PE20220168A1 - Compuestos y metodos para reducir la expresion de kcnt1 - Google Patents
Compuestos y metodos para reducir la expresion de kcnt1Info
- Publication number
- PE20220168A1 PE20220168A1 PE2021001518A PE2021001518A PE20220168A1 PE 20220168 A1 PE20220168 A1 PE 20220168A1 PE 2021001518 A PE2021001518 A PE 2021001518A PE 2021001518 A PE2021001518 A PE 2021001518A PE 20220168 A1 PE20220168 A1 PE 20220168A1
- Authority
- PE
- Peru
- Prior art keywords
- kcnt1
- modified
- seizures
- reducing
- amount
- Prior art date
Links
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
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/341—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
-
- 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
- C12N2310/351—Conjugate
-
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Pain & Pain Management (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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 |
|---|---|
| PE20220168A1 true PE20220168A1 (es) | 2022-01-28 |
Family
ID=72520445
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2021001518A PE20220168A1 (es) | 2019-03-15 | 2020-03-13 | Compuestos y metodos para reducir la expresion de kcnt1 |
Country Status (19)
| Country | Link |
|---|---|
| US (2) | US20220177893A1 (https=) |
| EP (1) | EP3938514A4 (https=) |
| JP (2) | JP7564817B2 (https=) |
| KR (1) | KR20210141983A (https=) |
| CN (3) | CN113661241A (https=) |
| AU (1) | AU2020241693B2 (https=) |
| BR (1) | BR112021015494A2 (https=) |
| CA (1) | CA3133247A1 (https=) |
| CL (1) | CL2021002398A1 (https=) |
| CO (1) | CO2021013371A2 (https=) |
| CR (1) | CR20210519A (https=) |
| IL (1) | IL285546A (https=) |
| JO (1) | JOP20210254A1 (https=) |
| MX (1) | MX2021011132A (https=) |
| PE (1) | PE20220168A1 (https=) |
| PH (1) | PH12021552220A1 (https=) |
| SG (1) | SG11202108625WA (https=) |
| TW (1) | TW202102675A (https=) |
| WO (1) | WO2020190740A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202001685TA (en) | 2017-08-28 | 2020-03-30 | Enanta Pharm Inc | Hepatitis b antiviral agents |
| IL283190B2 (en) | 2018-11-21 | 2025-08-01 | Enanta Pharm Inc | Functionalized heterocycles as antiviral agents |
| WO2021188414A1 (en) | 2020-03-16 | 2021-09-23 | Enanta Pharmaceuticals, Inc. | Functionalized heterocyclic compounds as antiviral agents |
| EP4441229A1 (en) * | 2021-12-01 | 2024-10-09 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of epilepsies |
| IL326478A (en) * | 2023-08-16 | 2026-04-01 | Servier Lab | Oligonucleotides for modulation of KCNT1 expression |
Family Cites Families (5)
| 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 |
| CA2817256A1 (en) * | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| TW202246503A (zh) | 2013-07-19 | 2022-12-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| 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 |
-
2020
- 2020-03-13 CR CR20210519A patent/CR20210519A/es unknown
- 2020-03-13 PH PH1/2021/552220A patent/PH12021552220A1/en unknown
- 2020-03-13 EP EP20773689.3A patent/EP3938514A4/en active Pending
- 2020-03-13 US US17/437,507 patent/US20220177893A1/en not_active Abandoned
- 2020-03-13 CN CN202080020884.0A patent/CN113661241A/zh active Pending
- 2020-03-13 JP JP2021555448A patent/JP7564817B2/ja active Active
- 2020-03-13 CN CN202511762645.3A patent/CN121674396A/zh active Pending
- 2020-03-13 CN CN202311150264.0A patent/CN117106778A/zh active Pending
- 2020-03-13 AU AU2020241693A patent/AU2020241693B2/en not_active Ceased
- 2020-03-13 CA CA3133247A patent/CA3133247A1/en active Pending
- 2020-03-13 SG SG11202108625WA patent/SG11202108625WA/en unknown
- 2020-03-13 MX MX2021011132A patent/MX2021011132A/es unknown
- 2020-03-13 BR BR112021015494-9A patent/BR112021015494A2/pt unknown
- 2020-03-13 PE PE2021001518A patent/PE20220168A1/es unknown
- 2020-03-13 WO PCT/US2020/022680 patent/WO2020190740A1/en not_active Ceased
- 2020-03-13 KR KR1020217032716A patent/KR20210141983A/ko not_active Withdrawn
- 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
- 2021-09-14 JO JOP/2021/0254A patent/JOP20210254A1/ar unknown
- 2021-10-06 CO CONC2021/0013371A patent/CO2021013371A2/es unknown
-
2024
- 2024-09-27 JP JP2024168231A patent/JP2025000805A/ja active Pending
-
2025
- 2025-06-27 US US19/252,993 patent/US20250320505A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020190740A1 (en) | 2020-09-24 |
| CR20210519A (es) | 2021-11-24 |
| CO2021013371A2 (es) | 2021-10-20 |
| CN117106778A (zh) | 2023-11-24 |
| SG11202108625WA (en) | 2021-09-29 |
| CN121674396A (zh) | 2026-03-17 |
| CN113661241A (zh) | 2021-11-16 |
| TW202102675A (zh) | 2021-01-16 |
| JP7564817B2 (ja) | 2024-10-09 |
| JP2022526267A (ja) | 2022-05-24 |
| AU2020241693B2 (en) | 2024-01-04 |
| BR112021015494A2 (pt) | 2021-10-05 |
| US20250320505A1 (en) | 2025-10-16 |
| CL2021002398A1 (es) | 2022-06-03 |
| JP2025000805A (ja) | 2025-01-07 |
| CA3133247A1 (en) | 2020-09-24 |
| PH12021552220A1 (en) | 2022-05-30 |
| MX2021011132A (es) | 2021-10-14 |
| US20220177893A1 (en) | 2022-06-09 |
| AU2020241693A1 (en) | 2021-09-02 |
| EP3938514A4 (en) | 2023-05-03 |
| EP3938514A1 (en) | 2022-01-19 |
| IL285546A (en) | 2021-09-30 |
| KR20210141983A (ko) | 2021-11-23 |
| JOP20210254A1 (ar) | 2023-01-30 |
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