WO2024020346A3 - Gene editing components, systems, and methods of use - Google Patents

Gene editing components, systems, and methods of use Download PDF

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Publication number
WO2024020346A3
WO2024020346A3 PCT/US2023/070339 US2023070339W WO2024020346A3 WO 2024020346 A3 WO2024020346 A3 WO 2024020346A3 US 2023070339 W US2023070339 W US 2023070339W WO 2024020346 A3 WO2024020346 A3 WO 2024020346A3
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WO
WIPO (PCT)
Prior art keywords
systems
methods
gene editing
editing components
typcv
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Application number
PCT/US2023/070339
Other languages
French (fr)
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WO2024020346A2 (en
Inventor
Giedrius GASIUNAS
Alim Ladha
Vladimir PRESNYAK
Muthusamy Jayaraman
Original Assignee
Renagade Therapeutics Management Inc.
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Publication date
Priority claimed from US18/297,346 external-priority patent/US20240084274A1/en
Application filed by Renagade Therapeutics Management Inc. filed Critical Renagade Therapeutics Management Inc.
Priority to US18/481,393 priority Critical patent/US20240141382A1/en
Publication of WO2024020346A2 publication Critical patent/WO2024020346A2/en
Publication of WO2024020346A3 publication Critical patent/WO2024020346A3/en

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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)

Abstract

The present disclosure provides methods and compositions comprising novel Cas TypcV programmable nucleases and lipid nanoparticles capable of delivering the Cas TypcV programmable nucleases and genome editing systems comprising same. For therapeutic applications, as well as plants and industrial biotechnology.
PCT/US2023/070339 2022-07-18 2023-07-17 Gene editing components, systems, and methods of use WO2024020346A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US18/481,393 US20240141382A1 (en) 2022-07-18 2023-10-05 Gene editing components, systems, and methods of use

Applications Claiming Priority (32)

Application Number Priority Date Filing Date Title
US202263368737P 2022-07-18 2022-07-18
US202263368724P 2022-07-18 2022-07-18
US202263368731P 2022-07-18 2022-07-18
US202263368730P 2022-07-18 2022-07-18
US202263368736P 2022-07-18 2022-07-18
US202263368734P 2022-07-18 2022-07-18
US202263368735P 2022-07-18 2022-07-18
US202263368738P 2022-07-18 2022-07-18
US202263368722P 2022-07-18 2022-07-18
US202263368741P 2022-07-18 2022-07-18
US202263368742P 2022-07-18 2022-07-18
US202263368726P 2022-07-18 2022-07-18
US202263368728P 2022-07-18 2022-07-18
US202263368744P 2022-07-18 2022-07-18
US63/368,737 2022-07-18
US63/368,742 2022-07-18
US63/368,738 2022-07-18
US63/368,735 2022-07-18
US63/368,724 2022-07-18
US63/368,726 2022-07-18
US63/368,744 2022-07-18
US63/368,730 2022-07-18
US63/368,731 2022-07-18
US63/368,734 2022-07-18
US63/368,741 2022-07-18
US63/368,722 2022-07-18
US63/368,728 2022-07-18
US63/368,736 2022-07-18
US18/297,346 US20240084274A1 (en) 2022-07-18 2023-04-07 Gene editing components, systems, and methods of use
US18/297,346 2023-04-07
US202363495198P 2023-04-10 2023-04-10
US63/495,198 2023-04-10

Related Parent Applications (1)

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US18/297,346 Continuation-In-Part US20240084274A1 (en) 2022-07-18 2023-04-07 Gene editing components, systems, and methods of use

Related Child Applications (1)

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US18/481,393 Continuation US20240141382A1 (en) 2022-07-18 2023-10-05 Gene editing components, systems, and methods of use

Publications (2)

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WO2024020346A3 true WO2024020346A3 (en) 2024-02-29

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