JP2021536239A - スプライス調節化合物を使用したネオアンチゲン操作 - Google Patents

スプライス調節化合物を使用したネオアンチゲン操作 Download PDF

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JP2021536239A
JP2021536239A JP2021510744A JP2021510744A JP2021536239A JP 2021536239 A JP2021536239 A JP 2021536239A JP 2021510744 A JP2021510744 A JP 2021510744A JP 2021510744 A JP2021510744 A JP 2021510744A JP 2021536239 A JP2021536239 A JP 2021536239A
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rna
oligonucleotide
splice
target
peptide epitope
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JP2021536239A5 (enExample
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カミレ ドゥモン エリクセン
マッツ イェンセン
トロルス コッホ
ヨーナス ヴィケサー
ジャンルイジ リチンチ
ラース ヨーンソン
クラウス イェンセン
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ロシュ イノベーション センター コペンハーゲン エーエス
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    • A—HUMAN NECESSITIES
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    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39583—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials not provided for elsewhere, e.g. haptens, coenzymes
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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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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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
    • C12N15/113—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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    • C12N15/00—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
    • C12N15/113—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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Wood Science & Technology (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
JP2021510744A 2018-08-28 2019-08-28 スプライス調節化合物を使用したネオアンチゲン操作 Pending JP2021536239A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
EP18191271.8 2018-08-28
EP18191271 2018-08-28
EP18194983.5 2018-09-18
EP18194983 2018-09-18
EP19178168.1 2019-06-04
EP19178168 2019-06-04
PCT/EP2019/072898 WO2020043750A1 (en) 2018-08-28 2019-08-28 Neoantigen engineering using splice modulating compounds

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JP2021536239A true JP2021536239A (ja) 2021-12-27
JP2021536239A5 JP2021536239A5 (enExample) 2022-09-01

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US (1) US20220160870A1 (enExample)
EP (1) EP3844274A1 (enExample)
JP (1) JP2021536239A (enExample)
CN (1) CN113728102A (enExample)
WO (1) WO2020043750A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220098578A1 (en) * 2019-01-31 2022-03-31 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
WO2023235838A2 (en) * 2022-06-02 2023-12-07 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of foxp3-associated conditions and diseases
CN118615431A (zh) * 2023-03-08 2024-09-10 广州医科大学 一种抗原表位在制备治疗肿瘤的产品中的应用
CN118620958A (zh) * 2023-03-08 2024-09-10 广州医科大学 一种抗原表位的制备方法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015534811A (ja) * 2012-11-02 2015-12-07 バウアー，ヨハン 癌の治療に使用されるｒｎａトランススプライシング分子（ｒｔｍ）
WO2018083071A1 (en) * 2016-11-02 2018-05-11 Universität Basel Immunologically discernible cell surface variants for use in cell therapy
JP2018522822A (ja) * 2015-05-20 2018-08-16 ザ・ブロード・インスティテュート・インコーポレイテッド 共通ネオ抗原

Family Cites Families (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
EP1015469B2 (en) 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
BRPI0008131B8 (pt) 1999-02-12 2021-05-25 Daiichi Sankyo Co Ltd composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US20070082337A1 (en) * 2004-01-27 2007-04-12 Compugen Ltd. Methods of identifying putative gene products by interspecies sequence comparison and biomolecular sequences uncovered thereby
EP2284269B1 (en) 2002-11-18 2017-08-09 Roche Innovation Center Copenhagen A/S Antisense design
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
WO2007058894A2 (en) 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
DK2205737T3 (da) * 2007-10-04 2013-05-21 Santaris Pharma As Mikromirer
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2445538A2 (en) * 2009-06-23 2012-05-02 University Of Miami Aptamer-targeted sirna to inhibit nonsense mediated decay
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US8846637B2 (en) 2010-06-08 2014-09-30 Isis Pharmaceuticals, Inc. Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
EP2922972B1 (en) * 2012-11-26 2017-04-26 Ecole Polytechnique Fédérale de Lausanne (EPFL) Colorectal cancer classification with differential prognosis and personalized therapeutic responses
EP2983721B2 (en) 2013-04-12 2021-01-20 Evox Therapeutics Limited Therapeutic delivery vesicles
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
US10358643B2 (en) 2014-01-30 2019-07-23 Hoffmann-La Roche, Inc. Poly oligomer compound with biocleavable conjugates
AU2015329974B2 (en) 2014-10-10 2019-06-20 F. Hoffmann-La Roche Ag GaINAc phosphoramidites, nucleic acid conjugates thereof and their use
AU2015367378A1 (en) 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
WO2016172598A1 (en) 2015-04-22 2016-10-27 The Broad Institute Inc. Exosomes and uses thereof
WO2017010556A1 (ja) 2015-07-14 2017-01-19 学校法人福岡大学 部位特異的ｒｎａ変異導入方法およびそれに使用する標的編集ガイドｒｎａならびに標的ｒｎａ－標的編集ガイドｒｎａ複合体
US11458097B2 (en) 2016-03-30 2022-10-04 The University Of North Carolina At Chapel Hill Biological agent-exosome compositions and uses thereof
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
WO2018041973A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
EP3512528A4 (en) * 2016-09-14 2020-05-13 Agency for Science, Technology and Research MODULATION OF THE TJP1 EXPRESSION FOR REGULATING THE REGENERATION OF HEART CELLS
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
WO2018141027A1 (en) * 2017-02-03 2018-08-09 The University Of Western Australia Novel treatment for neat1 associated disease
CN110612353A (zh) 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
CN111328290A (zh) * 2017-06-26 2020-06-23 博德研究所 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法
US10476825B2 (en) * 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions
IL316453A (en) 2017-10-06 2024-12-01 Univ Oregon Health & Science RNA editing agents and methods
US20200332272A1 (en) 2017-10-23 2020-10-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015534811A (ja) * 2012-11-02 2015-12-07 バウアー，ヨハン 癌の治療に使用されるｒｎａトランススプライシング分子（ｒｔｍ）
JP2018522822A (ja) * 2015-05-20 2018-08-16 ザ・ブロード・インスティテュート・インコーポレイテッド 共通ネオ抗原
WO2018083071A1 (en) * 2016-11-02 2018-05-11 Universität Basel Immunologically discernible cell surface variants for use in cell therapy

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ACTA HISTOCHEM., vol. 120, [1], JPN6023029665, January 2018 (2018-01-01), pages 56 - 63, ISSN: 0005471716 *
BREAST CANCER RES., vol. 20, [1], JPN6023029664, July 2018 (2018-07-01), pages 73 - 1, ISSN: 0005471717 *

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WO2020043750A9 (en) 2020-05-22
CN113728102A (zh) 2021-11-30
EP3844274A1 (en) 2021-07-07
US20220160870A1 (en) 2022-05-26

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