IL310811A - Compounds and Methods for Modulating SCN1A Expression - Google Patents
Compounds and Methods for Modulating SCN1A ExpressionInfo
- Publication number
- IL310811A IL310811A IL310811A IL31081124A IL310811A IL 310811 A IL310811 A IL 310811A IL 310811 A IL310811 A IL 310811A IL 31081124 A IL31081124 A IL 31081124A IL 310811 A IL310811 A IL 310811A
- Authority
- IL
- Israel
- Prior art keywords
- modulating
- compounds
- methods
- scn1a expression
- scn1a
- Prior art date
Links
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
-
- 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
-
- 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
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
-
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- 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
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
-
- 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
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Pain & Pain Management (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163237898P | 2021-08-27 | 2021-08-27 | |
| PCT/US2022/075493 WO2023028575A2 (en) | 2021-08-27 | 2022-08-26 | Compounds and methods for modulating scn1a expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL310811A true IL310811A (en) | 2024-04-01 |
Family
ID=85322277
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL310811A IL310811A (en) | 2021-08-27 | 2022-08-26 | Compounds and Methods for Modulating SCN1A Expression |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240360449A1 (enExample) |
| EP (1) | EP4392562A4 (enExample) |
| JP (1) | JP2024534157A (enExample) |
| KR (1) | KR20240049349A (enExample) |
| CN (1) | CN118451183A (enExample) |
| AU (1) | AU2022334739A1 (enExample) |
| CA (1) | CA3230299A1 (enExample) |
| IL (1) | IL310811A (enExample) |
| MX (1) | MX2024002553A (enExample) |
| WO (1) | WO2023028575A2 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| US12338437B2 (en) | 2020-05-11 | 2025-06-24 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| CN116334086A (zh) * | 2023-03-29 | 2023-06-27 | 广州医科大学附属第二医院 | 一种用于修复或抑制SCN1A基因异常剪接的U1-snRNA及其用途 |
| KR20260055532A (ko) * | 2023-07-24 | 2026-04-23 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료 방법 |
| WO2026052819A1 (en) * | 2024-09-06 | 2026-03-12 | Ospedale San Raffaele S.R.L. | Gene therapy |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20190321387A1 (en) * | 2016-07-15 | 2019-10-24 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of transcript processing |
| KR20260009385A (ko) * | 2017-08-25 | 2026-01-19 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| JP2022537581A (ja) * | 2019-06-21 | 2022-08-26 | クラリス コーポレーション | Ｐｐｍ１阻害剤およびその使用方法 |
| KR20220062517A (ko) * | 2019-08-15 | 2022-05-17 | 아이오니스 파마수티컬즈, 인코포레이티드 | 결합 변형된 올리고머 화합물 및 이의 용도 |
| KR20220113743A (ko) * | 2019-12-06 | 2022-08-16 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| JP2023515974A (ja) * | 2020-02-28 | 2023-04-17 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | Ｓｃｎ１ａ発現を調節するための化合物及び方法 |
-
2022
- 2022-08-26 US US18/685,362 patent/US20240360449A1/en active Pending
- 2022-08-26 WO PCT/US2022/075493 patent/WO2023028575A2/en not_active Ceased
- 2022-08-26 CN CN202280065211.6A patent/CN118451183A/zh active Pending
- 2022-08-26 EP EP22862285.8A patent/EP4392562A4/en active Pending
- 2022-08-26 IL IL310811A patent/IL310811A/en unknown
- 2022-08-26 AU AU2022334739A patent/AU2022334739A1/en active Pending
- 2022-08-26 MX MX2024002553A patent/MX2024002553A/es unknown
- 2022-08-26 JP JP2024513011A patent/JP2024534157A/ja active Pending
- 2022-08-26 CA CA3230299A patent/CA3230299A1/en active Pending
- 2022-08-26 KR KR1020247009728A patent/KR20240049349A/ko active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4392562A2 (en) | 2024-07-03 |
| CN118451183A (zh) | 2024-08-06 |
| JP2024534157A (ja) | 2024-09-18 |
| EP4392562A4 (en) | 2025-09-24 |
| CA3230299A1 (en) | 2023-03-02 |
| US20240360449A1 (en) | 2024-10-31 |
| WO2023028575A2 (en) | 2023-03-02 |
| AU2022334739A1 (en) | 2024-02-29 |
| MX2024002553A (es) | 2024-03-13 |
| KR20240049349A (ko) | 2024-04-16 |
| WO2023028575A3 (en) | 2023-04-13 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL286681B1 (en) | Compounds and methods for modulating UBE3A-ATS | |
| IL287398A (en) | Methods and preparations for modulating composition and translation | |
| PH12019502646A1 (en) | Pyrazole magl inhibitors | |
| UY34325A (es) | ?composiciones y métodos para controlar malezas comprendiendo un polinucleótido y agente de transferencia, y que modulan 5?enolpiruvilshikimato?3?fosfato sintasa? | |
| EP3137605A4 (en) | Compositions and methods for modulating angiopoietin-like 3 expression | |
| PH12019501102A1 (en) | Magl inhibitors | |
| IL296413B2 (en) | GPR52 modulator compounds | |
| IL307625A (en) | Compounds and methods for modulating pnpla3 expression | |
| MX2019000619A (es) | Compuestos y metodos para la modulacion de smn2. | |
| JOP20190106A1 (ar) | مثبطات أحادي أسيل جليسرول ليباز (magl) | |
| SMT202200294T1 (it) | Derivati di n-(fenil)-2-(fenil)pirimidina-4-carbossammide e composti correlati come inibitori di hpk1 per trattare il cancro | |
| ZA202102609B (en) | Compounds, compositions, and methods for modulating cdk9 activity | |
| HUE068561T2 (hu) | Az angiotenzinogén expressziójának módosítására szolgáló vegyületek és eljárások | |
| IL286733A (en) | Compound, method and pharmaceutical composition for dux4 expression adjustment | |
| IL304611A (en) | gcn2-modulating compounds and uses thereof | |
| IL301007B1 (en) | Gpr52 modulator compounds | |
| EP4110919A4 (en) | COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION | |
| HUE072835T2 (hu) | Vegyületek és eljárások a GFAP modulálására | |
| SMT202500449T1 (it) | Composti modulatori di gpr52 | |
| HK40109366A (en) | Compounds and methods for modulating scn1a expression | |
| CA3283069A1 (en) | Compounds and methods for modulating alpha-synuclein expression | |
| GB202105605D0 (en) | Microbiota modulation | |
| HK40099806A (en) | Compositions and methods for modulating pnpla3 expression | |
| HK40079948A (en) | Compounds and methods for modulating smn2 | |
| IL313343A (en) | Compounds and methods for modulating glycogen synthesis 1 |