AU2013346767B2 - Oligonucleotide conjugates - Google Patents
Oligonucleotide conjugates Download PDFInfo
- Publication number
- AU2013346767B2 AU2013346767B2 AU2013346767A AU2013346767A AU2013346767B2 AU 2013346767 B2 AU2013346767 B2 AU 2013346767B2 AU 2013346767 A AU2013346767 A AU 2013346767A AU 2013346767 A AU2013346767 A AU 2013346767A AU 2013346767 B2 AU2013346767 B2 AU 2013346767B2
- Authority
- AU
- Australia
- Prior art keywords
- region
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- oligomer
- oligomeric compound
- conjugate
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Ceased
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- 0 CC[C@@]([C@@](CN)O*)O Chemical compound CC[C@@]([C@@](CN)O*)O 0.000 description 3
- BPLCXEOSKQVKCX-YSLANXFLSA-O B[C@@H]1O[C@H](C[OH2+])C(C)C1 Chemical compound B[C@@H]1O[C@H](C[OH2+])C(C)C1 BPLCXEOSKQVKCX-YSLANXFLSA-O 0.000 description 1
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- 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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- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P9/00—Drugs for disorders of the cardiovascular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
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- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Obesity (AREA)
- Virology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Vascular Medicine (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2019202288A AU2019202288A1 (en) | 2012-11-15 | 2019-04-03 | Oligonucleotide conjugates |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP12192773 | 2012-11-15 | ||
| EP12192773.5 | 2012-11-15 | ||
| EP13153296 | 2013-01-30 | ||
| EP13153296.2 | 2013-01-30 | ||
| EP13157237 | 2013-02-28 | ||
| EP13157237.2 | 2013-02-28 | ||
| EP13174092.0 | 2013-06-27 | ||
| EP13174092 | 2013-06-27 | ||
| PCT/EP2013/073858 WO2014076195A1 (en) | 2012-11-15 | 2013-11-14 | Oligonucleotide conjugates |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019202288A Division AU2019202288A1 (en) | 2012-11-15 | 2019-04-03 | Oligonucleotide conjugates |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| AU2013346767A1 AU2013346767A1 (en) | 2015-05-28 |
| AU2013346767A2 AU2013346767A2 (en) | 2015-06-18 |
| AU2013346767B2 true AU2013346767B2 (en) | 2019-04-11 |
Family
ID=49584733
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2013346767A Ceased AU2013346767B2 (en) | 2012-11-15 | 2013-11-14 | Oligonucleotide conjugates |
| AU2019202288A Abandoned AU2019202288A1 (en) | 2012-11-15 | 2019-04-03 | Oligonucleotide conjugates |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019202288A Abandoned AU2019202288A1 (en) | 2012-11-15 | 2019-04-03 | Oligonucleotide conjugates |
Country Status (22)
| Country | Link |
|---|---|
| US (4) | US20150291958A1 (enExample) |
| EP (3) | EP2920304B1 (enExample) |
| JP (3) | JP6452614B2 (enExample) |
| KR (2) | KR20150083920A (enExample) |
| CN (3) | CN104837996A (enExample) |
| AU (2) | AU2013346767B2 (enExample) |
| BR (2) | BR112015011112A2 (enExample) |
| CA (2) | CA2889044A1 (enExample) |
| DK (2) | DK2920307T3 (enExample) |
| ES (1) | ES2724853T3 (enExample) |
| HK (2) | HK1210213A1 (enExample) |
| HR (1) | HRP20190826T1 (enExample) |
| IL (2) | IL238448B (enExample) |
| MX (2) | MX2015005792A (enExample) |
| MY (1) | MY173826A (enExample) |
| NZ (1) | NZ708171A (enExample) |
| PL (1) | PL2920304T3 (enExample) |
| RU (3) | RU2653438C2 (enExample) |
| SG (2) | SG11201503821YA (enExample) |
| SI (1) | SI2920304T1 (enExample) |
| WO (2) | WO2014076196A1 (enExample) |
| ZA (1) | ZA201503375B (enExample) |
Families Citing this family (308)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2752488B1 (en) | 2002-11-18 | 2020-02-12 | Roche Innovation Center Copenhagen A/S | Antisense design |
| HRP20171969T1 (hr) | 2008-10-15 | 2018-04-06 | Ionis Pharmaceuticals, Inc. | Modulacija ekspresije faktora 11 |
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| WO2012068405A2 (en) | 2010-11-17 | 2012-05-24 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| US9580708B2 (en) | 2011-09-14 | 2017-02-28 | Rana Therapeutics, Inc. | Multimeric oligonucleotides compounds |
| SG11201407483YA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| BR112014028634A2 (pt) | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| CA2873769A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating hemoglobin gene family expression |
| BR112014028644A2 (pt) | 2012-05-16 | 2017-08-15 | Rana Therapeutics Inc | Composições e métodos para modulação da expressão de atp2a2 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
| KR20240148947A (ko) | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| EP2895200B1 (en) | 2012-09-14 | 2019-11-06 | Translate Bio MA, Inc. | Multimeric oligonucleotide compounds |
| JP6452614B2 (ja) * | 2012-11-15 | 2019-01-16 | ロシュ イノベーション センター コペンハーゲン エーエス | オリゴヌクレオチドコンジュゲート |
| DK2951305T3 (en) * | 2013-01-30 | 2018-10-29 | Hoffmann La Roche | LNA oligonucleotide KULHYDRATKONJUGATER |
| PE20152002A1 (es) | 2013-05-01 | 2016-01-21 | Isis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion de ttr y vhb |
| JP6649248B2 (ja) | 2013-05-01 | 2020-02-19 | レグルス セラピューティクス インコーポレイテッド | 細胞取り込みの向上のための化合物および方法 |
| SG11201508925WA (en) | 2013-05-01 | 2015-11-27 | Regulus Therapeutics Inc | Microrna compounds and methods for modulating mir-122 |
| WO2015042447A1 (en) | 2013-09-20 | 2015-03-26 | Isis Pharmaceuticals, Inc. | Targeted therapeutic nucleosides and their use |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| US9994846B2 (en) | 2013-10-25 | 2018-06-12 | Regulus Therapeutics Inc. | MicroRNA compounds and methods for modulating miR-21 activity |
| 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 |
| 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 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| KR102287532B1 (ko) | 2014-01-30 | 2021-08-11 | 에프. 호프만-라 로슈 아게 | 생분해성 컨쥬게이트를 갖는 폴리 올리고머 화합물 |
| EP3119888B1 (en) | 2014-03-19 | 2021-07-28 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| SI3757214T1 (sl) | 2014-04-01 | 2022-08-31 | Biogen Ma Inc. | Sestave za moduliranje izražanja SOD-1 |
| RS59182B1 (sr) | 2014-05-01 | 2019-10-31 | Ionis Pharmaceuticals Inc | Kompozicije i postupci za modulaciju ekspresije faktora b komplementa |
| ES2844593T3 (es) | 2014-05-01 | 2021-07-22 | Ionis Pharmaceuticals Inc | Composiciones y procedimientos para modular la expresión de la angiopoyetina de tipo 3 |
| ES2812099T3 (es) | 2014-05-01 | 2021-03-16 | Ionis Pharmaceuticals Inc | Composiciones y métodos para modular la expresión del receptor de la hormona del crecimiento |
| HUE052709T2 (hu) | 2014-05-01 | 2021-05-28 | Ionis Pharmaceuticals Inc | Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására |
| GB201408623D0 (en) | 2014-05-15 | 2014-07-02 | Santaris Pharma As | Oligomers and oligomer conjugates |
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