AU2022301997A1 - Engineered guide rnas and polynucleotides - Google Patents

Engineered guide rnas and polynucleotides Download PDF

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Publication number
AU2022301997A1
AU2022301997A1 AU2022301997A AU2022301997A AU2022301997A1 AU 2022301997 A1 AU2022301997 A1 AU 2022301997A1 AU 2022301997 A AU2022301997 A AU 2022301997A AU 2022301997 A AU2022301997 A AU 2022301997A AU 2022301997 A1 AU2022301997 A1 AU 2022301997A1
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AU
Australia
Prior art keywords
rna
target
relative
guide
internal loop
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
AU2022301997A
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English (en)
Inventor
Brian John BOOTH
Adrian Briggs
Yiannis SAVVA
Richard Thomas SULLIVAN
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 AU2022301997A1 publication Critical patent/AU2022301997A1/en
Pending legal-status Critical Current

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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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2022301997A 2021-06-29 2022-06-28 Engineered guide rnas and polynucleotides Pending AU2022301997A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163216178P 2021-06-29 2021-06-29
US63/216,178 2021-06-29
US202163277701P 2021-11-10 2021-11-10
US63/277,701 2021-11-10
US202263303680P 2022-01-27 2022-01-27
US63/303,680 2022-01-27
US202263345059P 2022-05-24 2022-05-24
US63/345,059 2022-05-24
PCT/US2022/035320 WO2023278449A1 (en) 2021-06-29 2022-06-28 Engineered guide rnas and polynucleotides

Publications (1)

Publication Number Publication Date
AU2022301997A1 true AU2022301997A1 (en) 2024-01-04

Family

ID=82701937

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022301997A Pending AU2022301997A1 (en) 2021-06-29 2022-06-28 Engineered guide rnas and polynucleotides

Country Status (6)

Country Link
US (1) US20240271139A1 (https=)
EP (1) EP4363579A1 (https=)
JP (1) JP2024528540A (https=)
AU (1) AU2022301997A1 (https=)
CA (1) CA3223109A1 (https=)
WO (1) WO2023278449A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3236391A1 (en) * 2021-10-26 2023-05-04 Yiannis SAVVA Rna-editing compositions and methods of use
WO2024137993A2 (en) * 2022-12-23 2024-06-27 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
WO2026006483A1 (en) * 2024-06-25 2026-01-02 Shape Therapeutics Inc. Snca vectors and engineered guide rnas
WO2026006334A1 (en) * 2024-06-25 2026-01-02 Shape Therapeutics Inc. Dux4 vectors and engineered guide rnas

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021113270A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
CA3177380A1 (en) * 2020-05-26 2021-12-02 Shape Therapeutics Inc. Compositions and methods for modifying target rnas
WO2022103852A1 (en) * 2020-11-11 2022-05-19 Shape Therapeutics Inc. Rna-editing compositions and methods of use
WO2022119975A2 (en) * 2020-12-01 2022-06-09 Shape Therapeutics Inc. High throughput rna-editing screening methods

Also Published As

Publication number Publication date
US20240271139A1 (en) 2024-08-15
WO2023278449A1 (en) 2023-01-05
CA3223109A1 (en) 2023-01-05
EP4363579A1 (en) 2024-05-08
JP2024528540A (ja) 2024-07-30

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