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
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)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
Abstract
Se proporcionan compuestos, métodos y composiciones farmacéuticas para modular la proteína y/o ARN de SCN1A en una célula o sujeto. Tales compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma de una enfermedad encefalopática epiléptica o del desarrollo, tal como el síndrome de Dravet. Dichos síntomas incluyen convulsiones, muerte súbita inesperada en la epilepsia, estado epiléptico, retrasos en el comportamiento y el desarrollo, disfunciones del movimiento y el equilibrio, afecciones ortopédicas, disfunciones motoras y cognitivas, problemas de retraso en el lenguaje y el habla, disfunciones de integración visomotora, disfunciones de percepción visual, disfunciones ejecutivas, problemas de crecimiento y nutrición.
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 (es) |
EP (1) | EP4110919A4 (es) |
JP (1) | JP2023515974A (es) |
TW (1) | TW202140788A (es) |
UY (1) | UY39104A (es) |
WO (1) | WO2021174036A1 (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2019239971A1 (en) * | 2018-03-21 | 2020-09-03 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
IL310811A (en) * | 2021-08-27 | 2024-04-01 | Ionis Pharmaceuticals Inc | Compounds and Methods for Modulating SCN1A Expression |
Family Cites Families (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN111278991B (zh) * | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
EP3840733A4 (en) * | 2018-08-20 | 2022-07-20 | Rogcon, Inc. | ANTISENSE OLIGONUCLEOTIDES DIRECTED TO SCN2A FOR THE TREATMENT OF SCN1A ENCEPHALOPATHIES |
CA3159162A1 (en) * | 2019-12-06 | 2021-06-10 | Isabel AZNAREZ | Antisense oligomers for treatment of conditions and diseases |
-
2021
- 2021-02-26 EP EP21760424.8A patent/EP4110919A4/en active Pending
- 2021-02-26 TW TW110107122A patent/TW202140788A/zh unknown
- 2021-02-26 JP JP2022551593A patent/JP2023515974A/ja active Pending
- 2021-02-26 US US17/802,324 patent/US20230043648A1/en active Pending
- 2021-02-26 WO PCT/US2021/019960 patent/WO2021174036A1/en unknown
- 2021-03-01 UY UY0001039104A patent/UY39104A/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP4110919A1 (en) | 2023-01-04 |
EP4110919A4 (en) | 2024-06-05 |
US20230043648A1 (en) | 2023-02-09 |
JP2023515974A (ja) | 2023-04-17 |
WO2021174036A1 (en) | 2021-09-02 |
TW202140788A (zh) | 2021-11-01 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
UY39104A (es) | Compuestos y métodos para modular la expresión de scn1a | |
NI200700040A (es) | Analogos de biaril piperazinil-piridina sustituidos | |
NO20080282L (no) | Fremgangsmate for fremstilling av hydrokarboner innen dieselomrade | |
CR20110650A (es) | Mezclas fungicidas | |
AR077042A1 (es) | Metodo para tratar una composicion que contiene pirofiofitina | |
IN2015DN01022A (es) | ||
CO2017012545A2 (es) | Sulfonas tricíclicas como moduladores del receptor huérfano relacionado con retinoide gamma (rorγ) | |
GT201200099A (es) | Derivados que contienen antraquinona como productos agricolas bioquimicos | |
US9682150B1 (en) | Anti-aging nutritional supplement compositions for animals | |
ECSP13013012A (es) | Análogos del ácido epoxieicosatrienoicos y métodos de preparación | |
BR112022020495A2 (pt) | Composições compreendendo algas e métodos de uso das mesmas para aumentar a produção de produto animal | |
CU20090158A7 (es) | Método para la producción de un tabaco altamente mutado productor de semillas | |
PE20141068A1 (es) | Formulaciones topicas de tipo suspension que comprenden depsipeptido ciclico | |
CL2011002160A1 (es) | Compuestos derivados de 3-amino-2-mercaptoquinolinas sustituidas; composicion farmaceutica que contiene dichos compuestos; y su uso para tratar dolor, epilepsis, incontinencia urinaria, manias, migraña, trastornos bipolares, enfermedades cognitivas, discinesia asociada a la distonia y/o incontinencia urinaria. | |
ECSP21088111A (es) | Analogos de 3(5metil1,3tiazol2il)n{(1r)1[2(trifluormetil)pirimidin5il]etil}benzamida | |
CL2017001275A1 (es) | Análogos ligados de urea sustituidos como moduladores de sirtuina | |
CO2021013371A2 (es) | Compuestos y métodos para reducir la expresión de kcnt1 | |
RU2011124942A (ru) | Применение кантаксантина и/или 25-он-d3 для улучшения выводимости домашней птицы | |
WO2012057946A3 (en) | Fuel and base oil blendstocks from a single feedstock | |
UY30921A1 (es) | Tetrahidro-pirazolo-piridinas ligados a carbono como moduladores de catepsina s | |
Gigova et al. | Significance of microalgae-grounds and areas | |
PE20211702A1 (es) | Acidos grasos de cadena muy larga | |
PE20211699A1 (es) | Composiciones insecticidas de diamida | |
CL2023000393A1 (es) | Compuestos y métodos para modular el scn2a | |
AR121445A1 (es) | Compuestos y métodos para modular la expresión de scn1a |