CA3112809A1 - 2' fana modified foxp3 antisense oligonucleotides and methods of use thereof - Google Patents

2' fana modified foxp3 antisense oligonucleotides and methods of use thereof Download PDF

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Publication number
CA3112809A1
CA3112809A1 CA3112809A CA3112809A CA3112809A1 CA 3112809 A1 CA3112809 A1 CA 3112809A1 CA 3112809 A CA3112809 A CA 3112809A CA 3112809 A CA3112809 A CA 3112809A CA 3112809 A1 CA3112809 A1 CA 3112809A1
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Prior art keywords
fana
seq
aon
aum
nos
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Pending
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CA3112809A
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English (en)
French (fr)
Inventor
Veenu AISHWARYA
Wayne W. Hancock
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Children's Hospital of Philadelphia
AUM Lifetech Inc
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Children's Hospital of Philadelphia
AUM Lifetech Inc
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Application filed by Children's Hospital of Philadelphia, AUM Lifetech Inc filed Critical Children's Hospital of Philadelphia
Publication of CA3112809A1 publication Critical patent/CA3112809A1/en
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61N—ELECTROTHERAPY; MAGNETOTHERAPY; RADIATION THERAPY; ULTRASOUND THERAPY
    • A61N5/00—Radiation therapy
    • A61N5/10—X-ray therapy; Gamma-ray therapy; Particle-irradiation therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Radiology & Medical Imaging (AREA)
  • Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3112809A 2018-09-26 2019-09-25 2' fana modified foxp3 antisense oligonucleotides and methods of use thereof Pending CA3112809A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862737061P 2018-09-26 2018-09-26
US62/737,061 2018-09-26
US201862739001P 2018-09-28 2018-09-28
US62/739,001 2018-09-28
PCT/US2019/053033 WO2020069044A1 (en) 2018-09-26 2019-09-25 2' fana modified foxp3 antisense oligonucleotides and methods of use thereof

Publications (1)

Publication Number Publication Date
CA3112809A1 true CA3112809A1 (en) 2020-04-02

Family

ID=69953290

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3112809A Pending CA3112809A1 (en) 2018-09-26 2019-09-25 2' fana modified foxp3 antisense oligonucleotides and methods of use thereof

Country Status (7)

Country Link
US (1) US20210340536A1 (enExample)
EP (1) EP3856919A4 (enExample)
JP (1) JP2022502062A (enExample)
CN (1) CN112969799A (enExample)
AU (1) AU2019347849B2 (enExample)
CA (1) CA3112809A1 (enExample)
WO (1) WO2020069044A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202028222A (zh) * 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
EP3845646A1 (en) 2019-12-30 2021-07-07 Secarna Pharmaceuticals GmbH & Co. KG Modified antisense oligonucleotide for inhibition of foxp3 expression
WO2023235838A2 (en) * 2022-06-02 2023-12-07 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of foxp3-associated conditions and diseases

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE346918T1 (de) * 1998-06-19 2006-12-15 Univ Mcgill Auf beta-arabinose, und dessen analogen, basierte antisense oligonukleotide
AU4031700A (en) * 1999-04-06 2000-11-02 East Carolina University Low adenosine anti-sense oligonucleotide, compositions, kit and method for treatment of airway disorders associated with bronchoconstriction, lung inflammation,allergy(ies) and surfactant depletion
CA2419563C (en) * 2000-09-06 2018-03-20 Mcgill University Chimeric antisense of arabinofuranose analogue and deoxyribose nucleotides
DK1470144T3 (da) * 2002-02-01 2009-04-06 Univ Mcgill Oligonukleotider omfattende alternerende segmenter og anvendelser deraf
CA2652539A1 (en) * 2006-05-19 2007-11-29 Topigen Pharmaceuticals Inc. Oligonucleotides affecting expression of phosphodiesterases
WO2008066784A2 (en) * 2006-11-27 2008-06-05 Ludwig Institute For Cancer Research Expression of foxp3 by cancer cells
US20100310635A1 (en) * 2007-04-25 2010-12-09 Engin Ozkaynak Compositions and Methods for Regulating T-Cell Activity
EP2285961A4 (en) * 2008-05-15 2012-10-17 Topigen Pharmaceuticals Inc OLIGONUCLEOTIDES FOR THE TREATMENT OF INFLAMMATION AND NEOPLASTIC CELL REPRODUCTION
WO2010048673A1 (en) * 2008-10-30 2010-05-06 Murdoch Childrens Research Institute Modulation of cellular differentiation and uses therefor
WO2010065671A2 (en) * 2008-12-04 2010-06-10 Curna, Inc. Treatment of vascular endothelial growth factor (vegf) related diseases by inhibition of natural antisense transcript to vegf
KR20160027968A (ko) * 2013-06-07 2016-03-10 라나 테라퓨틱스, 인크. Ｆｏｘｐ3 발현을 조절하기 위한 조성물 및 방법
WO2017181026A1 (en) * 2016-04-15 2017-10-19 Translate Bio Ma, Inc. Selective modulation of foxp3 expression
US20200030361A1 (en) * 2016-09-23 2020-01-30 City Of Hope Oligonucleotides containing 2'-deoxy-2'fluoro-beta-d-arabinose nucleic acid (2'-fana) for treatment and diagnosis of retroviral diseases
FR3058061A1 (fr) * 2016-10-27 2018-05-04 Selexel Nouvelle utilisation d'oligonucleotides double brin

Also Published As

Publication number Publication date
AU2019347849B2 (en) 2025-08-28
EP3856919A4 (en) 2023-08-02
EP3856919A1 (en) 2021-08-04
WO2020069044A1 (en) 2020-04-02
CN112969799A (zh) 2021-06-15
AU2019347849A1 (en) 2021-05-20
JP2022502062A (ja) 2022-01-11
US20210340536A1 (en) 2021-11-04

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