MX2021009178A - Composiciones de oligonucleotidos y metodos de las mismas. - Google Patents
Composiciones de oligonucleotidos y metodos de las mismas.Info
- Publication number
- MX2021009178A MX2021009178A MX2021009178A MX2021009178A MX2021009178A MX 2021009178 A MX2021009178 A MX 2021009178A MX 2021009178 A MX2021009178 A MX 2021009178A MX 2021009178 A MX2021009178 A MX 2021009178A MX 2021009178 A MX2021009178 A MX 2021009178A
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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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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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- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- 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
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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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/08—Solutions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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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.
-
- 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
-
- 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
- C12N2310/346—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
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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
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Entre otras cosas, la presente divulgación proporciona oligonucleótidos, composiciones y métodos para la prevención y/o el tratamiento de diversas afecciones, trastornos o enfermedades. En algunas realizaciones, los oligonucleótidos proporcionados comprenden modificaciones de nucleobase, modificaciones de azúcar, modificaciones de unión internucleotídica y/o patrones de las mismas y tienen propiedades, actividades y/o selectividades mejoradas. En algunas realizaciones, la presente divulgación proporciona oligonucleótidos, composiciones y métodos para afecciones, trastornos o enfermedades relacionados con el HTT, tales como la enfermedad de Huntington.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962800409P | 2019-02-01 | 2019-02-01 | |
US201962911335P | 2019-10-06 | 2019-10-06 | |
PCT/US2020/015971 WO2020160336A1 (en) | 2019-02-01 | 2020-01-30 | Oligonucleotide compositions and methods thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021009178A true MX2021009178A (es) | 2021-09-08 |
Family
ID=71840465
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021009178A MX2021009178A (es) | 2019-02-01 | 2020-01-30 | Composiciones de oligonucleotidos y metodos de las mismas. |
Country Status (14)
Country | Link |
---|---|
US (1) | US20220098585A1 (es) |
EP (1) | EP3917497A4 (es) |
JP (1) | JP2022519019A (es) |
KR (1) | KR20210121199A (es) |
CN (1) | CN113423385A (es) |
AU (1) | AU2020216186A1 (es) |
BR (1) | BR112021014940A2 (es) |
CA (1) | CA3126845A1 (es) |
IL (1) | IL284882A (es) |
MA (1) | MA54875A (es) |
MX (1) | MX2021009178A (es) |
SG (1) | SG11202107318YA (es) |
TW (1) | TW202045189A (es) |
WO (1) | WO2020160336A1 (es) |
Families Citing this family (24)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
RU2015104762A (ru) | 2012-07-13 | 2018-08-31 | Уэйв Лайф Сайенсес Лтд. | Хиральный контроль |
PT2872485T (pt) | 2012-07-13 | 2021-03-05 | Wave Life Sciences Ltd | Grupo auxiliar assimétrico |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
MA43822A (fr) | 2016-03-13 | 2018-11-28 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
EP3463386A4 (en) | 2016-06-03 | 2020-03-04 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDES, COMPOSITIONS AND METHOD THEREFOR |
US10988763B2 (en) | 2016-06-22 | 2021-04-27 | Proqr Therapeutics Ii B.V. | Single-stranded RNA-editing oligonucleotides |
MA46905A (fr) | 2016-11-23 | 2019-10-02 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides |
CN111050806A (zh) | 2017-06-02 | 2020-04-21 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
CN110997692A (zh) | 2017-06-02 | 2020-04-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
US11718638B2 (en) | 2017-06-21 | 2023-08-08 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
WO2019032607A1 (en) | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
AU2018333065B2 (en) | 2017-09-18 | 2024-04-18 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
SG11202001783YA (en) | 2017-10-12 | 2020-03-30 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
CA3090901A1 (en) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
KR20230162024A (ko) | 2021-03-29 | 2023-11-28 | 알닐람 파마슈티칼스 인코포레이티드 | 헌팅틴(HTT) iRNA 제제 조성물 및 이의 사용 방법 |
WO2023076450A2 (en) * | 2021-10-29 | 2023-05-04 | Alnylam Pharmaceuticals, Inc. | HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
WO2024013361A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
WO2024035946A1 (en) * | 2022-08-11 | 2024-02-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
WO2024110565A1 (en) | 2022-11-24 | 2024-05-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis |
GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
Family Cites Families (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JPH09507836A (ja) * | 1993-11-16 | 1997-08-12 | ジンタ・インコーポレイテッド | 非ホスホネート・ヌクレオシジル間結合と混合したキラリティー的に純粋なホスホネート・ヌクレオシジル間結合を有する合成オリゴマー |
US8987222B2 (en) * | 2009-04-08 | 2015-03-24 | University Of Massachusetts | Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease |
WO2011097644A2 (en) * | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
WO2013022966A1 (en) * | 2011-08-11 | 2013-02-14 | Isis Pharmaceuticals, Inc. | Linkage modified gapped oligomeric compounds and uses thereof |
US20150275208A1 (en) * | 2012-10-12 | 2015-10-01 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
MA43072A (fr) * | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
MA45270A (fr) * | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
WO2019032607A1 (en) * | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
-
2020
- 2020-01-30 JP JP2021541152A patent/JP2022519019A/ja active Pending
- 2020-01-30 AU AU2020216186A patent/AU2020216186A1/en active Pending
- 2020-01-30 MX MX2021009178A patent/MX2021009178A/es unknown
- 2020-01-30 BR BR112021014940-6A patent/BR112021014940A2/pt unknown
- 2020-01-30 CN CN202080011722.0A patent/CN113423385A/zh active Pending
- 2020-01-30 WO PCT/US2020/015971 patent/WO2020160336A1/en unknown
- 2020-01-30 KR KR1020217027787A patent/KR20210121199A/ko unknown
- 2020-01-30 US US17/426,511 patent/US20220098585A1/en active Pending
- 2020-01-30 CA CA3126845A patent/CA3126845A1/en active Pending
- 2020-01-30 MA MA054875A patent/MA54875A/fr unknown
- 2020-01-30 EP EP20748395.9A patent/EP3917497A4/en active Pending
- 2020-01-30 SG SG11202107318YA patent/SG11202107318YA/en unknown
- 2020-01-31 TW TW109103034A patent/TW202045189A/zh unknown
-
2021
- 2021-07-15 IL IL284882A patent/IL284882A/en unknown
Also Published As
Publication number | Publication date |
---|---|
CN113423385A (zh) | 2021-09-21 |
CA3126845A1 (en) | 2020-08-06 |
BR112021014940A2 (pt) | 2021-09-28 |
EP3917497A4 (en) | 2023-06-07 |
SG11202107318YA (en) | 2021-08-30 |
US20220098585A1 (en) | 2022-03-31 |
EP3917497A1 (en) | 2021-12-08 |
MA54875A (fr) | 2021-12-08 |
TW202045189A (zh) | 2020-12-16 |
JP2022519019A (ja) | 2022-03-18 |
KR20210121199A (ko) | 2021-10-07 |
AU2020216186A1 (en) | 2021-07-29 |
WO2020160336A1 (en) | 2020-08-06 |
IL284882A (en) | 2021-08-31 |
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