SG11202108346WA - Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence - Google Patents

Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence

Info

Publication number
SG11202108346WA
SG11202108346WA SG11202108346WA SG11202108346WA SG11202108346WA SG 11202108346W A SG11202108346W A SG 11202108346WA SG 11202108346W A SG11202108346W A SG 11202108346WA SG 11202108346W A SG11202108346W A SG 11202108346WA SG 11202108346W A SG11202108346W A SG 11202108346WA
Authority
SG
Singapore
Prior art keywords
nucleobase
modifying
target sequence
immune cells
adenosine deaminase
Prior art date
Application number
SG11202108346WA
Other languages
English (en)
Inventor
Nicole Gaudelli
Michael Packer
Ian Slaymaker
Yi Yu
Bernd Zetsche
David A Born
Seung-Joo Lee
Jason M Gehrke
Original Assignee
Beam 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 Beam Therapeutics Inc filed Critical Beam Therapeutics Inc
Publication of SG11202108346WA publication Critical patent/SG11202108346WA/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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2510/00—Genetically modified cells
SG11202108346WA 2019-02-13 2020-02-13 Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence SG11202108346WA (en)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US201962931722P 2019-11-06 2019-11-06
US201962941523P 2019-11-27 2019-11-27
US201962941569P 2019-11-27 2019-11-27
US202062966526P 2020-01-27 2020-01-27
PCT/US2020/018178 WO2020168122A1 (fr) 2019-02-13 2020-02-13 Cellules immunitaires modifiées ayant des éditeurs de base d'adénosine désaminase pour modifier une nucléobase dans une séquence cible

Publications (1)

Publication Number Publication Date
SG11202108346WA true SG11202108346WA (en) 2021-08-30

Family

ID=72043867

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202108346WA SG11202108346WA (en) 2019-02-13 2020-02-13 Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence

Country Status (9)

Country Link
US (1) US20230080198A1 (fr)
EP (1) EP3924480A4 (fr)
JP (1) JP2022520233A (fr)
KR (1) KR20210138603A (fr)
CN (1) CN114026227A (fr)
AU (1) AU2020221279A1 (fr)
CA (1) CA3129157A1 (fr)
SG (1) SG11202108346WA (fr)
WO (1) WO2020168122A1 (fr)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018229351B2 (en) 2017-02-28 2024-01-04 Vor Biopharma, Inc. Compositions and methods for inhibition of lineage specific proteins
EP4257696A3 (fr) 2018-03-14 2024-01-24 Arbor Biotechnologies, Inc. Nouveaux systèmes et enzymes de ciblage d'adn crispr
US11389485B2 (en) 2018-08-28 2022-07-19 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
CN114787352A (zh) * 2019-08-28 2022-07-22 Vor生物制药股份有限公司 用于cd123修饰的组合物和方法
US20220380776A1 (en) * 2019-10-22 2022-12-01 Fred Hutchinson Cancer Center Base editor-mediated cd33 reduction to selectively protect therapeutic cells
US20230235315A1 (en) * 2020-07-10 2023-07-27 Horizon Discovery Limited Method for producing genetically modified cells
JP2023540277A (ja) * 2020-08-28 2023-09-22 ブイオーアール バイオファーマ インコーポレーテッド Ｃｄ１２３改変のための組成物および方法
WO2023034408A1 (fr) * 2021-08-31 2023-03-09 The Texas A&M University System Plateforme de thérapie par lymphocyte t de récepteur antigénique chimérique (car)
WO2023034475A1 (fr) * 2021-09-01 2023-03-09 Arbor Biotechnologies, Inc. Cellules modifiées par un polypeptide cas12i
WO2023088440A1 (fr) * 2021-11-18 2023-05-25 Correctsequence Therapeutics Régénération de cellules à antigène négatif de surface
CN114634923B (zh) * 2022-04-07 2024-02-23 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
CN116590237B (zh) * 2023-05-29 2023-10-31 上海贝斯昂科生物科技有限公司 一种遗传修饰的自然杀伤细胞及其制备和用途

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2782125T3 (es) * 2014-03-11 2020-09-10 Cellectis Método para generar linfocitos T compatibles para trasplante alogénico
EP3365357B1 (fr) * 2015-10-23 2024-02-14 President and Fellows of Harvard College Protéines cas9 évoluées pour l'édition génétique
AU2017306676B2 (en) * 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
SG11201903089RA (en) * 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
AU2018229351B2 (en) * 2017-02-28 2024-01-04 Vor Biopharma, Inc. Compositions and methods for inhibition of lineage specific proteins
AU2018270088A1 (en) * 2017-05-18 2020-01-16 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
JP2020534795A (ja) * 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物

Also Published As

Publication number Publication date
JP2022520233A (ja) 2022-03-29
KR20210138603A (ko) 2021-11-19
WO2020168122A1 (fr) 2020-08-20
EP3924480A1 (fr) 2021-12-22
US20230080198A1 (en) 2023-03-16
AU2020221279A1 (en) 2021-08-26
CN114026227A (zh) 2022-02-08
CA3129157A1 (fr) 2020-08-20
EP3924480A4 (fr) 2023-08-02

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