NZ630921A - Compounds and methods for enhanced cellular uptake - Google Patents
Compounds and methods for enhanced cellular uptakeInfo
- Publication number
- NZ630921A NZ630921A NZ630921A NZ63092114A NZ630921A NZ 630921 A NZ630921 A NZ 630921A NZ 630921 A NZ630921 A NZ 630921A NZ 63092114 A NZ63092114 A NZ 63092114A NZ 630921 A NZ630921 A NZ 630921A
- Authority
- NZ
- New Zealand
- Prior art keywords
- modified
- cellular uptake
- compounds
- methods
- enhanced cellular
- Prior art date
Links
Classifications
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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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
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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
- C12N2310/11—Antisense
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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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 nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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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
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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
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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
- C12N2310/323—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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- 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
- C12N2310/351—Conjugate
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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
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/999—Small molecules not provided for elsewhere
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicinal Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361818441P | 2013-05-01 | 2013-05-01 | |
| US201361818447P | 2013-05-01 | 2013-05-01 | |
| US201361822115P | 2013-05-10 | 2013-05-10 | |
| US201361895708P | 2013-10-25 | 2013-10-25 | |
| PCT/US2014/036136 WO2014179445A1 (en) | 2013-05-01 | 2014-04-30 | Compounds and methods for enhanced cellular uptake |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ630921A true NZ630921A (en) | 2017-12-22 |
Family
ID=50943547
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ630921A NZ630921A (en) | 2013-05-01 | 2014-04-30 | Compounds and methods for enhanced cellular uptake |
Country Status (10)
| Country | Link |
|---|---|
| US (3) | US9506030B2 (https=) |
| EP (1) | EP2992096B1 (https=) |
| JP (1) | JP6649248B2 (https=) |
| CN (1) | CN105164261B (https=) |
| AU (1) | AU2014259953B2 (https=) |
| CA (1) | CA2909868C (https=) |
| IL (1) | IL241973B (https=) |
| NZ (1) | NZ630921A (https=) |
| TW (1) | TWI680767B (https=) |
| WO (1) | WO2014179445A1 (https=) |
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| EP3459549B1 (en) | 2012-10-12 | 2022-04-06 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| US9506030B2 (en) | 2013-05-01 | 2016-11-29 | Regulus Therapeutics Inc. | Compounds and methods for enhanced cellular uptake |
| SG10201803157XA (en) | 2013-05-01 | 2018-05-30 | Regulus Therapeutics Inc | Microrna compounds and methods for modulating mir-122 |
| US9994846B2 (en) | 2013-10-25 | 2018-06-12 | Regulus Therapeutics Inc. | MicroRNA compounds and methods for modulating miR-21 activity |
| WO2015179693A1 (en) | 2014-05-22 | 2015-11-26 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| EP3151839A4 (en) * | 2014-06-06 | 2018-02-28 | Ionis Pharmaceuticals, Inc. | Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds |
| KR20170033439A (ko) | 2014-08-07 | 2017-03-24 | 레굴루스 테라퓨틱스 인크 | 대사 장애에 대한 마이크로rna의 표적화 |
| US10577388B2 (en) | 2015-10-02 | 2020-03-03 | Roche Innovation Center Copenhagen A/S | Oligonucleotide conjugation process |
| CN113817735A (zh) | 2015-10-08 | 2021-12-21 | Ionis制药公司 | 用于调节血管紧张素原表达的化合物和方法 |
| US10955407B2 (en) | 2015-10-22 | 2021-03-23 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
| AU2017286811A1 (en) * | 2016-06-17 | 2018-11-22 | Ionis Pharmaceuticals, Inc. | Modulation of gys1 expression |
| JP7012033B2 (ja) | 2016-06-17 | 2022-02-10 | エフ．ホフマン－ラ ロシュ アーゲー | インビトロ腎毒性スクリーニングアッセイ |
| CN109328236B (zh) | 2016-06-17 | 2022-10-25 | 豪夫迈·罗氏有限公司 | 体外肾毒性筛选测定法 |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| CR20200260A (es) * | 2017-12-15 | 2020-08-01 | Bayer Animal Health Gmbh | Composiciones inmunoestimulantes |
| CN111655851A (zh) | 2018-01-18 | 2020-09-11 | 哥本哈根罗氏创新中心 | 靶向srebp1的反义寡核苷酸 |
| CN112105745A (zh) | 2018-05-07 | 2020-12-18 | 罗氏创新中心哥本哈根有限公司 | 用于寡核苷酸治疗剂的大规模平行发现方法 |
| US12005120B2 (en) | 2018-05-08 | 2024-06-11 | Regulus Therapeutics Inc. | Galnac conjugated modified oligonucleotides as miR-122 inhibitor having HCV antiviral activity with reduced hyperbilirubinemia side-effect |
| WO2020007826A1 (en) | 2018-07-05 | 2020-01-09 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides targeting mbtps1 |
| WO2020011744A2 (en) | 2018-07-11 | 2020-01-16 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides targeting cers5 |
| WO2020011745A2 (en) | 2018-07-11 | 2020-01-16 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides targeting cers6 |
| JP2021531278A (ja) * | 2018-07-20 | 2021-11-18 | レグルス セラピューティクス インコーポレイテッド | オリゴヌクレオチドの経口送達のための方法 |
| CN111377985B (zh) * | 2018-12-29 | 2023-11-10 | 苏州瑞博生物技术股份有限公司 | 化合物和缀合物及其制备方法和用途 |
| CN111041025B (zh) | 2019-12-17 | 2021-06-18 | 深圳市瑞吉生物科技有限公司 | 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法 |
| CN111744019B (zh) | 2020-07-01 | 2023-08-04 | 深圳瑞吉生物科技有限公司 | 基于甘露糖的mRNA靶向递送系统及其应用 |
| CN116916968A (zh) * | 2020-10-30 | 2023-10-20 | 托德·斯皮尔 | 基于寡核苷酸的治疗剂及其用途 |
| LT4136092T (lt) | 2020-11-18 | 2024-09-25 | Ionis Pharmaceuticals, Inc. | Junginiai ir būdai, skirti angiotenzinogeno raiškos moduliavimui |
| WO2022186350A1 (ja) * | 2021-03-03 | 2022-09-09 | 国立大学法人東京大学 | 非環式スレオニノール核酸 |
| US12042509B2 (en) | 2021-10-01 | 2024-07-23 | Adarx Pharmaceuticals, Inc. | Prekallikrein-modulating compositions and methods of use thereof |
| AU2023408479A1 (en) | 2022-12-19 | 2025-05-01 | Arnatar Therapeutics, Inc | Advanced rna targeting (arnatar) |
| ES3030929T3 (en) | 2022-12-19 | 2025-07-02 | Arnatar Therapeutics Inc | Arnatar compounds and methods for enhanced cellular uptake |
| TW202448484A (zh) | 2023-04-20 | 2024-12-16 | 美商雅迪克斯製藥公司 | Ｍａｐｔ調節組合物及其使用方法 |
| CN121335980A (zh) | 2023-05-26 | 2026-01-13 | 阿达尔克斯制药有限公司 | Sod1调节组合物及其使用方法 |
| EP4562158A1 (en) | 2023-06-13 | 2025-06-04 | Arnatar Therapeutics, Inc | Advanced rna targeting (arnatar) for angiotensinogen |
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| US9506030B2 (en) | 2016-11-29 |
| CN105164261B (zh) | 2022-03-18 |
| US10941400B2 (en) | 2021-03-09 |
| JP2016518841A (ja) | 2016-06-30 |
| CA2909868A1 (en) | 2014-11-06 |
| EP2992096B1 (en) | 2021-03-03 |
| HK1221257A1 (zh) | 2017-05-26 |
| AU2014259953B2 (en) | 2020-07-02 |
| AU2014259953A1 (en) | 2015-11-05 |
| EP2992096A1 (en) | 2016-03-09 |
| US20190309292A1 (en) | 2019-10-10 |
| IL241973B (en) | 2019-11-28 |
| CN105164261A (zh) | 2015-12-16 |
| US20150031130A1 (en) | 2015-01-29 |
| WO2014179445A1 (en) | 2014-11-06 |
| US10240151B2 (en) | 2019-03-26 |
| TW201505657A (zh) | 2015-02-16 |
| US20170096668A1 (en) | 2017-04-06 |
| TWI680767B (zh) | 2020-01-01 |
| CA2909868C (en) | 2021-10-19 |
| JP6649248B2 (ja) | 2020-02-19 |
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