AU2020263581A1 - Methods and compositions for modulating splicing of alternative introns - Google Patents

Methods and compositions for modulating splicing of alternative introns Download PDF

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Publication number
AU2020263581A1
AU2020263581A1 AU2020263581A AU2020263581A AU2020263581A1 AU 2020263581 A1 AU2020263581 A1 AU 2020263581A1 AU 2020263581 A AU2020263581 A AU 2020263581A AU 2020263581 A AU2020263581 A AU 2020263581A AU 2020263581 A1 AU2020263581 A1 AU 2020263581A1
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Prior art keywords
fold
mrna
aic
intron
therapeutic agent
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AU2020263581A
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English (en)
Inventor
Isabel AZNAREZ
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Stoke Therapeutics Inc
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Stoke Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • C12N15/1138—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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2020263581A 2019-04-26 2020-04-24 Methods and compositions for modulating splicing of alternative introns Pending AU2020263581A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962839572P 2019-04-26 2019-04-26
US62/839,572 2019-04-26
PCT/US2020/029953 WO2020219977A1 (en) 2019-04-26 2020-04-24 Methods and compositions for modulating splicing of alternative introns

Publications (1)

Publication Number Publication Date
AU2020263581A1 true AU2020263581A1 (en) 2021-12-16

Family

ID=72941393

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020263581A Pending AU2020263581A1 (en) 2019-04-26 2020-04-24 Methods and compositions for modulating splicing of alternative introns

Country Status (10)

Country Link
US (1) US20220162599A1 (https=)
EP (1) EP3959313A4 (https=)
JP (2) JP2022532998A (https=)
KR (1) KR20220019676A (https=)
CN (1) CN114127286A (https=)
AU (1) AU2020263581A1 (https=)
BR (1) BR112021021384A2 (https=)
CA (1) CA3134947A1 (https=)
MX (1) MX2021013081A (https=)
WO (1) WO2020219977A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023348346A1 (en) * 2022-09-30 2025-03-27 Contera Pharma A/S Antisense oligonucleotides for the treatment of canavan disease
CN121059628A (zh) * 2024-06-03 2025-12-05 时夕(广州)生物科技有限公司 调控RNA剪接的向导agRNA

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060058253A1 (en) * 2002-08-12 2006-03-16 Benoit Chabot Methods to reprogram splice site selection in pre-messenger rnas
EP2555778A4 (en) * 2010-04-06 2014-05-21 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING CD274 / PD-L1 GENE EXPRESSION
AU2015206603B9 (en) * 2014-01-14 2019-07-18 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of melanoma using PD-L1 isoforms
AU2015327836B2 (en) * 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2017100587A1 (en) * 2015-12-09 2017-06-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting programmed cell death 1 ligand 1 (pd-l1) and methods of use 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
RU2747822C2 (ru) * 2016-03-14 2021-05-14 Ф. Хоффманн-Ля Рош Аг Олигонуклеотиды для понижения экспрессии pd-l1
SG10201705285SA (en) * 2017-06-27 2019-01-30 Agency Science Tech & Res Antisense oligonucleotides

Also Published As

Publication number Publication date
EP3959313A4 (en) 2023-09-27
BR112021021384A2 (pt) 2021-12-21
US20220162599A1 (en) 2022-05-26
CN114127286A (zh) 2022-03-01
KR20220019676A (ko) 2022-02-17
MX2021013081A (es) 2021-12-10
WO2020219977A1 (en) 2020-10-29
EP3959313A1 (en) 2022-03-02
JP2022532998A (ja) 2022-07-21
JP2025128180A (ja) 2025-09-02
CA3134947A1 (en) 2020-10-29

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