UY39104A - Compuestos y métodos para modular la expresión de scn1a - Google Patents
Compuestos y métodos para modular la expresión de scn1aInfo
- Publication number
- UY39104A UY39104A UY0001039104A UY39104A UY39104A UY 39104 A UY39104 A UY 39104A UY 0001039104 A UY0001039104 A UY 0001039104A UY 39104 A UY39104 A UY 39104A UY 39104 A UY39104 A UY 39104A
- Authority
- UY
- Uruguay
- Prior art keywords
- dysfunctions
- compounds
- methods
- pharmaceutical compositions
- modulate
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 3
- 101000631760 Homo sapiens Sodium channel protein type 1 subunit alpha Proteins 0.000 title abstract 2
- 102100028910 Sodium channel protein type 1 subunit alpha Human genes 0.000 title abstract 2
- 230000004064 dysfunction Effects 0.000 abstract 3
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 208000024891 symptom Diseases 0.000 abstract 2
- 206010010904 Convulsion Diseases 0.000 abstract 1
- 201000007547 Dravet syndrome Diseases 0.000 abstract 1
- 206010070246 Executive dysfunction Diseases 0.000 abstract 1
- 206010061296 Motor dysfunction Diseases 0.000 abstract 1
- 208000036572 Myoclonic epilepsy Diseases 0.000 abstract 1
- 206010073677 Severe myoclonic epilepsy of infancy Diseases 0.000 abstract 1
- 208000023944 Sudden Unexpected Death in Epilepsy Diseases 0.000 abstract 1
- 208000010877 cognitive disease Diseases 0.000 abstract 1
- 230000001934 delay Effects 0.000 abstract 1
- 230000003111 delayed effect Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000003181 encephalopathic effect Effects 0.000 abstract 1
- 230000001037 epileptic effect Effects 0.000 abstract 1
- 230000012010 growth Effects 0.000 abstract 1
- 230000010354 integration Effects 0.000 abstract 1
- 230000035764 nutrition Effects 0.000 abstract 1
- 235000016709 nutrition Nutrition 0.000 abstract 1
- 230000000399 orthopedic effect Effects 0.000 abstract 1
- 208000005809 status epilepticus Diseases 0.000 abstract 1
- 230000016776 visual perception Effects 0.000 abstract 1
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
- C12N15/1138—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 receptors or cell surface proteins
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
-
- 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
- C12N2310/33—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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3527—Other alkyl chain
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Peptides Or Proteins (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062983555P | 2020-02-28 | 2020-02-28 | |
| US202063085111P | 2020-09-29 | 2020-09-29 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| UY39104A true UY39104A (es) | 2021-08-31 |
Family
ID=77491960
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| UY0001039104A UY39104A (es) | 2020-02-28 | 2021-03-01 | Compuestos y métodos para modular la expresión de scn1a |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230043648A1 (https=) |
| EP (1) | EP4110919A4 (https=) |
| JP (1) | JP2023515974A (https=) |
| TW (1) | TW202140788A (https=) |
| UY (1) | UY39104A (https=) |
| WO (1) | WO2021174036A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7507093B2 (ja) * | 2018-03-21 | 2024-06-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法 |
| EP4392562A4 (en) * | 2021-08-27 | 2025-09-24 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6453212B2 (ja) * | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
| ES2935658T3 (es) * | 2016-07-15 | 2023-03-09 | Ionis Pharmaceuticals Inc | Compuestos y métodos para la modulación de SMN2 |
| SG11202001590RA (en) * | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| BR112021003224A2 (pt) * | 2018-08-20 | 2021-07-20 | Rogcon, Inc. | oligonucleotídeos antissentido direcionados a scn2a para o tratamento de encefalopatias por scn1a |
| BR112022010882A2 (pt) * | 2019-12-06 | 2022-10-04 | Stoke Therapeutics Inc | Oligômeros antissenso para tratamento de condições e doenças |
-
2021
- 2021-02-26 WO PCT/US2021/019960 patent/WO2021174036A1/en not_active Ceased
- 2021-02-26 TW TW110107122A patent/TW202140788A/zh unknown
- 2021-02-26 EP EP21760424.8A patent/EP4110919A4/en active Pending
- 2021-02-26 US US17/802,324 patent/US20230043648A1/en active Pending
- 2021-02-26 JP JP2022551593A patent/JP2023515974A/ja active Pending
- 2021-03-01 UY UY0001039104A patent/UY39104A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021174036A1 (en) | 2021-09-02 |
| TW202140788A (zh) | 2021-11-01 |
| EP4110919A1 (en) | 2023-01-04 |
| JP2023515974A (ja) | 2023-04-17 |
| US20230043648A1 (en) | 2023-02-09 |
| EP4110919A4 (en) | 2024-06-05 |
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