BR112023019877A2 - Tratamento de esclerose lateral amiotrófica - Google Patents
Tratamento de esclerose lateral amiotróficaInfo
- Publication number
- BR112023019877A2 BR112023019877A2 BR112023019877A BR112023019877A BR112023019877A2 BR 112023019877 A2 BR112023019877 A2 BR 112023019877A2 BR 112023019877 A BR112023019877 A BR 112023019877A BR 112023019877 A BR112023019877 A BR 112023019877A BR 112023019877 A2 BR112023019877 A2 BR 112023019877A2
- Authority
- BR
- Brazil
- Prior art keywords
- treatment
- lateral sclerosis
- amyotrophic lateral
- sod1
- amiotrophic lateral
- Prior art date
Links
- 201000010901 lateral sclerosis Diseases 0.000 title 1
- 208000005264 motor neuron disease Diseases 0.000 title 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 abstract 3
- 101100203568 Halobacterium salinarum (strain ATCC 700922 / JCM 11081 / NRC-1) sod2 gene Proteins 0.000 abstract 2
- 101100111809 Schizosaccharomyces pombe (strain 972 / ATCC 24843) bsu1 gene Proteins 0.000 abstract 2
- 101150062190 sod1 gene Proteins 0.000 abstract 2
- 101150048620 sodN gene Proteins 0.000 abstract 2
- 101000979333 Homo sapiens Neurofilament light polypeptide Proteins 0.000 abstract 1
- 102100023057 Neurofilament light polypeptide Human genes 0.000 abstract 1
- 108091034117 Oligonucleotide Proteins 0.000 abstract 1
- 102000008221 Superoxide Dismutase-1 Human genes 0.000 abstract 1
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
Classifications
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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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- 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
- 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
- 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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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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
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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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3531—Hydrogen
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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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Investigating Or Analysing Biological Materials (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163168972P | 2021-03-31 | 2021-03-31 | |
| PCT/US2022/022485 WO2022212459A1 (en) | 2021-03-31 | 2022-03-30 | Treatment of amyotrophic lateral sclerosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023019877A2 true BR112023019877A2 (pt) | 2023-11-07 |
Family
ID=81346346
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023019877A BR112023019877A2 (pt) | 2021-03-31 | 2022-03-30 | Tratamento de esclerose lateral amiotrófica |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240182903A1 (https=) |
| EP (1) | EP4314294A1 (https=) |
| JP (1) | JP2024511767A (https=) |
| KR (1) | KR20230172502A (https=) |
| CN (1) | CN117062911A (https=) |
| AU (1) | AU2022252236A1 (https=) |
| BR (1) | BR112023019877A2 (https=) |
| CA (1) | CA3214585A1 (https=) |
| MX (1) | MX2023011214A (https=) |
| TW (1) | TW202304471A (https=) |
| WO (1) | WO2022212459A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN120350010B (zh) * | 2025-06-24 | 2025-09-09 | 羿美诚健(上海)生物医药有限公司 | 靶向PIKfyve基因的小核酸及其药物组合物和用途 |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2452458A1 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| HUE050704T2 (hu) | 2014-04-01 | 2020-12-28 | Biogen Ma Inc | Összetételek a SOD-1 expressziójának modulálására |
| TWI906204B (zh) * | 2018-12-14 | 2025-12-01 | 美商百健Ｍａ公司 | 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法 |
-
2022
- 2022-03-30 BR BR112023019877A patent/BR112023019877A2/pt unknown
- 2022-03-30 JP JP2023557719A patent/JP2024511767A/ja active Pending
- 2022-03-30 CN CN202280024040.2A patent/CN117062911A/zh active Pending
- 2022-03-30 EP EP22717995.9A patent/EP4314294A1/en active Pending
- 2022-03-30 US US18/283,526 patent/US20240182903A1/en active Pending
- 2022-03-30 TW TW111112225A patent/TW202304471A/zh unknown
- 2022-03-30 AU AU2022252236A patent/AU2022252236A1/en active Pending
- 2022-03-30 CA CA3214585A patent/CA3214585A1/en active Pending
- 2022-03-30 WO PCT/US2022/022485 patent/WO2022212459A1/en not_active Ceased
- 2022-03-30 KR KR1020237037099A patent/KR20230172502A/ko active Pending
- 2022-03-30 MX MX2023011214A patent/MX2023011214A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230172502A (ko) | 2023-12-22 |
| WO2022212459A1 (en) | 2022-10-06 |
| US20240182903A1 (en) | 2024-06-06 |
| AU2022252236A1 (en) | 2023-10-12 |
| TW202304471A (zh) | 2023-02-01 |
| CN117062911A (zh) | 2023-11-14 |
| EP4314294A1 (en) | 2024-02-07 |
| AU2022252236A9 (en) | 2023-10-19 |
| JP2024511767A (ja) | 2024-03-15 |
| CA3214585A1 (en) | 2022-10-06 |
| MX2023011214A (es) | 2023-12-08 |
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