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MX2021014769A - Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos. - Google Patents

Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos.

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Publication number
MX2021014769A
MX2021014769A MX2021014769A MX2021014769A MX2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A
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MX
Mexico
Prior art keywords
glutathione
sensitive
moiety
oligonucleotide
compositions
Prior art date
Application number
MX2021014769A
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English (en)
Inventor
Venkata Krishnamurthy
Weimin Wang
Original Assignee
Dicerna Pharmaceuticals Inc
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Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of MX2021014769A publication Critical patent/MX2021014769A/es

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Abstract

En el presente documento se describen oligonucleótidos sensibles a glutatión y métodos para usarlos. Cualquier oligonucleótido de interés puede modificarse con una fracción sensible a glutatión, incluyendo los oligonucleótidos utilizados para liberación in vivo, tales como moléculas inhibidoras de ácido nucleico. Típicamente, la fracción sensible a glutatión se usa para modificar reversiblemente el carbono 2' de una fracción azúcar en uno o más nucleótidos en el oligonucleótido, aunque otras posiciones de carbono también pueden modificarse con la fracción sensible a glutatión. También se describen monómeros de nucleótidos y nucleósidos sensibles a glutatión, que incluyen fosforamiditos de nucleósido sensibles a glutatión que pueden usarse, por ejemplo, en métodos de síntesis de oligonucleótidos estándar. Además, los monómeros de nucleótidos y nucleósidos sensibles a glutatión sin un fosforamidito se pueden usar terapéuticamente, por ejemplo, como agentes antivirales.
MX2021014769A 2016-08-23 2019-02-21 Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos. MX2021014769A (es)

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Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017321892A1 (en) * 2016-09-02 2019-02-28 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
WO2019136157A2 (en) 2018-01-05 2019-07-11 Dicerna Pharmaceuticals, Inc. Reducing beta-catenin and ido expression to potentiate immunotherapy
CA3086409A1 (en) 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
SG11202110896WA (en) 2019-04-04 2021-10-28 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting gene expression in the central nervous system
JP2023517008A (ja) * 2020-03-03 2023-04-21 ネクスモス カンパニー リミテッド グルタチオンに特異的に結合するdnaアプタマー及びその用途
CA3187220A1 (en) 2020-08-04 2022-02-10 Dicerna Pharmaceuticals, Inc. Systemic delivery of oligonucleotides
WO2022077024A1 (en) 2020-10-08 2022-04-14 Dicerna Pharmaceuticals, Inc. Selective delivery of oligonucleotides to glial cells
WO2022147223A2 (en) * 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
TWI857290B (zh) 2021-04-14 2024-10-01 美商戴瑟納製藥股份有限公司 用於調節pnpla3表現之組合物及方法
CA3227661A1 (en) 2021-08-05 2023-02-09 Weimin Wang 1'-alkyl modified ribose derivatives and methods of use
US20230241224A1 (en) 2021-09-22 2023-08-03 Sanegene Bio Usa Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives and methods of use
US20230105319A1 (en) * 2021-09-24 2023-04-06 Crispr Therapeutics Ag PRODRUG INCORPORATED sgRNA SYNTHESIS
WO2023059695A1 (en) 2021-10-05 2023-04-13 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
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
CA3252536A1 (en) 2022-02-22 2023-08-31 Sanegene Bio Usa Inc. CARBOCYCLIC RIBONUCLEOTIDE DERIVATIVES MODIFIED AT THE 5' POSITION AND METHODS OF USE
KR20250005108A (ko) 2022-04-15 2025-01-09 다이서나 파마수이티컬, 인크. Scap 활성을 조절하기 위한 조성물 및 방법
WO2023220561A1 (en) 2022-05-09 2023-11-16 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
AU2023307152A1 (en) 2022-07-11 2025-01-09 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
US20240052348A1 (en) 2022-08-05 2024-02-15 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
AU2023358547A1 (en) 2022-10-14 2025-04-24 Sanegene Bio Usa Inc. Small interfering rna targeting c3 and uses thereof
WO2024112800A1 (en) * 2022-11-22 2024-05-30 Ionis Pharmaceuticals, Inc. Methods for deprotecting and purifying oligonucleotide compounds
CN120569477A (zh) 2022-12-19 2025-08-29 美国圣因生物股份有限公司 靶向cfb的小干扰rna及其用途
WO2024175111A2 (en) 2023-02-24 2024-08-29 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2024186673A2 (en) 2023-03-03 2024-09-12 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
US20250027089A1 (en) 2023-06-21 2025-01-23 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
WO2025024523A1 (en) 2023-07-24 2025-01-30 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6114513A (en) * 1990-01-11 2000-09-05 Isis Pharmaceuticals, Inc. Thiol-derivatized oligonucleotides
EP0537299A1 (en) 1990-03-29 1993-04-21 Gilead Sciences, Inc. Oligonucleotide-transport agent disulfide conjugates
US7282590B2 (en) 2004-02-12 2007-10-16 The Research Foundation Of State University Of New York Drug conjugates
US20110008395A1 (en) 2007-07-06 2011-01-13 Vladimir Torchilin Mixed micelles including amphipathic conjugates of rna agents, and uses thereof
EP3067359A1 (en) 2008-09-23 2016-09-14 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
KR101885383B1 (ko) 2009-07-06 2018-08-03 웨이브 라이프 사이언시스 리미티드 신규한 핵산 프로드러그 및 그의 사용 방법
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
WO2013188607A1 (en) * 2012-06-14 2013-12-19 The Trustees Of The University Of Pennsylvania One step n-terminal tagging of proteins
WO2014088923A1 (en) 2012-12-06 2014-06-12 Merck Sharp & Dohme Corp. Nucleoside kinase bypass compositions and methods
CN104903307A (zh) 2012-12-06 2015-09-09 默沙东公司 二硫化物屏蔽的前体药物组合物和方法
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
CN106061981A (zh) 2013-11-06 2016-10-26 索尔斯蒂斯生物有限公司 具有二硫化物基团的多核苷酸构建体
WO2015188197A2 (en) * 2014-06-06 2015-12-10 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups

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