CN118530989A - 用于抑制ldha表达的方法和组合物 - Google Patents
用于抑制ldha表达的方法和组合物 Download PDFInfo
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- CN118530989A CN118530989A CN202410538409.2A CN202410538409A CN118530989A CN 118530989 A CN118530989 A CN 118530989A CN 202410538409 A CN202410538409 A CN 202410538409A CN 118530989 A CN118530989 A CN 118530989A
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- oligonucleotide
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- antisense strand
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- ldha
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- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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Applications Claiming Priority (8)
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| US201762572398P | 2017-10-13 | 2017-10-13 | |
| US201762572403P | 2017-10-13 | 2017-10-13 | |
| US62/572398 | 2017-10-13 | ||
| US62/572403 | 2017-10-13 | ||
| US201862726950P | 2018-09-04 | 2018-09-04 | |
| US62/726950 | 2018-09-04 | ||
| CN201880080394.2A CN111448319B (zh) | 2017-10-13 | 2018-10-12 | 用于抑制ldha表达的方法和组合物 |
| PCT/US2018/055735 WO2019075419A1 (en) | 2017-10-13 | 2018-10-12 | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
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| CN201880080394.2A Active CN111448319B (zh) | 2017-10-13 | 2018-10-12 | 用于抑制ldha表达的方法和组合物 |
| CN202410538408.8A Pending CN118440940A (zh) | 2017-10-13 | 2018-10-12 | 用于抑制ldha表达的方法和组合物 |
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| CN202410538408.8A Pending CN118440940A (zh) | 2017-10-13 | 2018-10-12 | 用于抑制ldha表达的方法和组合物 |
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| RS (1) | RS64483B1 (https=) |
| WO (1) | WO2019075419A1 (https=) |
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| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| WO2019075419A1 (en) * | 2017-10-13 | 2019-04-18 | Dicerna Pharmaceuticals, Inc. | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
| UA128786C2 (uk) * | 2017-10-20 | 2024-10-23 | Дайсерна Фармасьютикалз, Інк | Олігонуклеотид для лікування інфекції гепатиту в |
| KR20200109311A (ko) | 2018-01-16 | 2020-09-22 | 다이서나 파마수이티컬, 인크. | Aldh2 발현을 억제하기 위한 조성물 및 방법 |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| WO2021041247A1 (en) * | 2019-08-23 | 2021-03-04 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| AU2020358016A1 (en) * | 2019-10-02 | 2022-04-21 | Dicerna Pharmaceuticals, Inc. | Chemical modifications of small interfering RNA with minimal fluorine content |
| EP4121534A1 (en) | 2020-03-18 | 2023-01-25 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant |
| EP4153586A4 (en) * | 2020-05-18 | 2024-07-10 | Chinook Therapeutics Canada, Inc. | SUBSTITUTED PYRAZOLYL COMPOUNDS AND METHODS OF USE |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| EP4244356A1 (en) * | 2020-11-13 | 2023-09-20 | Dicerna Pharmaceuticals, Inc. | Chemical modifications for inhibiting expression of aldh2 |
| EP4161942B1 (en) | 2021-03-19 | 2023-08-02 | Bachem Holding AG | Improved oligonucleotide synthesis suppressing depurination |
| US20250282813A1 (en) | 2021-04-09 | 2025-09-11 | Bachem Holding Ag | Pseudo solid phase protecting group and methods for the synthesis of oligonucleotides and oligonucleotide conjugates |
| WO2022223515A2 (en) * | 2021-04-19 | 2022-10-27 | Novo Nordisk A/S | Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression |
| TW202308662A (zh) * | 2021-05-11 | 2023-03-01 | 美商黛瑟納製藥公司 | 用於靶定中樞神經系統的神經元之脂質結合物 |
| US11649260B2 (en) | 2021-06-18 | 2023-05-16 | Hongene Biotech Corporation | Functionalized N-acetylgalactosamine nucleosides |
| WO2023003805A1 (en) | 2021-07-19 | 2023-01-26 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder |
| EP4392556A1 (en) * | 2021-08-25 | 2024-07-03 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting ?lpha-1 antitrypsin expression |
| EP4396193A4 (en) | 2021-08-30 | 2025-07-02 | Hongene Biotech Corp | FUNCTIONALIZED N-ACETYLGALACTOSAMINE ANALOGUES |
| AR127260A1 (es) * | 2021-10-05 | 2024-01-03 | Dicerna Pharmaceuticals Inc | Composiciones y métodos para inhibir el receptor de melanocortina 2 y la expresión del citocromo p450 11b |
| JP2024543195A (ja) * | 2021-12-01 | 2024-11-19 | 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 | Ａｐｏｃ３発現を調節するための組成物及び方法 |
| JP2024546585A (ja) | 2021-12-15 | 2024-12-26 | ホンジーン バイオテック コーポレイション | 官能基化ｎ－アセチルガラクトサミンアナログ |
| US20260001902A1 (en) | 2022-09-19 | 2026-01-01 | Bachem Holding Ag | Improved oligonucleotide synthesis |
| JP2025531341A (ja) * | 2022-09-19 | 2025-09-19 | カイロンノヴァ （シャーメン） バイオファーマ カンパニー，リミテッド | 炭水化物－オリゴヌクレオチド複合体、薬物組成物および治療への応用 |
| WO2024083746A1 (en) | 2022-10-17 | 2024-04-25 | Bachem Holding Ag | Method and composition for oligonucleotide synthesis |
| WO2024118503A1 (en) | 2022-11-28 | 2024-06-06 | Hongene Biotech Corporation | Functionalized n-acetylgalactosamine analogs |
| EP4695265A1 (en) | 2023-10-16 | 2026-02-18 | Bachem Holding AG | Method and composition for oligonucleotide synthesis |
| US12553050B2 (en) | 2024-03-22 | 2026-02-17 | Takeda Pharmaceutical Company Limited | Compositions and methods for inhibiting cytochrome P450 family 7 subfamily a member 1 (CYP7A1) expression |
| CN118460650B (zh) * | 2024-07-10 | 2025-02-14 | 凯莱英医药集团（天津）股份有限公司 | 一种Nedosiran的制备方法 |
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| WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
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| CA2559955C (en) | 2004-03-15 | 2016-02-16 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded rna |
| WO2005116204A1 (ja) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
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| CA2729757A1 (en) | 2008-07-01 | 2010-01-07 | The Johns Hopkins University | Methods for treating neoplasia by inhibiting lactate dehydrogenase and/or nicotinamide phosphoribosyltransferase |
| US20110288147A1 (en) | 2008-09-22 | 2011-11-24 | Bob Dale Brown | Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop |
| DK2341943T3 (en) | 2008-09-22 | 2019-02-25 | Dicerna Pharmaceuticals Inc | COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS |
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| US20120121515A1 (en) | 2009-03-13 | 2012-05-17 | Lenny Dang | Methods and compositions for cell-proliferation-related disorders |
| SI2539451T1 (sl) | 2010-02-24 | 2016-04-29 | Arrowhead Research Corporation | Sestavki za ciljano dostavo sirna |
| CA2800728C (en) | 2010-05-28 | 2020-10-27 | Genentech, Inc. | Decreasing lactate level and increasing polypeptide production by downregulating the expression of lactate dehydrogenase and pyruvate dehydrogenase kinase |
| WO2012156513A1 (en) | 2011-05-18 | 2012-11-22 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Method for determining whether a subject is at risk of having or developing a chronic kidney disease |
| SI3366775T2 (sl) | 2011-11-18 | 2025-12-31 | Alnylam Pharmaceuticals, Inc. | Modificirana sredstva RNAI |
| TW201808342A (zh) | 2012-05-02 | 2018-03-16 | 喜納製藥公司 | 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法 |
| AU2015330670B2 (en) | 2014-10-10 | 2022-01-06 | Novo Nordisk Health Care Ag | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| DK3569711T3 (da) * | 2014-12-15 | 2021-02-22 | Dicerna Pharmaceuticals Inc | Ligandmodificerede dobbeltstrengede nukleinsyrer |
| AU2015364602B2 (en) * | 2014-12-18 | 2021-12-09 | The Regents Of The University Of California | Detection of nucleic acid polymerase conformational changes using a nanotube |
| AR104754A1 (es) * | 2015-05-29 | 2017-08-09 | Arrowhead Pharmaceuticals Inc | Agentes de unión de tetrapéptido escindibles biológicamente |
| WO2019075419A1 (en) | 2017-10-13 | 2019-04-18 | Dicerna Pharmaceuticals, Inc. | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
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2018
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- 2018-10-12 ES ES18867097T patent/ES2955045T3/es active Active
- 2018-10-12 HU HUE18867097A patent/HUE063026T2/hu unknown
- 2018-10-12 MX MX2020003836A patent/MX2020003836A/es unknown
- 2018-10-12 KR KR1020207013207A patent/KR102609396B1/ko active Active
- 2018-10-12 PL PL18867097.0T patent/PL3679141T3/pl unknown
- 2018-10-12 IL IL273875A patent/IL273875B2/en unknown
- 2018-10-12 KR KR1020237041035A patent/KR20230169413A/ko active Pending
- 2018-10-12 JP JP2020542046A patent/JP7308213B2/ja active Active
- 2018-10-12 CN CN202410538409.2A patent/CN118530989A/zh active Pending
- 2018-10-12 CA CA3078933A patent/CA3078933A1/en active Pending
- 2018-10-12 CN CN201880080394.2A patent/CN111448319B/zh active Active
- 2018-10-12 US US16/755,342 patent/US11286488B2/en active Active
- 2018-10-12 RS RS20230689A patent/RS64483B1/sr unknown
- 2018-10-12 AU AU2018346971A patent/AU2018346971B2/en active Active
- 2018-10-12 CN CN202410538408.8A patent/CN118440940A/zh active Pending
- 2018-10-12 EP EP23172235.6A patent/EP4265261A3/en active Pending
- 2018-10-12 HR HRP20231063TT patent/HRP20231063T1/hr unknown
- 2018-10-12 EP EP18867097.0A patent/EP3679141B1/en active Active
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2020
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2022
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2025
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