CR20180107A - Composiciones de oligonucleótidos y métodos de los mismos - Google Patents
Composiciones de oligonucleótidos y métodos de los mismosInfo
- Publication number
- CR20180107A CR20180107A CR20180107A CR20180107A CR20180107A CR 20180107 A CR20180107 A CR 20180107A CR 20180107 A CR20180107 A CR 20180107A CR 20180107 A CR20180107 A CR 20180107A CR 20180107 A CR20180107 A CR 20180107A
- Authority
- CR
- Costa Rica
- Prior art keywords
- chirally controlled
- oligonucleotide composition
- controlled oligonucleotide
- methods
- oligonucleotides
- Prior art date
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- C—CHEMISTRY; METALLURGY
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-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
- C12N2310/34—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
- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (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)
- Neurology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Abstract
Entre otras cosas, la presente divulgación se refiere a oligonucleótidos quiralmente controlados de diseños seleccionados, composiciones oligonucleotídicas quiralmente controladas y métodos de preparación y uso de ellos. En algunas formas de realización, una composición oligonucleotídica quiralmente controlada suministrada proporciona diferentes patrones de escisión de un polímero de ácido nucleico que una composición oligonucleotídica de referencia. En algunas formas de realización, una composición oligonucleotídica quiralmente controlada suministrada proporciona escisión de sitio único dentro de una secuencia complementaria de un polímero de ácido nucleico. En algunas formas de realización, una composición oligonucleotídica quiralmente controlada tiene cualquier secuencia de bases, y/o patrón o modificaciones de base, modificaciones de azúcar, modificaciones estructurales y/o estereoquímica o combinación de estos elementos descritos en la presente
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562195779P | 2015-07-22 | 2015-07-22 | |
| US201562236847P | 2015-10-02 | 2015-10-02 | |
| US201662331960P | 2016-05-04 | 2016-05-04 | |
| PCT/US2016/043542 WO2017015555A1 (en) | 2015-07-22 | 2016-07-22 | Oligonucleotide compositions and methods thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CR20180107A true CR20180107A (es) | 2018-04-03 |
Family
ID=57835219
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CR20180107A CR20180107A (es) | 2015-07-22 | 2016-07-22 | Composiciones de oligonucleótidos y métodos de los mismos |
Country Status (23)
| Country | Link |
|---|---|
| US (6) | US10479995B2 (es) |
| EP (3) | EP4344744A3 (es) |
| JP (6) | JP2018525357A (es) |
| KR (1) | KR20180028516A (es) |
| CN (3) | CN108025089A (es) |
| AU (3) | AU2016297155B2 (es) |
| BR (1) | BR112017028636A2 (es) |
| CA (1) | CA2989682A1 (es) |
| CL (1) | CL2018000145A1 (es) |
| CO (1) | CO2018001484A2 (es) |
| CR (1) | CR20180107A (es) |
| EC (1) | ECSP18010463A (es) |
| HK (2) | HK1255370A1 (es) |
| IL (2) | IL320434A (es) |
| MA (1) | MA43072A (es) |
| MX (2) | MX2018000796A (es) |
| NI (1) | NI201800009A (es) |
| PE (1) | PE20181174A1 (es) |
| PH (1) | PH12018500145A1 (es) |
| SG (1) | SG10201912900XA (es) |
| SV (1) | SV2018005619A (es) |
| TW (1) | TW201707711A (es) |
| WO (2) | WO2017015575A1 (es) |
Families Citing this family (158)
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| CA3244794A1 (en) | 2022-03-02 | 2023-09-07 | Wave Life Sciences Ltd. | OLIGONUCLEOTID COMPOSITIONS AND ASSOCIATED PROCESSES ENABLING EXON SKIPPING |
| AU2023253657A1 (en) | 2022-04-15 | 2024-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods relating thereto |
| CN119452084A (zh) | 2022-05-12 | 2025-02-14 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| KR20250051686A (ko) | 2022-08-11 | 2025-04-17 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 방법 |
| WO2025030155A1 (en) | 2023-08-03 | 2025-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
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2016
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2018
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2019
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2020
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2021
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