JP2025513757A - SNCA関連疾患を処置するためのSNCA標的化siRNA組成物 - Google Patents
SNCA関連疾患を処置するためのSNCA標的化siRNA組成物 Download PDFInfo
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- JP2025513757A JP2025513757A JP2024558028A JP2024558028A JP2025513757A JP 2025513757 A JP2025513757 A JP 2025513757A JP 2024558028 A JP2024558028 A JP 2024558028A JP 2024558028 A JP2024558028 A JP 2024558028A JP 2025513757 A JP2025513757 A JP 2025513757A
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- Prior art keywords
- phosphate
- nucleotide
- nucleotides
- seq
- snca
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- 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
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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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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- 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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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2310/3521—Methyl
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- C—CHEMISTRY; METALLURGY
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- 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
- C12N2310/3527—Other alkyl chain
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2310/3533—Halogen
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263326770P | 2022-04-01 | 2022-04-01 | |
| US63/326,770 | 2022-04-01 | ||
| US202263326813P | 2022-04-02 | 2022-04-02 | |
| US63/326,813 | 2022-04-02 | ||
| PCT/US2023/065196 WO2023192977A2 (en) | 2022-04-01 | 2023-03-31 | Snca-targeting sirna compositions for treating snca-associated disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2025513757A true JP2025513757A (ja) | 2025-04-30 |
| JP2025513757A5 JP2025513757A5 (https=) | 2026-04-03 |
| JPWO2023192977A5 JPWO2023192977A5 (https=) | 2026-04-03 |
Family
ID=88203639
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024558028A Pending JP2025513757A (ja) | 2022-04-01 | 2023-03-31 | SNCA関連疾患を処置するためのSNCA標的化siRNA組成物 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20250059541A1 (https=) |
| EP (1) | EP4504938A2 (https=) |
| JP (1) | JP2025513757A (https=) |
| KR (1) | KR20240166577A (https=) |
| CN (1) | CN119156448A (https=) |
| AU (1) | AU2023241905A1 (https=) |
| CA (1) | CA3254960A1 (https=) |
| CL (1) | CL2024002960A1 (https=) |
| CO (1) | CO2024012499A2 (https=) |
| IL (1) | IL315594A (https=) |
| MX (1) | MX2024011539A (https=) |
| TW (1) | TW202407100A (https=) |
| WO (1) | WO2023192977A2 (https=) |
-
2023
- 2023-03-31 CA CA3254960A patent/CA3254960A1/en active Pending
- 2023-03-31 JP JP2024558028A patent/JP2025513757A/ja active Pending
- 2023-03-31 AU AU2023241905A patent/AU2023241905A1/en active Pending
- 2023-03-31 EP EP23782086.5A patent/EP4504938A2/en active Pending
- 2023-03-31 IL IL315594A patent/IL315594A/en unknown
- 2023-03-31 CN CN202380038419.3A patent/CN119156448A/zh active Pending
- 2023-03-31 KR KR1020247036545A patent/KR20240166577A/ko active Pending
- 2023-03-31 MX MX2024011539A patent/MX2024011539A/es unknown
- 2023-03-31 WO PCT/US2023/065196 patent/WO2023192977A2/en not_active Ceased
- 2023-03-31 TW TW112112698A patent/TW202407100A/zh unknown
-
2024
- 2024-09-13 CO CONC2024/0012499A patent/CO2024012499A2/es unknown
- 2024-09-27 US US18/898,882 patent/US20250059541A1/en active Pending
- 2024-10-01 CL CL2024002960A patent/CL2024002960A1/es unknown
- 2024-12-12 US US18/978,602 patent/US20250115911A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CO2024012499A2 (es) | 2024-10-10 |
| CA3254960A1 (en) | 2023-10-05 |
| WO2023192977A2 (en) | 2023-10-05 |
| MX2024011539A (es) | 2024-09-26 |
| CL2024002960A1 (es) | 2025-03-07 |
| AU2023241905A1 (en) | 2024-09-26 |
| IL315594A (en) | 2024-11-01 |
| CN119156448A (zh) | 2024-12-17 |
| EP4504938A2 (en) | 2025-02-12 |
| KR20240166577A (ko) | 2024-11-26 |
| TW202407100A (zh) | 2024-02-16 |
| US20250059541A1 (en) | 2025-02-20 |
| US20250115911A1 (en) | 2025-04-10 |
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