WO2023220654A2 - Compositions de protéines effectrices et procédés d'utilisation associés - Google Patents

Compositions de protéines effectrices et procédés d'utilisation associés Download PDF

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Publication number
WO2023220654A2
WO2023220654A2 PCT/US2023/066848 US2023066848W WO2023220654A2 WO 2023220654 A2 WO2023220654 A2 WO 2023220654A2 US 2023066848 W US2023066848 W US 2023066848W WO 2023220654 A2 WO2023220654 A2 WO 2023220654A2
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
sequence
effector protein
seq
amino acid
Prior art date
Application number
PCT/US2023/066848
Other languages
English (en)
Other versions
WO2023220654A3 (fr
Inventor
Sean Chen
Lucas Benjamin HARRINGTON
William Douglass WRIGHT
Aaron DELOUGHERY
Wiputra Jaya HARTONO
Benjamin Julius RAUCH
Stepan TYMOSHENKO
Original Assignee
Mammoth Biosciences, 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 Mammoth Biosciences, Inc. filed Critical Mammoth Biosciences, Inc.
Publication of WO2023220654A2 publication Critical patent/WO2023220654A2/fr
Publication of WO2023220654A3 publication Critical patent/WO2023220654A3/fr

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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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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
    • 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 [CRISPRs]
    • 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

Abstract

La présente divulgation concerne des compositions, des systèmes et des procédés comprenant des protéines effectrices et des utilisations associées. Ces protéines effectrices peuvent être caractérisées en tant que protéines associées à CRISPR (Cas). Diverses compositions, divers systèmes et procédés de la présente divulgation peuvent tirer profit des activités de ces protéines effectrices pour la modification et l'ingénierie d'acides nucléiques.
PCT/US2023/066848 2022-05-10 2023-05-10 Compositions de protéines effectrices et procédés d'utilisation associés WO2023220654A2 (fr)

Applications Claiming Priority (14)

Application Number Priority Date Filing Date Title
US202263340433P 2022-05-10 2022-05-10
US202263340377P 2022-05-10 2022-05-10
US63/340,377 2022-05-10
US63/340,433 2022-05-10
US202263351714P 2022-06-13 2022-06-13
US63/351,714 2022-06-13
US202263353977P 2022-06-21 2022-06-21
US63/353,977 2022-06-21
US202263380933P 2022-10-25 2022-10-25
US63/380,933 2022-10-25
US202263383845P 2022-11-15 2022-11-15
US63/383,845 2022-11-15
US202363483907P 2023-02-08 2023-02-08
US63/483,907 2023-02-08

Publications (2)

Publication Number Publication Date
WO2023220654A2 true WO2023220654A2 (fr) 2023-11-16
WO2023220654A3 WO2023220654A3 (fr) 2023-12-28

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ID=88731219

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/066848 WO2023220654A2 (fr) 2022-05-10 2023-05-10 Compositions de protéines effectrices et procédés d'utilisation associés

Country Status (1)

Country Link
WO (1) WO2023220654A2 (fr)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022527809A (ja) * 2019-04-03 2022-06-06 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 抗体コード配列をセーフハーバー遺伝子座に挿入するための方法および組成物
CA3178670A1 (fr) * 2020-06-03 2021-12-09 Lucas Benjamin HARRINGTON Nucleases programmables et methodes d'utilisation

Also Published As

Publication number Publication date
WO2023220654A3 (fr) 2023-12-28

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