CR20210519A - Compuestos y métodos para reducir la expresión de kcnt1 - Google Patents
Compuestos y métodos para reducir la expresión de kcnt1Info
- Publication number
- CR20210519A CR20210519A CR20210519A CR20210519A CR20210519A CR 20210519 A CR20210519 A CR 20210519A CR 20210519 A CR20210519 A CR 20210519A CR 20210519 A CR20210519 A CR 20210519A CR 20210519 A CR20210519 A CR 20210519A
- Authority
- CR
- Costa Rica
- Prior art keywords
- compounds
- methods
- kcnt1
- reducing
- pharmaceutical compositions
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 4
- 239000008194 pharmaceutical composition Substances 0.000 abstract 3
- 101000944018 Homo sapiens Potassium channel subfamily T member 1 Proteins 0.000 abstract 2
- 208000035051 Malignant migrating focal seizures of infancy Diseases 0.000 abstract 2
- 102100033508 Potassium channel subfamily T member 1 Human genes 0.000 abstract 2
- 208000008233 autosomal dominant nocturnal frontal lobe epilepsy Diseases 0.000 abstract 2
- 208000013575 epilepsy of infancy with migrating focal seizures Diseases 0.000 abstract 2
- 230000000926 neurological effect Effects 0.000 abstract 2
- 208000024891 symptom Diseases 0.000 abstract 2
- 208000014644 Brain disease Diseases 0.000 abstract 1
- 206010010904 Convulsion Diseases 0.000 abstract 1
- 201000008009 Early infantile epileptic encephalopathy Diseases 0.000 abstract 1
- 206010071545 Early infantile epileptic encephalopathy with burst-suppression Diseases 0.000 abstract 1
- 208000032274 Encephalopathy Diseases 0.000 abstract 1
- 206010021750 Infantile Spasms Diseases 0.000 abstract 1
- 208000035899 Infantile spasms syndrome Diseases 0.000 abstract 1
- 201000006791 West syndrome Diseases 0.000 abstract 1
- 230000005856 abnormality Effects 0.000 abstract 1
- 230000003542 behavioural effect Effects 0.000 abstract 1
- 208000013257 developmental and epileptic encephalopathy Diseases 0.000 abstract 1
- 230000000694 effects Effects 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
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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
- C12N15/1138—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
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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
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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
- A61P25/08—Antiepileptics; Anticonvulsants
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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
- A61P25/24—Antidepressants
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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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
- C12N2310/33—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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
- C12N2310/351—Conjugate
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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
- 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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- 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
Abstract
Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o actividad del ARN de KCNT1 en una célula o sujeto, y en determinadas circunstancias reducir la cantidad de proteína de KCNT1 en una célula o un sujeto. Estos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o característica de una afección neurológica. Dichos síntomas y características incluyen convulsiones, encefalopatía y anomalías del comportamiento. Ejemplos no limitativos de afecciones neurológicas que se benefician de estos compuestos, métodos y composiciones farmacéuticas son la epilepsia de la infancia con crisis focales migratorias (EIMFS), la epilepsia del lóbulo frontal nocturna autosómica dominante (ADNFLE), el síndrome de West y el síndrome de Ohtahara.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962819344P | 2019-03-15 | 2019-03-15 | |
US201962884501P | 2019-08-08 | 2019-08-08 | |
PCT/US2020/022680 WO2020190740A1 (en) | 2019-03-15 | 2020-03-13 | Compounds and methods for reducing kcnt1 expression |
Publications (1)
Publication Number | Publication Date |
---|---|
CR20210519A true CR20210519A (es) | 2021-11-24 |
Family
ID=72520445
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CR20210519A CR20210519A (es) | 2019-03-15 | 2020-03-13 | Compuestos y métodos para reducir la expresión de kcnt1 |
Country Status (18)
Country | Link |
---|---|
US (1) | US20220177893A1 (es) |
EP (1) | EP3938514A4 (es) |
JP (1) | JP2022526267A (es) |
KR (1) | KR20210141983A (es) |
CN (2) | CN113661241A (es) |
AU (1) | AU2020241693B2 (es) |
BR (1) | BR112021015494A2 (es) |
CA (1) | CA3133247A1 (es) |
CL (1) | CL2021002398A1 (es) |
CO (1) | CO2021013371A2 (es) |
CR (1) | CR20210519A (es) |
IL (1) | IL285546A (es) |
JO (1) | JOP20210254A1 (es) |
MX (1) | MX2021011132A (es) |
PE (1) | PE20220168A1 (es) |
SG (1) | SG11202108625WA (es) |
TW (1) | TW202102675A (es) |
WO (1) | WO2020190740A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2020106816A1 (en) | 2018-11-21 | 2020-05-28 | Enanta Pharmaceuticals, Inc. | Functionalized heterocycles as antiviral agents |
WO2021188414A1 (en) | 2020-03-16 | 2021-09-23 | Enanta Pharmaceuticals, Inc. | Functionalized heterocyclic compounds as antiviral agents |
WO2023102490A1 (en) * | 2021-12-01 | 2023-06-08 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of epilepsies |
Family Cites Families (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20050288242A1 (en) * | 2001-05-18 | 2005-12-29 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of RAS gene expression using short interfering nucleic acid (siNA) |
US7601501B2 (en) * | 2006-08-11 | 2009-10-13 | The Scripps Research Institute | Controlling osteogenesis by inhibition of osteogenic suppressors |
JP6336755B2 (ja) * | 2010-11-12 | 2018-06-06 | ザ ジェネラル ホスピタル コーポレイション | ポリコームに関連する非コードrna |
US20200129538A1 (en) * | 2017-06-13 | 2020-04-30 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating conditions associated with gain-of-function mutations in kcnt1 |
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2020
- 2020-03-13 US US17/437,507 patent/US20220177893A1/en active Pending
- 2020-03-13 CR CR20210519A patent/CR20210519A/es unknown
- 2020-03-13 MX MX2021011132A patent/MX2021011132A/es unknown
- 2020-03-13 SG SG11202108625WA patent/SG11202108625WA/en unknown
- 2020-03-13 PE PE2021001518A patent/PE20220168A1/es unknown
- 2020-03-13 KR KR1020217032716A patent/KR20210141983A/ko unknown
- 2020-03-13 JP JP2021555448A patent/JP2022526267A/ja active Pending
- 2020-03-13 WO PCT/US2020/022680 patent/WO2020190740A1/en active Application Filing
- 2020-03-13 EP EP20773689.3A patent/EP3938514A4/en active Pending
- 2020-03-13 CA CA3133247A patent/CA3133247A1/en active Pending
- 2020-03-13 CN CN202080020884.0A patent/CN113661241A/zh active Pending
- 2020-03-13 AU AU2020241693A patent/AU2020241693B2/en active Active
- 2020-03-13 CN CN202311150264.0A patent/CN117106778A/zh active Pending
- 2020-03-13 BR BR112021015494-9A patent/BR112021015494A2/pt unknown
- 2020-03-13 JO JOP/2021/0254A patent/JOP20210254A1/ar unknown
- 2020-03-16 TW TW109108639A patent/TW202102675A/zh unknown
-
2021
- 2021-08-11 IL IL285546A patent/IL285546A/en unknown
- 2021-09-14 CL CL2021002398A patent/CL2021002398A1/es unknown
- 2021-10-06 CO CONC2021/0013371A patent/CO2021013371A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
MX2021011132A (es) | 2021-10-14 |
JP2022526267A (ja) | 2022-05-24 |
BR112021015494A2 (pt) | 2021-10-05 |
EP3938514A1 (en) | 2022-01-19 |
CO2021013371A2 (es) | 2021-10-20 |
CL2021002398A1 (es) | 2022-06-03 |
SG11202108625WA (en) | 2021-09-29 |
PE20220168A1 (es) | 2022-01-28 |
AU2020241693A1 (en) | 2021-09-02 |
US20220177893A1 (en) | 2022-06-09 |
EP3938514A4 (en) | 2023-05-03 |
CN113661241A (zh) | 2021-11-16 |
IL285546A (en) | 2021-09-30 |
JOP20210254A1 (ar) | 2023-01-30 |
CN117106778A (zh) | 2023-11-24 |
TW202102675A (zh) | 2021-01-16 |
KR20210141983A (ko) | 2021-11-23 |
AU2020241693B2 (en) | 2024-01-04 |
WO2020190740A1 (en) | 2020-09-24 |
CA3133247A1 (en) | 2020-09-24 |
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