CA3163514A1 - Targeted transfer rnas for treatment of diseases - Google Patents

Targeted transfer rnas for treatment of diseases

Info

Publication number
CA3163514A1
CA3163514A1 CA3163514A CA3163514A CA3163514A1 CA 3163514 A1 CA3163514 A1 CA 3163514A1 CA 3163514 A CA3163514 A CA 3163514A CA 3163514 A CA3163514 A CA 3163514A CA 3163514 A1 CA3163514 A1 CA 3163514A1
Authority
CA
Canada
Prior art keywords
trna
variant
engineered
engineered trna
sequence
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
CA3163514A
Other languages
English (en)
French (fr)
Inventor
David HUSS
Liana STEIN
Richard Sullivan
Anupama Lakshmanan
Stephen BURLEIGH
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.)
Shape Therapeutics Inc
Original Assignee
Shape Therapeutics Inc
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 Shape Therapeutics Inc filed Critical Shape Therapeutics Inc
Publication of CA3163514A1 publication Critical patent/CA3163514A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • 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)
CA3163514A 2019-12-02 2020-12-01 Targeted transfer rnas for treatment of diseases Pending CA3163514A1 (en)

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
CA3163514A1 true CA3163514A1 (en) 2021-06-10

Family

ID=74046150

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3163514A Pending CA3163514A1 (en) 2019-12-02 2020-12-01 Targeted transfer rnas for treatment of diseases

Country Status (6)

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

Families Citing this family (11)

* 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
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

Family Cites Families (6)

* 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
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
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
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
US10968257B2 (en) * 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
CA3125567A1 (en) * 2019-01-18 2020-07-23 Flagship Pioneering, Inc. Trem compositions and uses thereof

Also Published As

Publication number Publication date
WO2021113218A1 (en) 2021-06-10
EP4069841A1 (en) 2022-10-12
AU2020396868A1 (en) 2022-07-14
CN115397983A (zh) 2022-11-25
JP2023504661A (ja) 2023-02-06

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Effective date: 20220920