KR20240142624A - 만능줄기세포에서 분화된 면역저하 베타세포 및 관련 용도 및 방법 - Google Patents

만능줄기세포에서 분화된 면역저하 베타세포 및 관련 용도 및 방법 Download PDF

Info

Publication number
KR20240142624A
KR20240142624A KR1020247026729A KR20247026729A KR20240142624A KR 20240142624 A KR20240142624 A KR 20240142624A KR 1020247026729 A KR1020247026729 A KR 1020247026729A KR 20247026729 A KR20247026729 A KR 20247026729A KR 20240142624 A KR20240142624 A KR 20240142624A
Authority
KR
South Korea
Prior art keywords
modified
cell
molecules
beta
equal
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.)
Pending
Application number
KR1020247026729A
Other languages
English (en)
Korean (ko)
Inventor
소냐 슈레퍼
제프리 알. 밀먼
Original Assignee
사나 바이오테크놀로지, 인크.
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 사나 바이오테크놀로지, 인크. filed Critical 사나 바이오테크놀로지, 인크.
Publication of KR20240142624A publication Critical patent/KR20240142624A/ko
Pending legal-status Critical Current

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
    • 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
    • C12N5/0676Pancreatic cells
    • 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
    • A61K35/37Digestive system
    • A61K35/39Pancreas; Islets of Langerhans
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/70539MHC-molecules, e.g. HLA-molecules
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • 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
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/10Growth factors
    • C12N2501/117Keratinocyte growth factors (KGF-1, i.e. FGF-7; KGF-2, i.e. FGF-12)
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/10Growth factors
    • C12N2501/15Transforming growth factor beta (TGF-β)
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/999Small molecules not provided for elsewhere
    • 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
    • C12N2506/00Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/45Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent 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
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Transplantation (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247026729A 2022-01-10 2023-01-09 만능줄기세포에서 분화된 면역저하 베타세포 및 관련 용도 및 방법 Pending KR20240142624A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202263298214P 2022-01-10 2022-01-10
US63/298,214 2022-01-10
US202263320691P 2022-03-16 2022-03-16
US63/320,691 2022-03-16
US202263322208P 2022-03-21 2022-03-21
US63/322,208 2022-03-21
US202263352605P 2022-06-15 2022-06-15
US63/352,605 2022-06-15
US202263353534P 2022-06-17 2022-06-17
US63/353,534 2022-06-17
PCT/US2023/060341 WO2023133568A2 (en) 2022-01-10 2023-01-09 Hypoimmune beta cells differentiated from pluripotent stem cells and related uses and methods

Publications (1)

Publication Number Publication Date
KR20240142624A true KR20240142624A (ko) 2024-09-30

Family

ID=87074351

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247026729A Pending KR20240142624A (ko) 2022-01-10 2023-01-09 만능줄기세포에서 분화된 면역저하 베타세포 및 관련 용도 및 방법

Country Status (9)

Country Link
US (1) US20250223564A1 (https=)
EP (1) EP4463171A4 (https=)
JP (1) JP2025504392A (https=)
KR (1) KR20240142624A (https=)
AU (1) AU2023204829A1 (https=)
CA (1) CA3246902A1 (https=)
IL (1) IL314029A (https=)
MX (1) MX2024008571A (https=)
WO (1) WO2023133568A2 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024229302A1 (en) * 2023-05-03 2024-11-07 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
IL325954A (en) * 2023-07-28 2026-03-01 Vertex Pharma Cell therapy for diabetes
CN116732099B (zh) * 2023-08-07 2023-11-24 北赛泓升(北京)生物科技有限公司 一种干细胞多重CRISPR/Cas基因组编辑方法
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
WO2025212859A1 (en) * 2024-04-03 2025-10-09 Solid Biosciences, Inc. Methods and constructs for increasing solid organ transplant survival

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE057135T2 (hu) * 2015-09-01 2022-04-28 Ncardia B V In vitro módszer egy humán pluripotens õssejtpopuláció kardiomiocita sejtpopulációvá történõ differenciálására
CN110177869A (zh) * 2017-01-13 2019-08-27 加利福尼亚大学董事会 免疫改造的多能细胞
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
BR112021000637A2 (pt) * 2018-07-17 2021-04-13 The Regents Of The University Of California Célula cardíaca hipoimune isolada, método para tratar um paciente que sofre de uma afecção ou doença cardíaca, método para produzir uma população de células cardíacas hipoimunes, célula endotelial hipoimune isolada, método para tratar um paciente que sofre de uma afecção ou doença vascular, método para produzir uma população de células endoteliais hipoimunes, neurônio dopaminérgico hipoimune isolado, método para tratar um paciente que sofre de uma doença ou afecção neurodegenerativa, método para produzir uma população de neurônios dopaminérgicos hipoimunes, célula de ilhota pancreática hipoimune isolada, método para tratar um paciente que sofre de diabetes, método para produzir uma população de células de ilhota pancreática hipoimunes, célula de epitélio pigmentado da retina (rpe) hipoimune isolada, método para tratar um paciente que sofre de uma afecção ocular, e método para produzir uma população de células de epitélio pigmentado da retina (rpe) hipoimunes
CN115916962A (zh) * 2020-04-27 2023-04-04 萨那生物技术股份有限公司 低免疫原性细胞的重复给药

Also Published As

Publication number Publication date
EP4463171A2 (en) 2024-11-20
WO2023133568A2 (en) 2023-07-13
CA3246902A1 (en) 2023-07-13
EP4463171A4 (en) 2026-03-18
US20250223564A1 (en) 2025-07-10
WO2023133568A3 (en) 2023-08-10
MX2024008571A (es) 2024-09-19
JP2025504392A (ja) 2025-02-12
IL314029A (en) 2024-08-01
AU2023204829A1 (en) 2024-07-11

Similar Documents

Publication Publication Date Title
US20250223564A1 (en) Hypoimmune beta cells differentiated from pluripotent stem cells and related uses and methods
CN105208866B (zh) 使用工程化锌指蛋白核酸酶靶向断裂t细胞受体基因
KR20230074718A (ko) 저면역원성 세포로 감작된 환자를 치료하는 방법 및 관련 방법 및 조성물
JP2022543112A (ja) Dux4発現細胞およびそれらの使用
US20240425820A1 (en) Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
CA3227613A1 (en) Inducible systems for altering gene expression in hypoimmunogenic cells
US20250127820A1 (en) Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
JP2024501971A (ja) Car-t活性を調節するための方法及び組成物
US20240010988A1 (en) Genetically modified primary cells for allogeneic cell therapy
US20240358761A1 (en) Genetically modified cells for allogeneic cell therapy
US20250101382A1 (en) Genetically modified cells and compositions and uses thereof
CN118043344A (zh) Y染色体连锁抗原在低免疫原性细胞中表达的改变
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
CN118871590A (zh) 从多能干细胞分化而来的低免疫β细胞以及相关用途和方法
WO2024151541A1 (en) Type-1 diabetes autoimmune mouse
CN121604968A (zh) 递送胰岛细胞的方法及相关方法
US20260048086A1 (en) Methods of dosing and administration of engineered islet cells
CN118434844A (zh) 用于减少补体介导的炎症反应的同种异体细胞疗法的基因修饰的细胞
CN118382693A (zh) 降低即刻经血液介导的炎性反应的同种异体细胞疗法的遗传修饰细胞
CN118401653A (zh) 用于同种异体细胞疗法的遗传修饰细胞

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

R18 Changes to party contact information recorded

Free format text: ST27 STATUS EVENT CODE: A-3-3-R10-R18-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000