JP2013511990A5 - - Google Patents
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- JP2013511990A5 JP2013511990A5 JP2012541211A JP2012541211A JP2013511990A5 JP 2013511990 A5 JP2013511990 A5 JP 2013511990A5 JP 2012541211 A JP2012541211 A JP 2012541211A JP 2012541211 A JP2012541211 A JP 2012541211A JP 2013511990 A5 JP2013511990 A5 JP 2013511990A5
- Authority
- JP
- Japan
- Prior art keywords
- double
- nucleic acid
- stranded nucleic
- acid molecule
- modified
- 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.)
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- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 22
- 108020004707 nucleic acids Proteins 0.000 claims 19
- 102000039446 nucleic acids Human genes 0.000 claims 19
- 150000007523 nucleic acids Chemical class 0.000 claims 19
- 239000002773 nucleotide Substances 0.000 claims 18
- 125000003729 nucleotide group Chemical group 0.000 claims 18
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 13
- 230000000692 anti-sense effect Effects 0.000 claims 10
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 8
- 108091081021 Sense strand Proteins 0.000 claims 8
- 108020004999 messenger RNA Proteins 0.000 claims 6
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 5
- 206010030924 Optic ischaemic neuropathy Diseases 0.000 claims 5
- 229960005305 adenosine Drugs 0.000 claims 5
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims 5
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 5
- 229940045145 uridine Drugs 0.000 claims 5
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims 4
- 208000010038 Ischemic Optic Neuropathy Diseases 0.000 claims 4
- 108020004459 Small interfering RNA Proteins 0.000 claims 4
- 150000003838 adenosines Chemical class 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 210000004072 lung Anatomy 0.000 claims 4
- 230000002194 synthesizing effect Effects 0.000 claims 4
- 238000002054 transplantation Methods 0.000 claims 4
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 claims 3
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims 3
- 201000007058 anterior ischemic optic neuropathy Diseases 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 230000004064 dysfunction Effects 0.000 claims 3
- 210000000056 organ Anatomy 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- DWRXFEITVBNRMK-JXOAFFINSA-N ribothymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 DWRXFEITVBNRMK-JXOAFFINSA-N 0.000 claims 3
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 2
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 claims 2
- MXHRCPNRJAMMIM-SHYZEUOFSA-N 2'-deoxyuridine Chemical compound C1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 MXHRCPNRJAMMIM-SHYZEUOFSA-N 0.000 claims 2
- 208000009304 Acute Kidney Injury Diseases 0.000 claims 2
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 claims 2
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 claims 2
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 claims 2
- 108091034117 Oligonucleotide Proteins 0.000 claims 2
- 108091030071 RNAI Proteins 0.000 claims 2
- 208000033626 Renal failure acute Diseases 0.000 claims 2
- 206010063837 Reperfusion injury Diseases 0.000 claims 2
- 201000011040 acute kidney failure Diseases 0.000 claims 2
- 208000012998 acute renal failure Diseases 0.000 claims 2
- 206010064930 age-related macular degeneration Diseases 0.000 claims 2
- 238000000137 annealing Methods 0.000 claims 2
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 claims 2
- MXHRCPNRJAMMIM-UHFFFAOYSA-N desoxyuridine Natural products C1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 MXHRCPNRJAMMIM-UHFFFAOYSA-N 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 230000009368 gene silencing by RNA Effects 0.000 claims 2
- 229940029575 guanosine Drugs 0.000 claims 2
- 210000003734 kidney Anatomy 0.000 claims 2
- 208000002780 macular degeneration Diseases 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 125000002652 ribonucleotide group Chemical group 0.000 claims 2
- 206010001052 Acute respiratory distress syndrome Diseases 0.000 claims 1
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 claims 1
- 206010011878 Deafness Diseases 0.000 claims 1
- 206010011985 Decubitus ulcer Diseases 0.000 claims 1
- 208000003556 Dry Eye Syndromes Diseases 0.000 claims 1
- 206010013774 Dry eye Diseases 0.000 claims 1
- 244000089409 Erythrina poeppigiana Species 0.000 claims 1
- 208000010412 Glaucoma Diseases 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 206010029155 Nephropathy toxic Diseases 0.000 claims 1
- 206010029350 Neurotoxicity Diseases 0.000 claims 1
- 206010030113 Oedema Diseases 0.000 claims 1
- 208000004210 Pressure Ulcer Diseases 0.000 claims 1
- 235000009776 Rathbunia alamosensis Nutrition 0.000 claims 1
- 206010044221 Toxic encephalopathy Diseases 0.000 claims 1
- 206010060872 Transplant failure Diseases 0.000 claims 1
- 208000027418 Wounds and injury Diseases 0.000 claims 1
- 230000001154 acute effect Effects 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 208000029028 brain injury Diseases 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 230000006378 damage Effects 0.000 claims 1
- 239000005547 deoxyribonucleotide Substances 0.000 claims 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims 1
- -1 deoxyribouridine Chemical compound 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000003176 fibrotic effect Effects 0.000 claims 1
- 230000010370 hearing loss Effects 0.000 claims 1
- 231100000888 hearing loss Toxicity 0.000 claims 1
- 208000016354 hearing loss disease Diseases 0.000 claims 1
- 210000002216 heart Anatomy 0.000 claims 1
- 208000014674 injury Diseases 0.000 claims 1
- 208000028867 ischemia Diseases 0.000 claims 1
- 208000012947 ischemia reperfusion injury Diseases 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 108091070501 miRNA Proteins 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 230000007694 nephrotoxicity Effects 0.000 claims 1
- 231100000417 nephrotoxicity Toxicity 0.000 claims 1
- 230000004770 neurodegeneration Effects 0.000 claims 1
- 208000015122 neurodegenerative disease Diseases 0.000 claims 1
- 230000007135 neurotoxicity Effects 0.000 claims 1
- 231100000228 neurotoxicity Toxicity 0.000 claims 1
- 201000002761 non-arteritic anterior ischemic optic neuropathy Diseases 0.000 claims 1
- 210000000496 pancreas Anatomy 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 230000010410 reperfusion Effects 0.000 claims 1
- 230000000241 respiratory effect Effects 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 101150054147 sina gene Proteins 0.000 claims 1
- 208000020431 spinal cord injury Diseases 0.000 claims 1
- 208000003265 stomatitis Diseases 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26466809P | 2009-11-26 | 2009-11-26 | |
| US61/264,668 | 2009-11-26 | ||
| US29572110P | 2010-01-17 | 2010-01-17 | |
| US61/295,721 | 2010-01-17 | ||
| US37207210P | 2010-08-09 | 2010-08-09 | |
| US61/372,072 | 2010-08-09 | ||
| PCT/US2010/058123 WO2011066475A1 (en) | 2009-11-26 | 2010-11-25 | Sirna compounds comprising terminal substitutions |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2013511990A JP2013511990A (ja) | 2013-04-11 |
| JP2013511990A5 true JP2013511990A5 (enExample) | 2014-01-09 |
Family
ID=43589717
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012541211A Pending JP2013511990A (ja) | 2009-11-26 | 2010-11-25 | 末端置換を含むｓｉＲＮＡ化合物 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US8796239B2 (enExample) |
| EP (1) | EP2504435B1 (enExample) |
| JP (1) | JP2013511990A (enExample) |
| KR (1) | KR20120102630A (enExample) |
| CN (1) | CN102597239A (enExample) |
| AU (1) | AU2010324658A1 (enExample) |
| CA (1) | CA2776568A1 (enExample) |
| DK (1) | DK2504435T3 (enExample) |
| ES (1) | ES2759003T3 (enExample) |
| IL (1) | IL218965A0 (enExample) |
| NZ (1) | NZ599237A (enExample) |
| TW (1) | TW201124160A (enExample) |
| WO (1) | WO2011066475A1 (enExample) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN104861066B (zh) | 2009-03-23 | 2018-05-08 | 夸克制药公司 | 治疗癌症和纤维化疾病的化合物组合物和方法 |
| AU2010324658A1 (en) * | 2009-11-26 | 2012-05-03 | Quark Pharmaceuticals, Inc. | siRNA compounds comprising terminal substitutions |
| ES2562499T3 (es) * | 2009-12-09 | 2016-03-04 | Nitto Denko Corporation | Modulación de la expresión de HSP47 |
| WO2011084193A1 (en) | 2010-01-07 | 2011-07-14 | Quark Pharmaceuticals, Inc. | Oligonucleotide compounds comprising non-nucleotide overhangs |
| EP2649181B1 (en) | 2010-12-06 | 2016-04-27 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising positional modifications |
| AU2012223366B2 (en) | 2011-03-03 | 2017-02-23 | Quark Pharmaceuticals, Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| US9796979B2 (en) | 2011-03-03 | 2017-10-24 | Quark Pharmaceuticals Inc. | Oligonucleotide modulators of the toll-like receptor pathway |
| SG192961A1 (en) | 2011-03-03 | 2013-09-30 | Quark Pharmaceuticals Inc | Compositions and methods for treating lung disease and injury |
| AU2012332517B9 (en) | 2011-11-03 | 2017-08-10 | Quark Pharmaceuticals, Inc. | Methods and compositions for neuroprotection |
| US20140323549A1 (en) | 2011-11-08 | 2014-10-30 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the nervous system |
| CA2895077A1 (en) * | 2011-12-12 | 2013-06-20 | Beverly Packard | In vivo delivery of oligonucleotides |
| JP2015509085A (ja) | 2012-01-01 | 2015-03-26 | キュービーアイ エンタープライゼズ リミテッドＱｂｉ Ｅｎｔｅｒｐｒｉｓｅｓ Ｌｔｄ． | 治療剤および診断剤の選択的送達のためのｅｎｄｏ１８０を標的とする粒子 |
| ES2704855T3 (es) | 2012-09-12 | 2019-03-20 | Quark Pharmaceuticals Inc | Moléculas de oligonucleótido de doble cadena para p53 y métodos de uso de las mismas |
| CA2880290C (en) | 2012-09-12 | 2020-10-27 | Quark Pharmaceuticals, Inc. | Double-stranded oligonucleotide molecules targeting p53 and methods of use thereof |
| US9611473B2 (en) | 2012-09-12 | 2017-04-04 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| US20160208247A1 (en) | 2013-07-31 | 2016-07-21 | Qbi Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| EP3027222A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Sphingolipid-polyalkylamine-oligonucleotide compounds |
| KR101696704B1 (ko) * | 2013-12-17 | 2017-01-16 | 주식회사 인코드젠 | 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도 |
| KR101627404B1 (ko) * | 2014-04-08 | 2016-06-03 | 성균관대학교산학협력단 | Dab2 유전자가 넉 다운된 수지상 세포를 포함하는 암 예방 또는 치료용 약학적 조성물 |
| SI3444349T1 (sl) | 2014-05-29 | 2021-07-30 | Quark Pharmaceuticals, Inc. | Postopki in sestavki za preprečevanje ishemične reperfuzijske poškodbe v organih |
| US20160089391A1 (en) * | 2014-09-30 | 2016-03-31 | Temple University-Of The Commonwealth System Of Higher Education | Molecular Targets for Cardiovascular Disease |
| MA45469A (fr) * | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques de bêta-caténine et leurs utilisations |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| KR102737464B1 (ko) * | 2017-09-14 | 2024-12-02 | 애로우헤드 파마슈티컬스 인코포레이티드 | 안지오포이에틴-유사 3 (ANGPTL3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물, 및 사용 방법 |
| WO2019165267A1 (en) | 2018-02-22 | 2019-08-29 | The Regents Of The University Of California | Methods for treating brain injury |
| CN112331258B (zh) * | 2020-11-06 | 2024-02-23 | 大连大学 | 基于dna笼状结构的人工神经元计算模型构建方法 |
| KR102404883B1 (ko) * | 2020-11-30 | 2022-06-07 | (주)이노보테라퓨틱스 | 벤즈브로마론을 포함하는 켈로이드 또는 비대흉터 예방 또는 치료용 약학 조성물 |
| JP2024528697A (ja) | 2021-07-20 | 2024-07-30 | エイジーエス・セラピューティクス・ソシエテ・パール・アクシオン・サンプリフィエ | 微細藻類由来の細胞外小胞、その調製および使用 |
| WO2023144127A1 (en) | 2022-01-31 | 2023-08-03 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their biodistribution upon administration, and uses |
| WO2023232976A1 (en) | 2022-06-03 | 2023-12-07 | Ags Therapeutics Sas | Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses |
| WO2024088808A1 (en) | 2022-10-24 | 2024-05-02 | Ags Therapeutics Sas | Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof |
| WO2025176843A1 (en) | 2024-02-21 | 2025-08-28 | Ags Therapeutics Sas | Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof |
| CN120574835B (zh) * | 2025-07-31 | 2025-10-03 | 羿美诚健(上海)生物医药有限公司 | 一种靶向rankl基因的小核酸及其在治疗骨科疾病中的应用 |
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-
2010
- 2010-11-25 AU AU2010324658A patent/AU2010324658A1/en not_active Abandoned
- 2010-11-25 US US13/511,111 patent/US8796239B2/en active Active
- 2010-11-25 EP EP10785571.0A patent/EP2504435B1/en not_active Not-in-force
- 2010-11-25 NZ NZ599237A patent/NZ599237A/en unknown
- 2010-11-25 JP JP2012541211A patent/JP2013511990A/ja active Pending
- 2010-11-25 ES ES10785571T patent/ES2759003T3/es active Active
- 2010-11-25 KR KR1020127012259A patent/KR20120102630A/ko not_active Withdrawn
- 2010-11-25 CA CA2776568A patent/CA2776568A1/en not_active Abandoned
- 2010-11-25 CN CN201080049901XA patent/CN102597239A/zh active Pending
- 2010-11-25 WO PCT/US2010/058123 patent/WO2011066475A1/en not_active Ceased
- 2010-11-25 TW TW099141067A patent/TW201124160A/zh unknown
- 2010-11-25 DK DK10785571T patent/DK2504435T3/da active
-
2012
- 2012-04-01 IL IL218965A patent/IL218965A0/en unknown
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2014
- 2014-08-04 US US14/451,233 patent/US9701960B2/en not_active Expired - Fee Related
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2017
- 2017-07-06 US US15/642,581 patent/US10494631B2/en not_active Expired - Fee Related
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