CN115397983A - 用于治疗疾病的靶向转移rna - Google Patents

用于治疗疾病的靶向转移rna Download PDF

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Publication number
CN115397983A
CN115397983A CN202080093906.6A CN202080093906A CN115397983A CN 115397983 A CN115397983 A CN 115397983A CN 202080093906 A CN202080093906 A CN 202080093906A CN 115397983 A CN115397983 A CN 115397983A
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trna
substitution
engineered
variant
seq
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Chinese (zh)
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大卫·胡斯
莉安娜·斯坦
理查德·沙利文
阿努帕马·拉克什马南
斯蒂芬·伯利
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Xipu Treatment Co
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Xipu Treatment Co
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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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • 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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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    • 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)
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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
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  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
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  • Molecular Biology (AREA)
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  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202080093906.6A 2019-12-02 2020-12-01 用于治疗疾病的靶向转移rna Pending CN115397983A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962942667P 2019-12-02 2019-12-02
US201962942690P 2019-12-02 2019-12-02
US62/942,667 2019-12-02
US62/942,690 2019-12-02
US202063010856P 2020-04-16 2020-04-16
US63/010,856 2020-04-16
US202063111856P 2020-11-10 2020-11-10
US63/111,856 2020-11-10
PCT/US2020/062671 WO2021113218A1 (en) 2019-12-02 2020-12-01 Targeted transfer rnas for treatment of diseases

Publications (1)

Publication Number Publication Date
CN115397983A true CN115397983A (zh) 2022-11-25

Family

ID=74046150

Family Applications (1)

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CN202080093906.6A Pending CN115397983A (zh) 2019-12-02 2020-12-01 用于治疗疾病的靶向转移rna

Country Status (6)

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EP (1) EP4069841A1 (https=)
JP (1) JP2023504661A (https=)
CN (1) CN115397983A (https=)
AU (1) AU2020396868A1 (https=)
CA (1) CA3163514A1 (https=)
WO (1) WO2021113218A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025036281A1 (en) * 2023-08-11 2025-02-20 Shanghai Codone Biotechnology Co. Engineered suppressor trna molecules and methods
WO2026001270A1 (en) * 2024-06-24 2026-01-02 Zhuhai Codone Biotechnology Co., Ltd. Engineered trna molecules and methods

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102930465B1 (ko) 2017-11-02 2026-02-25 더 위스타 인스티튜트 오브 아나토미 앤드 바이올로지 ACE-tRNA를 이용한 유전 재할당을 통한 정지 코돈의 구조 방법
MX2022012955A (es) 2020-04-14 2023-02-23 Flagship Pioneering Innovations Vi Llc Composiciones de trem y usos de las mismas.
WO2023097268A2 (en) * 2021-11-23 2023-06-01 Mantra Bio, Inc. Extracellular vesicles comprising non-naturally occurring modular rna hairpins and uses thereof
WO2023220342A2 (en) 2022-05-13 2023-11-16 Shape Therapeutics Inc. Engineered tranfer rnas
WO2024149415A1 (en) * 2023-01-10 2024-07-18 Biologicke Centrum Av Cr, V. V. I. Altered trnas for treatment of genetic diseases caused by premature termination codon
WO2024197081A1 (en) * 2023-03-20 2024-09-26 Hc Bioscience, Inc. Minimally-sized dna threads for trna therapy
EP4442826A1 (en) 2023-04-06 2024-10-09 Universität Hamburg Synthetic dna construct encoding transfer rna
EP4458967A1 (en) 2023-05-02 2024-11-06 Universität Hamburg Pharmaceutical composition comprising multiple suppressor transfer rnas
TW202504646A (zh) * 2023-06-16 2025-02-01 北京大學 利用環狀ｒｎａ抑制無義介導的降解的系統和方法

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108513575A (zh) * 2015-10-23 2018-09-07 哈佛大学的校长及成员们 核碱基编辑器及其用途
WO2018161032A1 (en) * 2017-03-03 2018-09-07 The Regents Of The University Of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
WO2019090169A1 (en) * 2017-11-02 2019-05-09 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with ace-trna
US20190300581A1 (en) * 2018-04-03 2019-10-03 The Broad Institute, Inc. Target recognition motifs and uses thereof

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012006551A2 (en) * 2010-07-08 2012-01-12 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
CA3125567A1 (en) * 2019-01-18 2020-07-23 Flagship Pioneering, Inc. Trem compositions and uses thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108513575A (zh) * 2015-10-23 2018-09-07 哈佛大学的校长及成员们 核碱基编辑器及其用途
WO2018161032A1 (en) * 2017-03-03 2018-09-07 The Regents Of The University Of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
WO2019090169A1 (en) * 2017-11-02 2019-05-09 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with ace-trna
US20190300581A1 (en) * 2018-04-03 2019-10-03 The Broad Institute, Inc. Target recognition motifs and uses thereof

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025036281A1 (en) * 2023-08-11 2025-02-20 Shanghai Codone Biotechnology Co. Engineered suppressor trna molecules and methods
WO2026001270A1 (en) * 2024-06-24 2026-01-02 Zhuhai Codone Biotechnology Co., Ltd. Engineered trna molecules and methods

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Publication number Publication date
WO2021113218A1 (en) 2021-06-10
EP4069841A1 (en) 2022-10-12
AU2020396868A1 (en) 2022-07-14
CA3163514A1 (en) 2021-06-10
JP2023504661A (ja) 2023-02-06

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