DK2560687T3 - Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types - Google Patents

Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types Download PDF

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Publication number
DK2560687T3
DK2560687T3 DK11718678.3T DK11718678T DK2560687T3 DK 2560687 T3 DK2560687 T3 DK 2560687T3 DK 11718678 T DK11718678 T DK 11718678T DK 2560687 T3 DK2560687 T3 DK 2560687T3
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conjugate
group
oligonucleotide
serotonin
nucleic acid
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DK11718678.3T
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Danish (da)
English (en)
Inventor
Urbina Gabriel Alvarado
Andrés Pablo Montefeltro
Biassoni Analia Bortolozzi
Pérez Francesc Artigas
Bover Miquel Vila
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Nlife Therapeutics S L
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Priority claimed from EP10382087A external-priority patent/EP2380595A1/en
Application filed by Nlife Therapeutics S L filed Critical Nlife Therapeutics S L
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Publication of DK2560687T3 publication Critical patent/DK2560687T3/en

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    • 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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    • 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
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    • A61K47/59—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
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    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
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    • C07D217/00—Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems
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    • C07D217/04—Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems with only hydrogen atoms or radicals containing only carbon and hydrogen atoms, directly attached to carbon atoms of the nitrogen-containing ring; Alkylene-bis-isoquinolines with hydrocarbon or substituted hydrocarbon radicals attached to the ring nitrogen atom
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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DK11718678.3T 2010-04-19 2011-04-19 Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types DK2560687T3 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US32551510P 2010-04-19 2010-04-19
EP10382087A EP2380595A1 (en) 2010-04-19 2010-04-19 Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
EP11382031 2011-02-09
PCT/EP2011/056270 WO2011131693A2 (en) 2010-04-19 2011-04-19 Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types

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DK2560687T3 true DK2560687T3 (en) 2017-09-18

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Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003057179A2 (en) 2002-01-11 2003-07-17 Biomarin Pharmaceutical, Inc. Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
CA2796722C (en) 2010-04-19 2021-06-01 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
CA2817960C (en) 2010-11-17 2020-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN103747807B (zh) 2011-07-05 2016-12-07 比奥阿赛斯技术有限公司 P97‑抗体缀合物和使用方法
CN107115352B (zh) 2011-08-04 2020-10-30 耶达研究及发展有限公司 微rna和包含微rna的组合物
WO2013086207A1 (en) * 2011-12-06 2013-06-13 The Ohio State University Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
EP2831232A4 (en) 2012-03-30 2015-11-04 Univ Washington METHOD FOR MODULATING TAU EXPRESSION TO REDUCE PROBLEMS AND MODIFY A NEURODEGENERATIVE SYNDROME
CA3140358A1 (en) 2012-07-31 2014-02-06 Bioasis Technologies, Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
AU2013336582A1 (en) * 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to cell types
EP2911695B1 (en) * 2012-10-26 2023-11-29 Palomo Limited Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
EP2743695A1 (en) 2012-12-12 2014-06-18 Nanogap Sub NM Powder, S.A. Methods and reagents for the detection of biomolecules using luminescence
AU2014243816B2 (en) 2013-03-13 2019-01-31 Bioasis Technologies Inc. Fragments of p97 and uses thereof
CN105264091B (zh) 2013-03-14 2020-02-28 Ionis制药公司 用于调节tau表达的组合物和方法
CN104178515B (zh) * 2013-05-21 2018-08-31 成都先导药物开发有限公司 一种化合物的细胞透膜的方法
WO2014187315A1 (zh) * 2013-05-21 2014-11-27 成都先导药物开发有限公司 一种化合物给药前体及药物载体制剂
EP3000898B1 (en) * 2013-05-21 2020-11-18 Hitgen Inc. Drug target capturing method
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
JP6603227B2 (ja) 2014-02-03 2019-11-06 バイオアシス テクノロジーズ インコーポレイテッド Ｐ９７融合タンパク質
CA2936158C (en) 2014-02-05 2023-06-13 Yeda Research And Development Co. Ltd. Use of mir-135 or precursor thereof for the treatment and diagnosis of a bipolar disease
DK3107562T3 (da) 2014-02-19 2019-12-16 Bioasis Technologies Inc P97-ids-fusionsproteiner
AU2015252906C1 (en) * 2014-05-01 2020-09-17 Bioasis Technologies Inc. p97-polynucleotide conjugates
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
US10733023B1 (en) * 2015-08-06 2020-08-04 D2Iq, Inc. Oversubscription scheduling
DK3133166T4 (da) * 2015-08-21 2023-03-20 42 Life Sciences Gmbh & Co Kg Sammensætning og fremgangsmåde til hybridisering
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
GB2574525B (en) * 2015-12-21 2020-09-02 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
GB2545898B (en) 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
DK3473270T5 (da) * 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
KR101916652B1 (ko) * 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
US10208098B2 (en) * 2016-09-15 2019-02-19 Council Of Scientific & Industrial Research Recombinant protein-based method for the delivery of silencer RNA to target the brain
HUE065170T2 (hu) 2016-09-29 2024-05-28 Biogen Ma Inc Vegyületek és módszerek a TAU expresszió csökkentésére
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
US11642365B2 (en) 2017-09-22 2023-05-09 John Mansell Compositions and methods for treatment of sepsis-related disorders
EP3461487A1 (en) * 2017-09-29 2019-04-03 Nlife Therapeutics S.L. Compositions and methods for the delivery of mrna to hepatic cells
HUE070436T2 (hu) 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére
CN111787942B (zh) 2017-10-25 2024-03-29 杨森制药公司 磷酸化Tau肽的组合物及其用途
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
SG11202006142PA (en) 2018-01-12 2020-07-29 Roche Innovation Ct Copenhagen As Alpha-synuclein antisense oligonucleotides and uses thereof
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
CN119264211A (zh) 2018-08-27 2025-01-07 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
ES2698565B2 (es) * 2018-12-12 2019-07-16 Univ Madrid Complutense Procedimiento para la elaboración de nanopartículas lipídicas, y nanopartículas lipídicas con los macrófagos cerebrales como células diana
MY206596A (en) 2019-02-08 2024-12-25 Janssen Pharmaceuticals Inc Method of safe administration of phosphorylated tau peptide vaccine
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
US12478684B2 (en) 2019-04-11 2025-11-25 Northeastern University Oligonucleotide-polymer miktoarm conjugates and methods of use
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
JP2024503711A (ja) * 2021-01-18 2024-01-26 イェダ リサーチ アンド デベロップメント カンパニー リミテッド 修飾ｍｉＲ－１３５、そのコンジュゲートおよびその使用
WO2024069229A2 (en) 2022-08-03 2024-04-04 Sutura Therapeutics Ltd Biologically active compounds
US12396981B2 (en) 2023-03-09 2025-08-26 William Shulman Methods of using DMT

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003042162A1 (en) * 2001-11-17 2003-05-22 Neurosearch A/S Prodrugs of antidepressants and their use for treating depressions
EP1336406A1 (en) * 2002-02-14 2003-08-20 Solvay Pharmaceuticals B.V. Partial dopamine-D2 receptor agonist plus serotonin and/or noradrenaline inhibitory activity
JP2007525211A (ja) * 2004-01-19 2007-09-06 テクニオン リサーチ アンド ディベロップメント ファウンデーション リミテッド パーキンソン病の診断検査
WO2007050789A2 (en) * 2005-10-25 2007-05-03 Nëdken Corporation Glycosyl-oligonucleotide conjugates and methods
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
WO2008033285A2 (en) * 2006-09-15 2008-03-20 The Trustees Of Culumbia University In The City Of New York Delivery of double-stranded rna into the central nervous system
BRPI0821310A2 (pt) * 2007-12-20 2015-06-16 Angiochem Inc Conjugados polipeptídeos-ácido nucleico e usos dos mesmos
GB0809476D0 (en) * 2008-05-23 2008-07-02 Cambridge Entpr Ltd Treatment of neurodegenerative disorders
DE102009058769A1 (de) * 2009-12-16 2011-06-22 MagForce Nanotechnologies AG, 10589 Temperaturabhängige Aktivierung von katalytischen Nukleinsäuren zur kontrollierten Wirkstofffreisetzung
CA2796722C (en) 2010-04-19 2021-06-01 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
CA2817960C (en) * 2010-11-17 2020-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
AU2013336582A1 (en) 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to cell types
EP2911695B1 (en) 2012-10-26 2023-11-29 Palomo Limited Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types

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