP2015518714A (ja) | 遺伝子発現を調節するための組成物及び方法 | |
| PE20212131A1 (es) | Compuestos y metodos para reducir de la expresion de atxn3 | |
| JP2015532097A5 (2) | ||
| PH12021552257A1 (en) | Compound, method and pharmaceutical composition for modulating expression of dux4 | |
| NZ604094A (en) | Double stranded rna compounds to rhoa and use thereof | |
| WO2023013329A1 (ja) | ヘアピン核酸組成物 | |
| JP6049143B2 (ja) | オリゴヌクレオチド、グルココルチコイド感受性増強剤、医薬組成物、及び発現ベクター | |
| US20060217326A1 (en) | Group of nucleic acid fragments for prevention of hiv infection or aids and the usage thereof |