SV2018005619A - Composiciones de oligonucleotidos y metodos de los mismos - Google Patents
Composiciones de oligonucleotidos y metodos de los mismosInfo
- Publication number
- SV2018005619A SV2018005619A SV2018005619A SV2018005619A SV2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A SV 2018005619 A SV2018005619 A SV 2018005619A
- Authority
- SV
- El Salvador
- Prior art keywords
- quirally
- controlled
- oligonucleotydic
- forms
- composition
- Prior art date
Links
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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
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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
- 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
- C12N2310/322—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
Landscapes
- 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)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (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 (3)
| 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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SV2018005619A true SV2018005619A (es) | 2018-07-20 |
Family
ID=57835219
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SV2018005619A SV2018005619A (es) | 2015-07-22 | 2018-01-22 | Composiciones de oligonucleotidos y metodos de los mismos |
Country Status (23)
| Country | Link |
|---|---|
| US (6) | US20180216107A1 (es) |
| EP (3) | EP4344744A3 (es) |
| JP (6) | JP2018525357A (es) |
| KR (1) | KR20180028516A (es) |
| CN (3) | CN117965536A (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 (155)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8158768B2 (en) * | 2002-12-23 | 2012-04-17 | Dynavax Technologies Corporation | Immunostimulatory sequence oligonucleotides and methods of using the same |
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| BR112012000828A8 (pt) | 2009-07-06 | 2017-10-10 | Ontorii Inc | Novas pró-drogas de ácido nucleico e métodos de uso das mesmas |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| JP6492003B2 (ja) | 2012-03-30 | 2019-03-27 | ワシントン・ユニバーシティＷａｓｈｉｎｇｔｏｎ Ｕｎｉｖｅｒｓｉｔｙ | 発作を減少させるためおよび神経変性症候群を改質するためにタウ発現を調節する方法 |
| BR112015000784A8 (pt) | 2012-07-13 | 2018-04-03 | Wave Life Sciences Japan | Grupo auxiliar assimétrico |
| RU2015104762A (ru) | 2012-07-13 | 2018-08-31 | Уэйв Лайф Сайенсес Лтд. | Хиральный контроль |
| TWI702046B (zh) | 2013-07-19 | 2020-08-21 | 美商Ionis製藥公司 | 用於調節τ蛋白表現之組合物 |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| WO2015107425A2 (en) | 2014-01-16 | 2015-07-23 | Wave Life Sciences Pte. Ltd. | Chiral design |
| WO2016096938A1 (en) * | 2014-12-16 | 2016-06-23 | Roche Innovation Center Copenhagen A/S | Chiral toxicity screening method |
| DK3234134T3 (da) | 2014-12-17 | 2020-07-27 | Proqr Therapeutics Ii Bv | Målrettet rna-redigering |
| US11129844B2 (en) | 2015-03-03 | 2021-09-28 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating MECP2 expression |
| US9809817B2 (en) | 2015-04-03 | 2017-11-07 | University Of Massachusetts | Oligonucleotide compounds for targeting huntingtin mRNA |
| EP3929293A3 (en) | 2015-04-03 | 2022-03-16 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EP3334499A4 (en) | 2015-08-14 | 2019-04-17 | University of Massachusetts | BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES |
| MA46427A (fr) | 2015-10-09 | 2018-08-15 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| HUE055031T2 (hu) | 2015-12-10 | 2021-10-28 | Ptc Therapeutics Inc | Módszerek Huntington-kór kezelésére |
| CA3011894A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| CA3015823A1 (en) | 2016-03-13 | 2017-09-21 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| JP2019520339A (ja) | 2016-06-03 | 2019-07-18 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド、その組成物および方法 |
| KR102418185B1 (ko) | 2016-06-22 | 2022-07-06 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 단일 가닥 rna-편집 올리고뉴클레오타이드 |
| US11753638B2 (en) | 2016-08-12 | 2023-09-12 | University Of Massachusetts | Conjugated oligonucleotides |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
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| WO2018145009A1 (en) * | 2017-02-06 | 2018-08-09 | University Of Tennessee Research Foundation | Dna-zyme based methods & compositions for treating huntington's disease |
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| WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
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| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| JP2021525507A (ja) * | 2018-05-25 | 2021-09-27 | ニューヨーク インスティチュート オブ テクノロジーＮｅｗ Ｙｏｒｋ Ｉｎｓｔｉｔｕｔｅ Ｏｆ Ｔｅｃｈｎｏｌｏｇｙ | 直接核酸配列決定方法 |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP4219717A3 (en) | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
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