DK3234132T3 - Ligand-modificerede dobbeltstrengede nukleinsyrer - Google Patents
Ligand-modificerede dobbeltstrengede nukleinsyrer Download PDFInfo
- Publication number
- DK3234132T3 DK3234132T3 DK15817743.6T DK15817743T DK3234132T3 DK 3234132 T3 DK3234132 T3 DK 3234132T3 DK 15817743 T DK15817743 T DK 15817743T DK 3234132 T3 DK3234132 T3 DK 3234132T3
- Authority
- DK
- Denmark
- Prior art keywords
- structed
- ligand
- nucleic acids
- modified double
- double
- Prior art date
Links
- 0 CC(*Cc1ccccc1)=O Chemical compound CC(*Cc1ccccc1)=O 0.000 description 3
Classifications
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- 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
- C07H21/04—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 deoxyribosyl as saccharide radical
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H1/00—Processes for the preparation of sugar derivatives
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H99/00—Subject matter not provided for in other groups of this subclass
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
-
- 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.
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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
- C12N2310/351—Conjugate
-
- 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
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
-
- 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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462092241P | 2014-12-15 | 2014-12-15 | |
| US201462092238P | 2014-12-15 | 2014-12-15 | |
| US201562187848P | 2015-07-02 | 2015-07-02 | |
| US201562187856P | 2015-07-02 | 2015-07-02 | |
| PCT/US2015/065906 WO2016100401A1 (en) | 2014-12-15 | 2015-12-15 | Ligand-modified double-stranded nucleic acids |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3234132T3 true DK3234132T3 (da) | 2019-08-26 |
Family
ID=56127491
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK19180844.3T DK3569711T3 (da) | 2014-12-15 | 2015-12-15 | Ligandmodificerede dobbeltstrengede nukleinsyrer |
| DK15817743.6T DK3234132T3 (da) | 2014-12-15 | 2015-12-15 | Ligand-modificerede dobbeltstrengede nukleinsyrer |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK19180844.3T DK3569711T3 (da) | 2014-12-15 | 2015-12-15 | Ligandmodificerede dobbeltstrengede nukleinsyrer |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US20170305956A1 (da) |
| EP (3) | EP3569711B1 (da) |
| JP (1) | JP7105065B2 (da) |
| AU (2) | AU2015362630B2 (da) |
| CA (1) | CA2970801C (da) |
| DK (2) | DK3569711T3 (da) |
| ES (2) | ES2858403T3 (da) |
| WO (1) | WO2016100401A1 (da) |
Families Citing this family (86)
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|---|---|---|---|---|
| CN107257858B (zh) | 2014-10-10 | 2022-03-08 | 戴瑟纳制药公司 | 乳酸脱氢酶的治疗性抑制及其试剂 |
| JP6978099B2 (ja) | 2016-09-02 | 2021-12-08 | ディセルナ ファーマシューティカルズ インコーポレイテッド | ４’−リン酸アナログ及びそれを含むオリゴヌクレオチド |
| CA3037192A1 (en) * | 2016-11-07 | 2018-05-11 | nanoSUR LLC | Post-transcriptionally chemically modified double strand rnas |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| KR20200023427A (ko) * | 2017-06-29 | 2020-03-04 | 다이서나 파마수이티컬, 인크. | Hmgb1 발현을 억제하기 위한 조성물 및 방법 |
| CA3078933A1 (en) * | 2017-10-13 | 2019-04-18 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for inhibiting expression of ldha |
| KR20200078538A (ko) * | 2017-10-18 | 2020-07-01 | 다이서나 파마수이티컬, 인크. | 베타 카테닌 핵산 억제제 분자 |
| PT3684377T (pt) | 2017-10-20 | 2023-01-31 | Dicerna Pharmaceuticals Inc | Métodos de tratamento de infeção de hepatite b |
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| US11661603B2 (en) | 2018-01-16 | 2023-05-30 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting ALDH2 expression |
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| US11873488B2 (en) | 2018-04-13 | 2024-01-16 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules modified with Tm-increasing nucleotides |
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| WO2019222479A1 (en) | 2018-05-16 | 2019-11-21 | Alnylam Pharmaceuticals, Inc. | Modified rna agents with reduced off-target effect |
| CN113227371A (zh) * | 2018-08-27 | 2021-08-06 | 圣诺制药公司 | 微型发夹RNAi触发物(mxRNA)和其使用方法 |
| IL281135B2 (en) | 2018-09-28 | 2024-12-01 | Nutcracker Therapeutics Inc | Tertiary amino lipidated cationic peptides for nucleic acid delivery |
| SG11202106046XA (en) * | 2018-12-12 | 2021-07-29 | Dicerna Pharmaceuticals Inc | Double-stranded nucleic acid inhibitor molecules containing a triloop |
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| EP3963072A1 (en) | 2019-05-03 | 2022-03-09 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules with shortened sense strands |
| BR112022003861A2 (pt) | 2019-08-30 | 2022-05-24 | Dicerna Pharmaceuticals Inc | Ácidos nucleicos ligando-2'-modificados, síntese dos mesmos, e intermediários para os mesmos |
| US20220340903A1 (en) * | 2019-09-25 | 2022-10-27 | University Of Massachusetts | Targeting rlim to modulate body weight and obesity |
| WO2021067744A1 (en) | 2019-10-02 | 2021-04-08 | Dicerna Pharmaceuticals, Inc. | Chemical modifications of small interfering rna with minimal fluorine content |
| KR20220088437A (ko) * | 2019-10-25 | 2022-06-27 | 피토넥스, 인코포레이티드 | 폴리뉴클레오티드-연결된 바이오컨쥬게이트 및 제조 및 이용 방법 |
| EP4081639A1 (en) | 2019-12-24 | 2022-11-02 | F. Hoffmann-La Roche AG | Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv |
| JP2023509872A (ja) | 2019-12-24 | 2023-03-10 | エフ． ホフマン－ラ ロシュ アーゲー | Ｈｂｖを標的とする抗ウイルス剤及び／又はｈｂｖの処置のための免疫調節剤の医薬組合せ |
| US20230123981A1 (en) | 2020-01-15 | 2023-04-20 | Dicerna Pharmaceuticals, Inc. | 4'-o-methylene phosphonate nucleic acids and analogues thereof |
| JPWO2021153687A1 (da) | 2020-01-30 | 2021-08-05 | ||
| EP4121536A1 (en) | 2020-03-18 | 2023-01-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting angptl3 expression |
| TW202221120A (zh) | 2020-08-04 | 2022-06-01 | 美商黛瑟納製藥公司 | 用於治療代謝症候群之組成物及方法 |
| EP4192478A2 (en) | 2020-08-04 | 2023-06-14 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting plp1 expression |
| AU2021321289A1 (en) | 2020-08-04 | 2023-03-02 | Dicerna Pharmaceuticals, Inc. | Systemic delivery of oligonucleotides |
| BR112023001957A2 (pt) | 2020-08-05 | 2023-04-25 | Dicerna Pharmaceuticals Inc | Composições e métodos para inibir expressão de lpa |
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| AR124713A1 (es) * | 2021-01-28 | 2023-04-26 | Dicerna Pharmaceuticals Inc | Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central |
| WO2022191485A1 (ko) | 2021-03-10 | 2022-09-15 | 에스에프씨 주식회사 | 리포터 및 이의 용도 |
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| MX2023013504A (es) | 2021-05-28 | 2024-02-23 | Novo Nordisk As | Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1). |
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| CN119173631A (zh) | 2022-05-12 | 2024-12-20 | 迪克纳制药公司 | 用于抑制mapt表达的组合物和方法 |
| CN119630791A (zh) | 2022-05-13 | 2025-03-14 | 迪克纳制药公司 | 用于抑制snca表达的组合物和方法 |
| AU2023288906A1 (en) * | 2022-06-24 | 2024-12-12 | Dicerna Pharmaceuticals Inc. | Compositions and methods for inhibiting transmembrane serine protease 6 (tmprss6) expression |
| KR20250034973A (ko) | 2022-07-11 | 2025-03-11 | 사네진 바이오 유에스에이 인크. | 최적화된 2'-변형된 리보스 유도체 및 사용 방법 |
| US20240052348A1 (en) | 2022-08-05 | 2024-02-15 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiotensinogen (agt) and methods of use thereof |
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| EP3569711B1 (en) | 2021-02-03 |
| EP3569711A1 (en) | 2019-11-20 |
| EP3234132B1 (en) | 2019-07-10 |
| JP7105065B2 (ja) | 2022-07-22 |
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