JP7853210B2 - 血液型抗原の修飾 - Google Patents

血液型抗原の修飾

Info

Publication number
JP7853210B2
JP7853210B2 JP2022542899A JP2022542899A JP7853210B2 JP 7853210 B2 JP7853210 B2 JP 7853210B2 JP 2022542899 A JP2022542899 A JP 2022542899A JP 2022542899 A JP2022542899 A JP 2022542899A JP 7853210 B2 JP7853210 B2 JP 7853210B2
Authority
JP
Japan
Prior art keywords
cells
gene
rhd
target sequences
abo
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.)
Active
Application number
JP2022542899A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023510872A (ja
JP2023510872A5 (https=
Inventor
ドゥードル,ウィリアム
メイ,アンドリュー
シュレプファー,ソーニャ
Original Assignee
サナ バイオテクノロジー,インコーポレイテッド
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 サナ バイオテクノロジー,インコーポレイテッド filed Critical サナ バイオテクノロジー,インコーポレイテッド
Publication of JP2023510872A publication Critical patent/JP2023510872A/ja
Publication of JP2023510872A5 publication Critical patent/JP2023510872A5/ja
Application granted granted Critical
Publication of JP7853210B2 publication Critical patent/JP7853210B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/48Reproductive organs
    • A61K35/54Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0652Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657Cardiomyocytes; Heart cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/01Hexosyltransferases (2.4.1)
    • C12Y204/01037Fucosylgalactoside 3-alpha-galactosyltransferase (2.4.1.37)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/01Hexosyltransferases (2.4.1)
    • C12Y204/0104Glycoprotein-fucosylgalactoside alpha-N-acetylgalactosaminyltransferase (2.4.1.40)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/01Hexosyltransferases (2.4.1)
    • C12Y204/01069Galactoside 2-alpha-L-fucosyltransferase (2.4.1.69)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Medicinal Chemistry (AREA)
  • Reproductive Health (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (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)
JP2022542899A 2020-01-13 2021-01-12 血液型抗原の修飾 Active JP7853210B2 (ja)

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
US62/960,607 2020-01-13
US62/960,617 2020-01-13
PCT/US2021/013140 WO2021146222A1 (en) 2020-01-13 2021-01-12 Modification of blood type antigens

Publications (3)

Publication Number Publication Date
JP2023510872A JP2023510872A (ja) 2023-03-15
JP2023510872A5 JP2023510872A5 (https=) 2024-01-22
JP7853210B2 true JP7853210B2 (ja) 2026-04-28

Family

ID=74562041

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022542899A Active JP7853210B2 (ja) 2020-01-13 2021-01-12 血液型抗原の修飾

Country Status (7)

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

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11162079B2 (en) 2019-05-10 2021-11-02 The Regents Of The University Of California Blood type O Rh-hypo-immunogenic pluripotent cells
US12221622B2 (en) 2019-05-10 2025-02-11 The Regents Of The University Of California Modified pluripotent cells
BR112023024231A2 (pt) * 2021-05-19 2024-01-30 Sana Biotechnology Inc Células t primárias rhd negativas hipoimunogênicas
US20240358761A1 (en) 2021-08-11 2024-10-31 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
JP2024535677A (ja) 2021-08-11 2024-10-02 サナ バイオテクノロジー,インコーポレイテッド 即時血液媒介性炎症反応を減少させるための同種細胞療法を目的とした遺伝子改変細胞
EP4384598A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
US20250129346A1 (en) * 2021-08-11 2025-04-24 Sana Biotechnology, Inc. Engineering cells for cell-based therapies, and associated compositions and methods
AU2022325232A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified primary 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
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018515139A (ja) 2015-05-08 2018-06-14 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 万能ドナー幹細胞および関連する方法
WO2018132783A1 (en) 2017-01-13 2018-07-19 The Regents Of The University Of California Immunoengineered pluripotent cells
WO2019084531A1 (en) 2017-10-27 2019-05-02 The Children's Hospital Of Philadelphia MODIFIED RED GLOBES HAVING PHENOTYPES OF RARE ANTIGENS

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8724885D0 (en) 1987-10-23 1987-11-25 Binns M M Fowlpox virus promotors
DE19539493A1 (de) 1995-10-24 1997-04-30 Thomae Gmbh Dr K Starker homologer Promotor aus Hamster
CA3111953C (en) 2011-04-05 2023-10-24 Cellectis Method for the generation of compact tale-nucleases and uses thereof
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2017194699A1 (en) * 2016-05-13 2017-11-16 University Of Copenhagen A cell-based array platform
IL270835B2 (en) 2017-06-12 2025-01-01 Sinai Health Sys Allograft tolerance without the need for systemic immunosuppression
WO2020018620A1 (en) * 2018-07-17 2020-01-23 The Regents Of The University Of California Chimeric antigen receptor t cells derived from immunoengineered pluripotent stem cells
CA3109078A1 (en) * 2018-07-17 2020-01-23 The Regents Of The University Of California Cells differentiated from immunoengineered 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
US12221622B2 (en) * 2019-05-10 2025-02-11 The Regents Of The University Of California Modified pluripotent cells

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018515139A (ja) 2015-05-08 2018-06-14 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 万能ドナー幹細胞および関連する方法
WO2018132783A1 (en) 2017-01-13 2018-07-19 The Regents Of The University Of California Immunoengineered pluripotent cells
WO2019084531A1 (en) 2017-10-27 2019-05-02 The Children's Hospital Of Philadelphia MODIFIED RED GLOBES HAVING PHENOTYPES OF RARE ANTIGENS

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
EMBO Molecular Medicine,2018年,10,e8454
NATURE,1990年,Vol.345,p.229-233

Also Published As

Publication number Publication date
US20230272429A1 (en) 2023-08-31
JP2023510872A (ja) 2023-03-15
CN116234906A (zh) 2023-06-06
WO2021146222A1 (en) 2021-07-22
EP4090743A1 (en) 2022-11-23
CA3164591A1 (en) 2021-07-22
AU2021207810A1 (en) 2022-08-04

Similar Documents

Publication Publication Date Title
JP7853210B2 (ja) 血液型抗原の修飾
JP7724834B2 (ja) 全身免疫抑制を必要としない同種免疫寛容
Papapetrou et al. Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells
US20240425878A1 (en) Genetically modified msc and therapeutic methods
US20260108610A1 (en) Drug-Resistant Immune Cells and Methods of Use Thereof
CN112673094B (zh) 病毒载体产生
AU2013296564A1 (en) HLA G-modified cells and methods
KR20220082045A (ko) Fc 격리를 통한 이식된 세포 보호
AU2019233929B2 (en) B cells genetically engineered to secrete follistatin and methods of using the same to treat follistatin-related diseases, conditions, disorders and to enhance muscle growth and strength
JP2019517281A (ja) 神経セロイドリポフスチン症の遺伝子治療
KR20230029659A (ko) 저면역원성 세포
CN113316637A (zh) 依靠人工反式激活物的选择
US20260062675A1 (en) SIRPalpha-SILENCED NATURAL KILLER (NK) CELLS
WO2011060534A1 (en) Trim5alpha mutants and uses thereof
US20250101382A1 (en) Genetically modified cells and compositions and uses thereof
EP4090349A2 (en) Transplanted cell protection via inhibition of polymorphonuclear cells
AU2020234518B2 (en) Optimised RAG1 deficient gene therapy
KR20230131816A (ko) 저면역원성 줄기세포, 줄기세포로부터 분화되거나 유래된 저면역원성 세포및 이의 제조방법
CA3250696A1 (en) GENE THERAPY
CN118076362A (zh) 用于改变低免疫原性细胞中的基因表达的诱导型系统
WO2024120427A9 (zh) 一种表达gsn的通用型细胞及其制备方法
WO2024120429A1 (zh) 一种表达stc1的通用型细胞及其制备方法
KR20260032524A (ko) 저면역원성의 범용성 유도만능줄기세포
Gutiérrez-Guerrero Gene editing as an alternative to retroviral vectors for Wiskott-Aldrich syndrome gene therapy
HK40040375A (en) B cells genetically engineered to secrete follistatin and methods of using the same to treat follistatin-related diseases, conditions, disorders and to enhance muscle growth and strength

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240112

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240112

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250206

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250501

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250704

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250806

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251031

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20260130

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260312

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20260325

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20260416