CN118987017A - 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 - Google Patents
用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 Download PDFInfo
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- CN118987017A CN118987017A CN202411184883.6A CN202411184883A CN118987017A CN 118987017 A CN118987017 A CN 118987017A CN 202411184883 A CN202411184883 A CN 202411184883A CN 118987017 A CN118987017 A CN 118987017A
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- antisense oligonucleotide
- nucleosides
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- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- 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
- C12N15/1137—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 against enzymes
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- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
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- 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
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- 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
- C12N2310/315—Phosphorothioates
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- 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
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- 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
- C12N2310/322—2'-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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862779916P | 2018-12-14 | 2018-12-14 | |
| US62/779,916 | 2018-12-14 | ||
| US201962807603P | 2019-02-19 | 2019-02-19 | |
| US62/807,603 | 2019-02-19 | ||
| US201962840879P | 2019-04-30 | 2019-04-30 | |
| US62/840,879 | 2019-04-30 | ||
| PCT/US2019/065936 WO2020123783A1 (en) | 2018-12-14 | 2019-12-12 | Compositions and methods for treating and preventing amyotrophic lateral sclerosis |
| CN201980080996.2A CN113330116A (zh) | 2018-12-14 | 2019-12-12 | 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980080996.2A Division CN113330116A (zh) | 2018-12-14 | 2019-12-12 | 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN118987017A true CN118987017A (zh) | 2024-11-22 |
Family
ID=69160351
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202411184883.6A Pending CN118987017A (zh) | 2018-12-14 | 2019-12-12 | 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 |
| CN201980080996.2A Pending CN113330116A (zh) | 2018-12-14 | 2019-12-12 | 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 |
| CN202411184930.7A Pending CN119185345A (zh) | 2018-12-14 | 2019-12-12 | 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980080996.2A Pending CN113330116A (zh) | 2018-12-14 | 2019-12-12 | 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 |
| CN202411184930.7A Pending CN119185345A (zh) | 2018-12-14 | 2019-12-12 | 用于治疗和预防肌肉萎缩性脊髓侧索硬化症的组合物和方法 |
Country Status (20)
| Country | Link |
|---|---|
| US (1) | US20220073930A1 (https=) |
| EP (1) | EP3894557A1 (https=) |
| JP (2) | JP7612575B2 (https=) |
| KR (2) | KR20250090384A (https=) |
| CN (3) | CN118987017A (https=) |
| AU (1) | AU2019396522B2 (https=) |
| BR (1) | BR112021011334A2 (https=) |
| CA (1) | CA3115549A1 (https=) |
| CL (1) | CL2021001136A1 (https=) |
| CO (1) | CO2021008187A2 (https=) |
| CR (1) | CR20210384A (https=) |
| IL (1) | IL283833A (https=) |
| JO (1) | JOP20210143A1 (https=) |
| MX (1) | MX2021005086A (https=) |
| MY (1) | MY209775A (https=) |
| PH (1) | PH12021551365A1 (https=) |
| SG (1) | SG11202103415WA (https=) |
| TW (1) | TWI906204B (https=) |
| UY (1) | UY38506A (https=) |
| WO (1) | WO2020123783A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| CN119679944A (zh) | 2018-01-25 | 2025-03-25 | 渤健马萨诸塞州股份有限公司 | 治疗脊髓性肌萎缩症的方法 |
| US12429487B2 (en) | 2018-12-06 | 2025-09-30 | Biogen Ma Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
| BR112023019877A2 (pt) * | 2021-03-31 | 2023-11-07 | Biogen Ma Inc | Tratamento de esclerose lateral amiotrófica |
| WO2025045130A1 (zh) * | 2023-08-29 | 2025-03-06 | 石药集团中奇制药技术（石家庄）有限公司 | 一种抑制sod1基因表达的dsrna分子及其应用 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2452458A1 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| WO2013134403A1 (en) * | 2012-03-06 | 2013-09-12 | The Washington University | Method of treating neurodegenerative diseases with microrna regulators |
| EP3119888B1 (en) * | 2014-03-19 | 2021-07-28 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| HUE050704T2 (hu) * | 2014-04-01 | 2020-12-28 | Biogen Ma Inc | Összetételek a SOD-1 expressziójának modulálására |
| WO2016077687A1 (en) * | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JP2020518258A (ja) * | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| WO2018218219A1 (en) * | 2017-05-26 | 2018-11-29 | University Of Miami | Determining onset of amyotrophic lateral sclerosis |
-
2019
- 2019-12-06 TW TW108144617A patent/TWI906204B/zh active
- 2019-12-11 UY UY0001038506A patent/UY38506A/es not_active Application Discontinuation
- 2019-12-12 JP JP2021525191A patent/JP7612575B2/ja active Active
- 2019-12-12 EP EP19836331.9A patent/EP3894557A1/en active Pending
- 2019-12-12 MX MX2021005086A patent/MX2021005086A/es unknown
- 2019-12-12 CR CR20210384A patent/CR20210384A/es unknown
- 2019-12-12 CA CA3115549A patent/CA3115549A1/en active Pending
- 2019-12-12 MY MYPI2021003225A patent/MY209775A/en unknown
- 2019-12-12 CN CN202411184883.6A patent/CN118987017A/zh active Pending
- 2019-12-12 SG SG11202103415WA patent/SG11202103415WA/en unknown
- 2019-12-12 KR KR1020257019188A patent/KR20250090384A/ko active Pending
- 2019-12-12 US US17/291,199 patent/US20220073930A1/en active Pending
- 2019-12-12 AU AU2019396522A patent/AU2019396522B2/en active Active
- 2019-12-12 WO PCT/US2019/065936 patent/WO2020123783A1/en not_active Ceased
- 2019-12-12 KR KR1020217020529A patent/KR102821047B1/ko active Active
- 2019-12-12 CN CN201980080996.2A patent/CN113330116A/zh active Pending
- 2019-12-12 CN CN202411184930.7A patent/CN119185345A/zh active Pending
- 2019-12-12 BR BR112021011334-7A patent/BR112021011334A2/pt unknown
-
2021
- 2021-04-29 CL CL2021001136A patent/CL2021001136A1/es unknown
- 2021-06-09 IL IL283833A patent/IL283833A/en unknown
- 2021-06-09 PH PH12021551365A patent/PH12021551365A1/en unknown
- 2021-06-09 JO JOP/2021/0143A patent/JOP20210143A1/ar unknown
- 2021-06-23 CO CONC2021/0008187A patent/CO2021008187A2/es unknown
-
2024
- 2024-12-06 JP JP2024213950A patent/JP2025023319A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20220073930A1 (en) | 2022-03-10 |
| KR20210131992A (ko) | 2021-11-03 |
| MX2021005086A (es) | 2021-08-16 |
| KR20250090384A (ko) | 2025-06-19 |
| TWI906204B (zh) | 2025-12-01 |
| EP3894557A1 (en) | 2021-10-20 |
| JOP20210143A1 (ar) | 2023-01-30 |
| UY38506A (es) | 2020-06-30 |
| CN113330116A (zh) | 2021-08-31 |
| JP7612575B2 (ja) | 2025-01-14 |
| TW202035694A (zh) | 2020-10-01 |
| BR112021011334A2 (pt) | 2021-09-08 |
| CA3115549A1 (en) | 2020-06-18 |
| MY209775A (en) | 2025-08-03 |
| CN119185345A (zh) | 2024-12-27 |
| KR102821047B1 (ko) | 2025-06-16 |
| CL2021001136A1 (es) | 2022-01-14 |
| AU2019396522B2 (en) | 2026-04-09 |
| JP2022513597A (ja) | 2022-02-09 |
| CR20210384A (es) | 2021-12-20 |
| SG11202103415WA (en) | 2021-06-29 |
| IL283833A (en) | 2021-07-29 |
| CO2021008187A2 (es) | 2021-09-09 |
| JP2025023319A (ja) | 2025-02-14 |
| WO2020123783A1 (en) | 2020-06-18 |
| PH12021551365A1 (en) | 2021-11-22 |
| AU2019396522A1 (en) | 2021-05-06 |
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