CA3164591A1 - Modification of blood type antigens - Google Patents

Modification of blood type antigens Download PDF

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Publication number
CA3164591A1
CA3164591A1 CA3164591A CA3164591A CA3164591A1 CA 3164591 A1 CA3164591 A1 CA 3164591A1 CA 3164591 A CA3164591 A CA 3164591A CA 3164591 A CA3164591 A CA 3164591A CA 3164591 A1 CA3164591 A1 CA 3164591A1
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Canada
Prior art keywords
cell
gene
seq
guide rna
exon
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Pending
Application number
CA3164591A
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English (en)
French (fr)
Inventor
William Dowdle
Andrew May
Sonja SCHREPFER
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Sana Biotechnology Inc
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Individual
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Publication of CA3164591A1 publication Critical patent/CA3164591A1/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
    • 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
    • C12N15/1137—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 enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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 [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01037—Fucosylgalactoside 3-alpha-galactosyltransferase (2.4.1.37)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/0104—Glycoprotein-fucosylgalactoside alpha-N-acetylgalactosaminyltransferase (2.4.1.40)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01069—Galactoside 2-alpha-L-fucosyltransferase (2.4.1.69)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2510/00—Genetically modified 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Reproductive Health (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Veterinary Medicine (AREA)
  • Rheumatology (AREA)
  • Cardiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CA3164591A 2020-01-13 2021-01-12 Modification of blood type antigens Pending CA3164591A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062960607P 2020-01-13 2020-01-13
US202062960617P 2020-01-13 2020-01-13
US62/960,617 2020-01-13
US62/960,607 2020-01-13
PCT/US2021/013140 WO2021146222A1 (en) 2020-01-13 2021-01-12 Modification of blood type antigens

Publications (1)

Publication Number Publication Date
CA3164591A1 true CA3164591A1 (en) 2021-07-22

Family

ID=74562041

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3164591A Pending CA3164591A1 (en) 2020-01-13 2021-01-12 Modification of blood type antigens

Country Status (7)

Country Link
US (1) US20230272429A1 (enExample)
EP (1) EP4090743A1 (enExample)
JP (1) JP7853210B2 (enExample)
CN (1) CN116234906A (enExample)
AU (1) AU2021207810A1 (enExample)
CA (1) CA3164591A1 (enExample)
WO (1) WO2021146222A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119913243A (zh) * 2025-04-01 2025-05-02 浙江省血液中心 Abo血型变异体的snp位点、b亚型变异型血型鉴定试剂盒和方法

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* Cited by examiner, † Cited by third party
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US11162079B2 (en) 2019-05-10 2021-11-02 The Regents Of The University Of California Blood type O Rh-hypo-immunogenic pluripotent cells
CA3138597A1 (en) 2019-05-10 2020-11-19 The Regents Of The University Of California Modified pluripotent cells
IL308637A (en) * 2021-05-19 2024-01-01 Sana Biotechnology Inc Primary hypoimmunogenic T cells are RHD negative
AU2022325955A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
JP2024534771A (ja) 2021-08-11 2024-09-26 サナ バイオテクノロジー，インコーポレイテッド 補体媒介性炎症反応を低減するための同種異系細胞療法のための遺伝子改変細胞
US20250129346A1 (en) * 2021-08-11 2025-04-24 Sana Biotechnology, Inc. Engineering cells for cell-based therapies, and associated compositions and methods
JP2024531234A (ja) 2021-08-11 2024-08-29 サナ バイオテクノロジー，インコーポレイテッド 同種異系細胞療法のための遺伝子改変された初代細胞
WO2023019226A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
WO2025117331A1 (en) 2023-12-01 2025-06-05 Eli Lilly And Company Methods of making stem cell-derived islet-like cells, as well as populations and compositions including the same
CN118291605A (zh) * 2024-04-10 2024-07-05 深圳市血液中心(深圳市输血医学研究所) 一种基于ngs的多系统红细胞血型基因分型方法和试剂盒
CN118638907A (zh) * 2024-06-26 2024-09-13 中国人民解放军联勤保障部队第九六〇医院 一种用于检测易漏检类孟买表型的snp标记

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EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
CN114634950A (zh) 2012-12-12 2022-06-17 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
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CA2988854A1 (en) 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods
US20190330601A1 (en) * 2016-05-13 2019-10-31 University Of Copenhagen A cell-based array platform
KR20190103373A (ko) 2017-01-13 2019-09-04 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 면역조작된 만능 세포
WO2018227286A1 (en) 2017-06-12 2018-12-20 Sinai Health System Allograft tolerance without the need for systemic immune suppression
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US20210308183A1 (en) * 2018-07-17 2021-10-07 The Regents Of The University Of California Chimeric antigen receptor t cells derived from immunoengineered pluripotent stem cells
CN119372151A (zh) * 2018-07-17 2025-01-28 加利福尼亚大学董事会 从免疫工程化多能细胞分化的细胞
CA3138597A1 (en) * 2019-05-10 2020-11-19 The Regents Of The University Of California Modified pluripotent cells
US11162079B2 (en) * 2019-05-10 2021-11-02 The Regents Of The University Of California Blood type O Rh-hypo-immunogenic pluripotent cells

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119913243A (zh) * 2025-04-01 2025-05-02 浙江省血液中心 Abo血型变异体的snp位点、b亚型变异型血型鉴定试剂盒和方法

Also Published As

Publication number Publication date
JP7853210B2 (ja) 2026-04-28
WO2021146222A1 (en) 2021-07-22
CN116234906A (zh) 2023-06-06
AU2021207810A1 (en) 2022-08-04
EP4090743A1 (en) 2022-11-23
US20230272429A1 (en) 2023-08-31
JP2023510872A (ja) 2023-03-15

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