TWI874546B - 誘導外顯子５０的跳讀的反義核酸 - Google Patents
誘導外顯子５０的跳讀的反義核酸 Download PDFInfo
- Publication number
- TWI874546B TWI874546B TW109146242A TW109146242A TWI874546B TW I874546 B TWI874546 B TW I874546B TW 109146242 A TW109146242 A TW 109146242A TW 109146242 A TW109146242 A TW 109146242A TW I874546 B TWI874546 B TW I874546B
- Authority
- TW
- Taiwan
- Prior art keywords
- antisense oligomer
- exon
- pharmaceutically acceptable
- hydrate
- acceptable salt
- Prior art date
Links
Classifications
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/4965—Non-condensed pyrazines
-
- 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
- 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
-
- 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
- C12N2310/30—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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
-
- 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
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (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 |
|---|---|---|---|
| JP2019-236704 | 2019-12-26 | ||
| JP2019236704 | 2019-12-26 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| TW202138560A TW202138560A (zh) | 2021-10-16 |
| TWI874546B true TWI874546B (zh) | 2025-03-01 |
Family
ID=76574765
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW109146242A TWI874546B (zh) | 2019-12-26 | 2020-12-25 | 誘導外顯子５０的跳讀的反義核酸 |
| TW114101879A TW202523843A (zh) | 2019-12-26 | 2020-12-25 | 誘導外顯子５０的跳讀的反義核酸 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW114101879A TW202523843A (zh) | 2019-12-26 | 2020-12-25 | 誘導外顯子５０的跳讀的反義核酸 |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US20230073008A1 (https=) |
| EP (1) | EP4083208A4 (https=) |
| JP (2) | JPWO2021132591A1 (https=) |
| KR (1) | KR20220122673A (https=) |
| CN (3) | CN114901823A (https=) |
| AU (1) | AU2020411964A1 (https=) |
| BR (1) | BR112022012622A2 (https=) |
| CA (1) | CA3165961A1 (https=) |
| CO (1) | CO2022008664A2 (https=) |
| IL (1) | IL294271A (https=) |
| MX (1) | MX2022007937A (https=) |
| MY (1) | MY209476A (https=) |
| PH (1) | PH12022551431A1 (https=) |
| TW (2) | TWI874546B (https=) |
| WO (1) | WO2021132591A1 (https=) |
| ZA (1) | ZA202206667B (https=) |
Families Citing this family (4)
| 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 |
| CN114901823A (zh) * | 2019-12-26 | 2022-08-12 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
| 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 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW201336859A (zh) * | 2011-12-28 | 2013-09-16 | Nippon Shinyaku Co Ltd | 反意義核酸 |
| TW201534719A (zh) * | 2008-10-24 | 2015-09-16 | Sarepta Therapeutics Inc | 用於杜顯氏肌肉萎縮症（ｄｍｄ）之多重外顯子跳躍組合物 |
Family Cites Families (26)
| 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 |
| 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 |
| 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 |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| 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 |
| CN101388842B (zh) | 2008-10-30 | 2012-04-04 | 华为技术有限公司 | 一种存储方法和装置 |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| 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 |
| US20140329762A1 (en) * | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| 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 |
| IL295755A (en) | 2015-10-09 | 2022-10-01 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| EP3858993A1 (en) * | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| 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 |
| IL318474A (en) | 2018-12-13 | 2025-03-01 | Sarepta Therapeutics Inc | Axon-skipping oligomer complexes for muscular dystrophy |
| CN114901823A (zh) * | 2019-12-26 | 2022-08-12 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
-
2020
- 2020-12-25 CN CN202080090310.0A patent/CN114901823A/zh active Pending
- 2020-12-25 PH PH1/2022/551431A patent/PH12022551431A1/en unknown
- 2020-12-25 MY MYPI2022003210A patent/MY209476A/en unknown
- 2020-12-25 KR KR1020227024957A patent/KR20220122673A/ko active Pending
- 2020-12-25 MX MX2022007937A patent/MX2022007937A/es unknown
- 2020-12-25 BR BR112022012622A patent/BR112022012622A2/pt unknown
- 2020-12-25 TW TW109146242A patent/TWI874546B/zh active
- 2020-12-25 CN CN202410219725.3A patent/CN118109468A/zh active Pending
- 2020-12-25 AU AU2020411964A patent/AU2020411964A1/en active Pending
- 2020-12-25 JP JP2021567687A patent/JPWO2021132591A1/ja not_active Withdrawn
- 2020-12-25 TW TW114101879A patent/TW202523843A/zh unknown
- 2020-12-25 WO PCT/JP2020/048803 patent/WO2021132591A1/ja not_active Ceased
- 2020-12-25 EP EP20906005.2A patent/EP4083208A4/en active Pending
- 2020-12-25 CN CN202410219814.8A patent/CN118109469A/zh active Pending
- 2020-12-25 US US17/788,826 patent/US20230073008A1/en not_active Abandoned
- 2020-12-25 CA CA3165961A patent/CA3165961A1/en active Pending
- 2020-12-25 IL IL294271A patent/IL294271A/en unknown
-
2022
- 2022-06-15 ZA ZA2022/06667A patent/ZA202206667B/en unknown
- 2022-06-16 JP JP2022097020A patent/JP7201192B2/ja active Active
- 2022-06-22 CO CONC2022/0008664A patent/CO2022008664A2/es unknown
- 2022-06-23 US US17/847,333 patent/US11655472B2/en active Active
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW201534719A (zh) * | 2008-10-24 | 2015-09-16 | Sarepta Therapeutics Inc | 用於杜顯氏肌肉萎縮症（ｄｍｄ）之多重外顯子跳躍組合物 |
| TW201336859A (zh) * | 2011-12-28 | 2013-09-16 | Nippon Shinyaku Co Ltd | 反意義核酸 |
Also Published As
| Publication number | Publication date |
|---|---|
| PH12022551431A1 (en) | 2023-11-20 |
| JP2022133312A (ja) | 2022-09-13 |
| EP4083208A1 (en) | 2022-11-02 |
| US11655472B2 (en) | 2023-05-23 |
| ZA202206667B (en) | 2023-12-20 |
| MX2022007937A (es) | 2022-07-27 |
| US20230073008A1 (en) | 2023-03-09 |
| JP7201192B2 (ja) | 2023-01-10 |
| CA3165961A1 (en) | 2021-07-01 |
| CO2022008664A2 (es) | 2022-07-19 |
| WO2021132591A1 (ja) | 2021-07-01 |
| BR112022012622A2 (pt) | 2022-09-06 |
| MY209476A (en) | 2025-07-11 |
| TW202138560A (zh) | 2021-10-16 |
| KR20220122673A (ko) | 2022-09-02 |
| JPWO2021132591A1 (https=) | 2021-07-01 |
| TW202523843A (zh) | 2025-06-16 |
| US20220333112A1 (en) | 2022-10-20 |
| CN118109468A (zh) | 2024-05-31 |
| CN114901823A (zh) | 2022-08-12 |
| IL294271A (en) | 2022-08-01 |
| CN118109469A (zh) | 2024-05-31 |
| EP4083208A4 (en) | 2024-01-03 |
| AU2020411964A1 (en) | 2022-06-16 |
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