BR112022017066A2 - Ácido nucleico antissentido indutor de salto do éxon 51 - Google Patents
Ácido nucleico antissentido indutor de salto do éxon 51Info
- Publication number
- BR112022017066A2 BR112022017066A2 BR112022017066A BR112022017066A BR112022017066A2 BR 112022017066 A2 BR112022017066 A2 BR 112022017066A2 BR 112022017066 A BR112022017066 A BR 112022017066A BR 112022017066 A BR112022017066 A BR 112022017066A BR 112022017066 A2 BR112022017066 A2 BR 112022017066A2
- Authority
- BR
- Brazil
- Prior art keywords
- nucleic acid
- antisense nucleic
- exon skipping
- skipping
- exon
- Prior art date
Links
- 230000000692 anti-sense effect Effects 0.000 title abstract 3
- 108020004707 nucleic acids Proteins 0.000 title abstract 2
- 150000007523 nucleic acids Chemical class 0.000 title abstract 2
- 102000039446 nucleic acids Human genes 0.000 title abstract 2
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 2
- 239000003814 drug Substances 0.000 abstract 1
- 229940079593 drug Drugs 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
Classifications
-
- 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
- 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
-
- 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
- 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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/50—Physical structure
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020033483 | 2020-02-28 | ||
| PCT/JP2021/007286 WO2021172498A1 (ja) | 2020-02-28 | 2021-02-26 | エクソン５１のスキッピングを誘導するアンチセンス核酸 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022017066A2 true BR112022017066A2 (pt) | 2022-11-16 |
Family
ID=77491251
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022017066A BR112022017066A2 (pt) | 2020-02-28 | 2021-02-26 | Ácido nucleico antissentido indutor de salto do éxon 51 |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US20230140736A1 (enExample) |
| EP (1) | EP4112083A4 (enExample) |
| JP (3) | JPWO2021172498A1 (enExample) |
| KR (1) | KR20220145865A (enExample) |
| CN (1) | CN115210376A (enExample) |
| AU (1) | AU2021226089A1 (enExample) |
| BR (1) | BR112022017066A2 (enExample) |
| CA (1) | CA3173049A1 (enExample) |
| CL (3) | CL2022002279A1 (enExample) |
| CO (1) | CO2022013685A2 (enExample) |
| EC (1) | ECSP22074446A (enExample) |
| IL (1) | IL295967A (enExample) |
| MX (1) | MX2022010545A (enExample) |
| PE (1) | PE20230237A1 (enExample) |
| TW (2) | TWI901457B (enExample) |
| WO (1) | WO2021172498A1 (enExample) |
| ZA (1) | ZA202209294B (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL293997A (en) * | 2019-12-19 | 2022-08-01 | Nippon Shinyaku Co Ltd | Antistrand nucleic acids that allow exon skipping |
| EP4458833A4 (en) | 2021-12-27 | 2025-12-31 | Nippon Shinyaku Co Ltd | PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
| AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
| IL115849A0 (en) | 1994-11-03 | 1996-01-31 | Merz & Co Gmbh & Co | Tangential filtration preparation of liposomal drugs and liposome product thereof |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| EP2392660A3 (en) | 2002-11-25 | 2012-03-28 | Masafumi Matsuo | ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor |
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| WO2009005793A2 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
| ES2639852T3 (es) | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| 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 |
| AU2009310557B2 (en) | 2008-10-27 | 2014-09-11 | Academisch Ziekenhuis Leiden | Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CA3092114A1 (en) | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| CA2861247C (en) * | 2011-12-28 | 2021-11-16 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids |
| 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 |
| EP2870246B1 (en) * | 2012-07-03 | 2019-09-11 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| JP6519842B2 (ja) | 2013-10-04 | 2019-05-29 | 国立大学法人神戸大学 | 福山型筋ジストロフィー治療用アンチセンス核酸 |
| DK3118311T3 (en) | 2014-03-12 | 2019-03-11 | Nippon Shinyaku Co Ltd | Antisense nucleic acid |
| MX373052B (es) * | 2014-06-17 | 2020-05-11 | Nippon Shinyaku Co Ltd | Acidos nucleicos antisentido. |
| AU2016264471B2 (en) | 2015-05-19 | 2022-01-27 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| AU2016302009B2 (en) | 2015-08-05 | 2021-09-30 | Eisai R&D Management Co., Ltd. | Chiral reagents for preparation of homogeneous oligomers |
| HUE050061T2 (hu) * | 2015-09-15 | 2020-12-28 | Nippon Shinyaku Co Ltd | Antiszensz nukleinsav |
| IL295755A (en) | 2015-10-09 | 2022-10-01 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
| KR102810425B1 (ko) * | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR20240006023A (ko) | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| PT3554552T (pt) | 2016-12-19 | 2022-10-03 | Sarepta Therapeutics Inc | Conjugados de oligómero de skipping de exão para distrofia muscular |
| WO2019241385A2 (en) | 2018-06-13 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystropy |
-
2021
- 2021-02-26 IL IL295967A patent/IL295967A/en unknown
- 2021-02-26 KR KR1020227032708A patent/KR20220145865A/ko active Pending
- 2021-02-26 AU AU2021226089A patent/AU2021226089A1/en active Pending
- 2021-02-26 JP JP2022503739A patent/JPWO2021172498A1/ja active Pending
- 2021-02-26 CA CA3173049A patent/CA3173049A1/en active Pending
- 2021-02-26 BR BR112022017066A patent/BR112022017066A2/pt unknown
- 2021-02-26 CN CN202180017313.6A patent/CN115210376A/zh active Pending
- 2021-02-26 TW TW113145565A patent/TWI901457B/zh active
- 2021-02-26 TW TW110107021A patent/TWI868323B/zh active
- 2021-02-26 MX MX2022010545A patent/MX2022010545A/es unknown
- 2021-02-26 WO PCT/JP2021/007286 patent/WO2021172498A1/ja not_active Ceased
- 2021-02-26 PE PE2022001832A patent/PE20230237A1/es unknown
- 2021-02-26 US US17/802,720 patent/US20230140736A1/en active Pending
- 2021-02-26 EP EP21760603.7A patent/EP4112083A4/en active Pending
-
2022
- 2022-08-18 ZA ZA2022/09294A patent/ZA202209294B/en unknown
- 2022-08-22 CL CL2022002279A patent/CL2022002279A1/es unknown
- 2022-08-25 US US17/895,186 patent/US11781140B2/en active Active
- 2022-09-06 JP JP2022141656A patent/JP7292636B2/ja active Active
- 2022-09-23 EC ECSENADI202274446A patent/ECSP22074446A/es unknown
- 2022-09-23 CO CONC2022/0013685A patent/CO2022013685A2/es unknown
-
2024
- 2024-05-16 CL CL2024001472A patent/CL2024001472A1/es unknown
- 2024-05-16 CL CL2024001471A patent/CL2024001471A1/es unknown
-
2025
- 2025-09-24 JP JP2025157620A patent/JP2026001070A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| ZA202209294B (en) | 2024-04-24 |
| JP2026001070A (ja) | 2026-01-06 |
| PE20230237A1 (es) | 2023-02-07 |
| TWI868323B (zh) | 2025-01-01 |
| CA3173049A1 (en) | 2021-09-02 |
| US20230140736A1 (en) | 2023-05-04 |
| CO2022013685A2 (es) | 2022-10-11 |
| WO2021172498A1 (ja) | 2021-09-02 |
| KR20220145865A (ko) | 2022-10-31 |
| AU2021226089A1 (en) | 2022-09-15 |
| JP2022180420A (ja) | 2022-12-06 |
| EP4112083A4 (en) | 2024-06-26 |
| MX2022010545A (es) | 2022-09-21 |
| US20230097387A1 (en) | 2023-03-30 |
| CN115210376A (zh) | 2022-10-18 |
| JPWO2021172498A1 (enExample) | 2021-09-02 |
| TW202200162A (zh) | 2022-01-01 |
| CL2024001471A1 (es) | 2024-09-27 |
| JP7292636B2 (ja) | 2023-06-19 |
| ECSP22074446A (es) | 2022-10-31 |
| IL295967A (en) | 2022-10-01 |
| CL2022002279A1 (es) | 2023-02-10 |
| TWI901457B (zh) | 2025-10-11 |
| EP4112083A1 (en) | 2023-01-04 |
| TW202515585A (zh) | 2025-04-16 |
| CL2024001472A1 (es) | 2024-09-27 |
| US11781140B2 (en) | 2023-10-10 |
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