JP2022543112A - Dux4発現細胞およびそれらの使用 - Google Patents
Dux4発現細胞およびそれらの使用 Download PDFInfo
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- JP2022543112A JP2022543112A JP2022506729A JP2022506729A JP2022543112A JP 2022543112 A JP2022543112 A JP 2022543112A JP 2022506729 A JP2022506729 A JP 2022506729A JP 2022506729 A JP2022506729 A JP 2022506729A JP 2022543112 A JP2022543112 A JP 2022543112A
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- cells
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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
- C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
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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
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0646—Natural killers cells [NK], NKT cells
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/15—Natural-killer [NK] cells; Natural-killer T [NKT] cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/17—Monocytes; Macrophages
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- A—HUMAN NECESSITIES
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- A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
- A61K40/24—Antigen-presenting cells [APC]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
- A61K40/41—Vertebrate antigens
- A61K40/42—Cancer antigens
- A61K40/4242—Transcription factors, e.g. SOX or c-MYC
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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
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- C—CHEMISTRY; METALLURGY
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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
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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]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
- A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
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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]
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- C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
- C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem cells
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- C—CHEMISTRY; METALLURGY
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- C12N2510/00—Genetically modified cells
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
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- C12N2800/00—Nucleic acids vectors
- C12N2800/40—Systems of functionally co-operating vectors
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/002—Vectors comprising a special translation-regulating system controllable or inducible
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- Life Sciences & Earth Sciences (AREA)
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- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Cell Biology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Hematology (AREA)
- Developmental Biology & Embryology (AREA)
- Pharmacology & Pharmacy (AREA)
- Mycology (AREA)
- Transplantation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962881840P | 2019-08-01 | 2019-08-01 | |
| US62/881,840 | 2019-08-01 | ||
| PCT/US2020/044635 WO2021022223A1 (en) | 2019-08-01 | 2020-07-31 | Dux4 expressing cells and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022543112A true JP2022543112A (ja) | 2022-10-07 |
| JP2022543112A5 JP2022543112A5 (https=) | 2023-08-08 |
Family
ID=72088403
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022506729A Pending JP2022543112A (ja) | 2019-08-01 | 2020-07-31 | Dux4発現細胞およびそれらの使用 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20220267732A1 (https=) |
| EP (1) | EP4007596A1 (https=) |
| JP (1) | JP2022543112A (https=) |
| WO (1) | WO2021022223A1 (https=) |
Families Citing this family (19)
| 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 |
| US11459372B2 (en) | 2020-11-30 | 2022-10-04 | Crispr Therapeutics Ag | Gene-edited natural killer cells |
| WO2022144632A1 (en) | 2020-12-30 | 2022-07-07 | Crispr Therapeutics Ag | Compositions and methods for differentiating stem cells into nk cells |
| JP2024501971A (ja) | 2020-12-31 | 2024-01-17 | サナ バイオテクノロジー,インコーポレイテッド | Car-t活性を調節するための方法及び組成物 |
| KR20240011831A (ko) * | 2021-05-26 | 2024-01-26 | 후지필름 셀룰러 다이내믹스, 인코포레이티드 | 만능 줄기 세포에서 유전자의 신속한 사일런싱을 방지하기 위한 방법 |
| WO2022269393A1 (en) * | 2021-06-23 | 2022-12-29 | Crispr Therapeutics Ag | Engineered cells with improved protection from natural killer cell killing |
| KR20240046319A (ko) | 2021-07-14 | 2024-04-08 | 사나 바이오테크놀로지, 인크. | 저면역원성 세포에서의 y 염색체-연결 항원의 변경된 발현 |
| AU2022327174A1 (en) | 2021-08-11 | 2024-02-15 | Sana Biotechnology, Inc. | Inducible systems for altering gene expression in hypoimmunogenic cells |
| EP4384598A1 (en) * | 2021-08-11 | 2024-06-19 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions |
| US20250339531A1 (en) * | 2021-11-12 | 2025-11-06 | Fred Hutchinson Cancer Center | Dux4 polypeptides and nucleic acids for treating inflammatory and autoimmune conditions |
| CN119072319A (zh) | 2021-12-23 | 2024-12-03 | 萨那生物技术股份有限公司 | 用于治疗自身免疫性疾病的嵌合抗原受体(car)t细胞及相关方法 |
| US20250302953A1 (en) | 2022-02-14 | 2025-10-02 | Sana Biotechnology, Inc. | Methods of treating patients exhibiting a prior failed therapy with hypoimmunogenic cells |
| IL318041A (en) * | 2022-07-01 | 2025-02-01 | Janssen Biotech Inc | Materials and methods for bioengineered iPSC populations |
| WO2024012420A1 (zh) * | 2022-07-11 | 2024-01-18 | 士泽生物医药(苏州)有限公司 | 一种表达faslg的通用型细胞及其制备方法 |
| WO2024125592A1 (zh) * | 2022-12-16 | 2024-06-20 | 士泽生物医药(苏州)有限公司 | 一种通用型细胞及其制备方法 |
| 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 |
| WO2025230941A1 (en) | 2024-04-30 | 2025-11-06 | Satellite Biosciences, Inc. | Hypoimmunogenic cell mixtures and methods of use thereof |
| WO2025230947A1 (en) | 2024-04-30 | 2025-11-06 | Satellite Biosciences, Inc. | Engineered hepatocytes for secreting polypeptides |
| GB202415025D0 (en) * | 2024-10-11 | 2024-11-27 | Ospedale San Raffaele Srl | Cell |
Citations (2)
| 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 |
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| US5792632A (en) | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| DE19539493A1 (de) | 1995-10-24 | 1997-04-30 | Thomae Gmbh Dr K | Starker homologer Promotor aus Hamster |
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-
2020
- 2020-07-31 US US17/632,026 patent/US20220267732A1/en active Pending
- 2020-07-31 EP EP20757473.2A patent/EP4007596A1/en active Pending
- 2020-07-31 JP JP2022506729A patent/JP2022543112A/ja active Pending
- 2020-07-31 WO PCT/US2020/044635 patent/WO2021022223A1/en not_active Ceased
Patent Citations (2)
| 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 |
Non-Patent Citations (2)
| Title |
|---|
| CHEW, G. L. ET AL: "DUX4 Suppresses MHC Class I to Promote Cancer Immune Evasion and Resistance to Checkpoint Blockade", DEVELOPMENTAL CELL, vol. 50, JPN6024024743, 18 July 2019 (2019-07-18), pages 658 - 671, ISSN: 0005355318 * |
| JAGANNATHAN, S. ET AL: "Model systems of DUX4 expression recapitulate the transcriptional profile of FSHD cells", HUMAN MOLECULAR GENETICS, vol. 25, no. 20, JPN6024024742, 2016, pages 4419 - 4431, ISSN: 0005355319 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021022223A1 (en) | 2021-02-04 |
| EP4007596A1 (en) | 2022-06-08 |
| US20220267732A1 (en) | 2022-08-25 |
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