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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
rna
oligonucleotide
splice
target
peptide epitope
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2021510744A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021536239A5 (https=
Inventor
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
Original Assignee
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
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 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 filed Critical 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
Publication of JP2021536239A publication Critical patent/JP2021536239A/ja
Publication of JP2021536239A5 publication Critical patent/JP2021536239A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39583Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials not provided for elsewhere, e.g. haptens, coenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/0005Vertebrate antigens
    • A61K39/0011Cancer antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/0005Vertebrate antigens
    • A61K39/0011Cancer antigens
    • A61K39/001154Enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1135Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/17Immunomodulatory nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Oncology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • 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 2018-08-28
EP18191271.8 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

Publications (2)

Publication Number Publication Date
JP2021536239A true JP2021536239A (ja) 2021-12-27
JP2021536239A5 JP2021536239A5 (https=) 2022-09-01

Family

ID=67742445

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021510744A Pending JP2021536239A (ja) 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

Country Status (5)

Country Link
US (1) US20220160870A1 (https=)
EP (1) EP3844274A1 (https=)
JP (1) JP2021536239A (https=)
CN (1) CN113728102A (https=)
WO (1) WO2020043750A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3918069A1 (en) * 2019-01-31 2021-12-08 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
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
AU2005206389A1 (en) * 2004-01-27 2005-08-04 Compugen Ltd. Methods of identifying putative gene products by interspecies sequence comparison and biomolecular sequences uncovered thereby
US8501703B2 (en) 2005-08-30 2013-08-06 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
WO2007047913A2 (en) 2005-10-20 2007-04-26 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
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP2623599B1 (en) * 2007-10-04 2019-01-02 Roche Innovation Center Copenhagen A/S Micromirs
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
EP2445538A2 (en) * 2009-06-23 2012-05-02 University Of Miami Aptamer-targeted sirna to inhibit nonsense mediated decay
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis 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
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
WO2014080381A1 (en) * 2012-11-26 2014-05-30 Ecole Polytechnique Federale De Lausanne (Epfl) Colorectal cancer classification with differential prognosis and personalized therapeutic responses
CA2910802A1 (en) 2013-04-12 2014-10-16 Samir El Andaloussi Therapeutic delivery vesicles
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
CN106795200B (zh) 2014-10-10 2020-06-19 豪夫迈·罗氏有限公司 Galnac亚磷酰胺、其核酸缀合物及其用途
EP3234134B1 (en) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
WO2016172598A1 (en) 2015-04-22 2016-10-27 The Broad Institute Inc. Exosomes and uses thereof
EP3323890A4 (en) 2015-07-14 2019-01-30 Fukuoka University METHOD OF INDUSTRYING SITE-SPECIFIC RNA MUTATIONS, TARGETED EDITING GUIDE RNA USED IN THE METHOD AND TARGET RNA TARGET EDITING GUIDE RNA COMPLEX
US11458097B2 (en) 2016-03-30 2022-10-04 The University Of North Carolina At Chapel Hill Biological agent-exosome compositions and uses thereof
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
US11820981B2 (en) * 2016-09-14 2023-11-21 Agency For Science, Technology And Research Modulation of TJP1 expression to regulate regeneration of heart cells
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
EP3571300A1 (en) 2017-01-19 2019-11-27 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
KR102761791B1 (ko) * 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
US10476825B2 (en) * 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions
WO2019071274A1 (en) 2017-10-06 2019-04-11 Oregon Health & Science University Compositions and methods for editing rna
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 *

Also Published As

Publication number Publication date
WO2020043750A1 (en) 2020-03-05
CN113728102A (zh) 2021-11-30
US20220160870A1 (en) 2022-05-26
WO2020043750A9 (en) 2020-05-22
EP3844274A1 (en) 2021-07-07

Similar Documents

Publication Publication Date Title
AU2022206704B2 (en) Nanoparticle formulations for delivery of nucleic acid complexes
KR102824513B1 (ko) 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드
CA3064103C (en) Compositions and methods for inhibiting gene expression of hepatitis b virus
CN114085836B (zh) 用于减少pd-l1表达的寡核苷酸
JP2021536239A (ja) スプライス調節化合物を使用したネオアンチゲン操作
US20170152511A9 (en) 5' targeting oligonucleotides for modulating rna
AU2013285698A1 (en) Oligonucleotide for the treatment of muscular dystrophy patients
US10023862B2 (en) Organic compositions to treat beta-catenin-related diseases
KR20240099149A (ko) 뒤센 근이영양증에서 엑손 45를 스키핑하기 위한 조성물 및 방법
TW202304519A (zh) FN3結構域-siRNA結合物及其用途
KR20240040112A (ko) 방법
TW201119681A (en) Compositions and methods for inhibiting expression of KIF10 genes
HK40062073A (en) Neoantigen engineering using splice modulating compounds
JP2024513131A (ja) オリゴヌクレオチド
WO2021010301A1 (ja) DUX4 pre-mRNAのスプライシングを変化させるアンチセンスオリゴヌクレオチド
WO2025111500A1 (en) Modulation of gene expression
KR20260030126A (ko) 고리형 핵산을 이용한 rna 치료제의 전달
WO2025076243A1 (en) Cell-targeting complexes and uses thereof
HK40061440A (zh) 用於抑制乙型肝炎病毒的基因表达的组合物和方法
CN121127248A (zh) 用于抑制线粒体偕胺肟还原组分1 (MARC1)表达的RNAi剂、其药用组合物及使用方法
WO2025006955A2 (en) Cd29 targeted oligonucleotides and uses thereof
TW202440921A (zh) 經修飾之寡核苷酸
TW201130494A (en) Compositions and methods for inhibiting expression of IL-18 genes
HK1193633B (en) Compositions and methods for inhibiting gene expression of hepatitis b virus

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220824

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220824

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230724

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231003

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240119

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20240305

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240502

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240730

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20241128