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MX2024001378A - Oligonucleotidos multi-conjugados definidos. - Google Patents

Oligonucleotidos multi-conjugados definidos.

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Publication number
MX2024001378A
MX2024001378A MX2024001378A MX2024001378A MX2024001378A MX 2024001378 A MX2024001378 A MX 2024001378A MX 2024001378 A MX2024001378 A MX 2024001378A MX 2024001378 A MX2024001378 A MX 2024001378A MX 2024001378 A MX2024001378 A MX 2024001378A
Authority
MX
Mexico
Prior art keywords
defined multi
conjugate
conjugate oligonucleotides
oligonucleotides
sirna
Prior art date
Application number
MX2024001378A
Other languages
English (en)
Inventor
Philipp Hadwiger
Jonathan Miles Brown
James Everett Dahlman
Kristin K H Neuman
Hans-Peter Vornlocher
Original Assignee
Mpeg La Llc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Mpeg La Llc filed Critical Mpeg La Llc
Publication of MX2024001378A publication Critical patent/MX2024001378A/es

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    • 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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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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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    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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Abstract

Se describen oligonucleótidos multi-conjugados definidos pueden tener tamaños y composiciones predeterminados. Por ejemplo, en diversas modalidades, los oligonucleótidos multi-conjugados definidos pueden tener propiedades adecuadas, por ejemplo en forma de siARN multi-conjugado definido (es decir, que incluye dos, tres o más siARN) que tiene administración intracelular potenciada y/o efectos de silenciamiento multigénico. En diversas modalidades, los oligonucleótidos multi-conjugados definidos pueden sintetizarse a través de nuevos intermedios sintéticos y métodos. Los oligonucleótidos multi-conjugados definidos pueden usarse, por ejemplo, para reducir la expresión génica, investigación biológica, tratar o prevenir afecciones médicas, o para producir fenotipos nuevos o alterados en células y organismos.
MX2024001378A 2015-06-15 2017-12-11 Oligonucleotidos multi-conjugados definidos. MX2024001378A (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562175709P 2015-06-15 2015-06-15
US201562175714P 2015-06-15 2015-06-15
US201562175718P 2015-06-15 2015-06-15
US201562203243P 2015-08-10 2015-08-10
US201562216317P 2015-09-09 2015-09-09
US201562216314P 2015-09-09 2015-09-09
US201562216318P 2015-09-09 2015-09-09

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MX2024001378A true MX2024001378A (es) 2024-02-27

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MX2017016088A MX2017016088A (es) 2015-06-15 2016-06-15 Oligonucleotidos multi-conjugados definidos.
MX2024001378A MX2024001378A (es) 2015-06-15 2017-12-11 Oligonucleotidos multi-conjugados definidos.

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US (3) US11352629B2 (es)
EP (2) EP4541374A2 (es)
JP (1) JP7033452B2 (es)
KR (2) KR20250016526A (es)
CN (1) CN108026527B (es)
AU (3) AU2016280709B2 (es)
CA (1) CA2988603A1 (es)
HK (1) HK1252970A1 (es)
IL (2) IL316159A (es)
MX (2) MX2017016088A (es)
WO (1) WO2016205410A2 (es)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
AU2017296195A1 (en) 2016-07-11 2019-01-24 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
US11299733B2 (en) 2016-07-15 2022-04-12 University Of Massachusetts Process of delivering small RNAs to sperm
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
SG11201906728TA (en) * 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
CA3075219A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited Hnf4a sarna compositions and methods of use
EP3737424A4 (en) * 2018-01-10 2021-10-27 Translate Bio MA, Inc. COMPOSITIONS AND METHODS TO FACILITATE THE DELIVERY OF SYNTHETIC NUCLEIC ACIDS INTO CELLS
WO2019194599A1 (ko) 2018-04-05 2019-10-10 주식회사 엘지화학 카바졸계 화합물 및 이를 포함하는 유기 발광 소자
AU2019316640A1 (en) 2018-08-10 2021-03-18 University Of Massachusetts Modified oligonucleotides targeting SNPs
EP3840759A4 (en) 2018-08-23 2022-06-01 University Of Massachusetts O-METHYL RICH FULLY STABILIZED OLIGONUCLEOTIDES
BR112021005777A2 (pt) * 2018-09-28 2022-04-19 Sirnaomics Inc Produtos e composições
AU2020207935A1 (en) 2019-01-18 2021-08-26 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
CN113994004A (zh) * 2019-03-04 2022-01-28 Mpeg La有限责任公司 具有增强生物活性的多聚寡核苷酸
EP3952919A2 (en) * 2019-04-11 2022-02-16 Northeastern University Oligonucleotide-polymer miktoarm conjugates and methods of use
CN114466929A (zh) * 2019-07-30 2022-05-10 Mpeg La有限责任公司 具有增强的生物活性的多聚体寡核苷酸的皮下递送
WO2021030213A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
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
CN112111524B (zh) * 2020-01-10 2024-02-27 深圳瑞吉生物科技有限公司 mRNA-GalNAc靶向分子的制备方法及其体内递送系统和应用
IL297570A (en) * 2020-04-30 2022-12-01 Mpeg La Llc Multimeric oligonucleotides with divided strands
IL298095A (en) * 2020-05-19 2023-01-01 Mpeg La L L C Orthogonally linked multimeric oligonucleotides
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
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
CN117377764A (zh) * 2021-02-08 2024-01-09 中美瑞康核酸技术(南通)研究院有限公司 多价寡核苷酸试剂及其使用方法
MX2023015523A (es) 2021-06-23 2024-03-11 Univ Massachusetts Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogenicos.
CN115010794A (zh) * 2022-06-30 2022-09-06 武汉瑞佶生物科技有限公司 蛋白质介导的mRNA靶向分子及其制备方法和应用
EP4590339A1 (en) * 2022-09-19 2025-07-30 Kylonova (Xiamen) Biopharma Co., Ltd. Carbohydrate-oligonucleotide conjugates, pharmaceutical compositions, and therapeutic applications
WO2024228030A2 (en) 2023-05-04 2024-11-07 Argonaute RNA Limited Dual silencing

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
GB9207381D0 (en) 1992-04-03 1992-05-13 Ici Plc Synthesis of oligonucleotides
US8202846B2 (en) 2000-03-16 2012-06-19 Cold Spring Harbor Laboratory Methods and compositions for RNA interference
JP2003526367A (ja) 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド Ｒｎａ干渉の方法とｒｎａ干渉組成物
JP4371812B2 (ja) 2001-09-28 2009-11-25 マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ マイクロｒｎａ分子
AU2003282722A1 (en) 2002-10-02 2004-04-23 Alnylam Pharmaceuticals Inc. Therapeutic compositions
US8110674B2 (en) 2003-03-07 2012-02-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
US8969543B2 (en) 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
CA2521464C (en) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Irna conjugates
JP5209204B2 (ja) 2003-04-13 2013-06-12 エンゾン ファーマスーティカルズ インコーポレイテッド ポリマーオリゴヌクレオチドプロドラッグ
EP1625138A4 (en) 2003-04-17 2010-06-23 Alnylam Pharmaceuticals Inc PROTECTED MONOMERS
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
EP2660322A3 (en) 2003-04-17 2013-11-13 Alnylam Pharmaceuticals Inc. Modified iRNA agents
WO2006046148A2 (en) 2004-10-25 2006-05-04 Devgen Nv Rna constructs
ES2503739T3 (es) 2004-11-12 2014-10-07 Asuragen, Inc. Procedimientos y composiciones que implican miARN y moléculas inhibidoras de miARN
ES2564303T3 (es) 2006-04-07 2016-03-21 Idera Pharmaceuticals, Inc. Compuestos de ARN inmunomodulador estabilizado (SIMRA) para TLR7 y TLR8
AU2007345648A1 (en) 2007-01-26 2008-08-07 City Of Hope Methods and compositions for the treatment of cancer or other diseases
US20080287383A1 (en) 2007-03-02 2008-11-20 Nastech Pharmaceutical Company Inc. Nucleic acid compounds for inhibiting erbb gene expression and uses thereof
US20080311040A1 (en) * 2007-03-06 2008-12-18 Flagship Ventures METHODS AND COMPOSITIONS FOR IMPROVED THERAPEUTIC EFFECTS WITH siRNA
EP2178567A4 (en) 2007-07-09 2010-11-03 Idera Pharmaceuticals Inc STABILIZED IMMUNOMODULATOR RNA COMPOUNDS (SIMRA)
EP2274425A2 (en) * 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2010021718A1 (en) 2008-08-19 2010-02-25 Nektar Therapeutics Complexes of small-interfering nucleic acids
US8637194B2 (en) * 2008-09-02 2014-01-28 Bio-Nano Power, Llc Bio-nano power cells and their uses
EP2370451B1 (en) * 2008-12-02 2016-11-16 Wave Life Sciences Japan, Inc. Method for the synthesis of phosphorus atom modified nucleic acids
WO2010090762A1 (en) * 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
KR101141544B1 (ko) * 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
CN104673795A (zh) 2009-08-27 2015-06-03 艾德拉药物股份有限公司 用于抑制基因表达的组合物及其用途
KR101678876B1 (ko) * 2010-01-15 2016-11-23 한국과학기술원 복합 유전자를 표적하는 ｓｉＲＮＡ 다중 접합체 및 이의 제조방법
CN102869774B (zh) 2010-02-24 2018-02-09 箭头研究公司 用于靶向递送siRNA的组合物
CA2791974A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
MX346144B (es) 2010-12-17 2017-03-09 Arrowhead Res Corp * Porcion activadora del modulador farmacocinetico del agregado de galactosa para arnsi.
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
EP2734208B1 (en) * 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
CA2848753C (en) 2011-09-14 2022-07-26 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
KR101340290B1 (ko) 2011-09-14 2013-12-11 한국과학기술원 유전자 표적용 ｓｉＲＮＡ 하이드로젤 및 그 제조방법
HK1206779A1 (en) 2012-04-02 2016-01-15 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
PT3241902T (pt) 2012-05-25 2018-05-28 Univ California Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição
AU2013315225B2 (en) 2012-09-14 2018-11-08 Translate Bio Ma, Inc. Multimeric oligonucleotide compounds
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
KR101629681B1 (ko) * 2013-06-24 2016-06-14 건국대학교 산학협력단 다중 리간드가 도입된 에스아이알엔에이 접합체
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
SG11201604692UA (en) 2013-12-12 2016-07-28 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
WO2015113922A1 (en) 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Poly oligomer compound with biocleavable conjugates
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
WO2017075030A1 (en) 2015-10-26 2017-05-04 Rana Therapeutics, Inc. Methods and compositions for increasing smn expression
SG10202001580SA (en) 2016-01-05 2020-04-29 Univ Leicester Methods for inhibiting fibrosis in a subject in need thereof
SG11201906728TA (en) 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
EP3733683A4 (en) 2017-11-30 2022-04-06 GeneMind Biosciences Company Limited NUCLEOSIDE ANALOGUES, METHOD OF MANUFACTURE AND USE
CN114466929A (zh) 2019-07-30 2022-05-10 Mpeg La有限责任公司 具有增强的生物活性的多聚体寡核苷酸的皮下递送

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