CA3156791A1 - Methods and compositions for treating a premature termination codon-mediated disorder - Google Patents

Methods and compositions for treating a premature termination codon-mediated disorder Download PDF

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Publication number
CA3156791A1
CA3156791A1 CA3156791A CA3156791A CA3156791A1 CA 3156791 A1 CA3156791 A1 CA 3156791A1 CA 3156791 A CA3156791 A CA 3156791A CA 3156791 A CA3156791 A CA 3156791A CA 3156791 A1 CA3156791 A1 CA 3156791A1
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Prior art keywords
trna
seq
gene
expression vector
nucleotide sequence
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Pending
Application number
CA3156791A
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English (en)
French (fr)
Inventor
Peter M. EIMON
Sean MCFARLAND
Ying-Hsin Chen
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.)
Tevard Biosciences Inc
Original Assignee
Chen Ying Hsin
Eimon Peter M
Mcfarland Sean
Tevard Biosciences Inc
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Application filed by Chen Ying Hsin, Eimon Peter M, Mcfarland Sean, Tevard Biosciences Inc filed Critical Chen Ying Hsin
Publication of CA3156791A1 publication Critical patent/CA3156791A1/en
Pending legal-status Critical Current

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    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CA3156791A 2019-11-01 2020-10-30 Methods and compositions for treating a premature termination codon-mediated disorder Pending CA3156791A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962929428P 2019-11-01 2019-11-01
US62/929,428 2019-11-01
PCT/US2020/058415 WO2021087401A1 (en) 2019-11-01 2020-10-30 Methods and compositions for treating a premature termination codon-mediated disorder

Publications (1)

Publication Number Publication Date
CA3156791A1 true CA3156791A1 (en) 2021-05-06

Family

ID=75715359

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3156791A Pending CA3156791A1 (en) 2019-11-01 2020-10-30 Methods and compositions for treating a premature termination codon-mediated disorder

Country Status (8)

Country Link
US (1) US12697350B2 (enExample)
EP (1) EP4051291A4 (enExample)
JP (2) JP7712270B2 (enExample)
KR (1) KR20220155981A (enExample)
CN (2) CN119955780A (enExample)
AU (1) AU2020375040A1 (enExample)
CA (1) CA3156791A1 (enExample)
WO (1) WO2021087401A1 (enExample)

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US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
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EP4334450A4 (en) * 2021-05-05 2025-06-18 Tevard Biosciences, Inc. Methods and compositions for treating a premature termination codon-mediated disorder
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EP4458967A1 (en) 2023-05-02 2024-11-06 Universität Hamburg Pharmaceutical composition comprising multiple suppressor transfer rnas
WO2025111506A1 (en) * 2023-11-22 2025-05-30 Tevard Biosciences, Inc. Modified transfer rnas and methods of use
WO2025119204A1 (en) * 2023-12-04 2025-06-12 Modit Therapeutics Beijing Limited Methods and compositions for improved readthrough of pseudouridine-modified codons
WO2026030209A1 (en) * 2024-07-29 2026-02-05 University Of Rochester Use of decoding trnas to boost protein expression or function
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Also Published As

Publication number Publication date
JP2025148444A (ja) 2025-10-07
US20240148772A1 (en) 2024-05-09
CN115209902B (zh) 2025-01-07
CN119955780A (zh) 2025-05-09
AU2020375040A1 (en) 2022-05-19
CN115209902A (zh) 2022-10-18
JP2022554357A (ja) 2022-12-28
JP7712270B2 (ja) 2025-07-23
WO2021087401A1 (en) 2021-05-06
EP4051291A1 (en) 2022-09-07
KR20220155981A (ko) 2022-11-24
US12697350B2 (en) 2026-08-04
EP4051291A4 (en) 2024-07-03

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