TW202241472A - 通用供體細胞 - Google Patents

通用供體細胞 Download PDF

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TW202241472A
TW202241472A TW111100145A TW111100145A TW202241472A TW 202241472 A TW202241472 A TW 202241472A TW 111100145 A TW111100145 A TW 111100145A TW 111100145 A TW111100145 A TW 111100145A TW 202241472 A TW202241472 A TW 202241472A
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locus
nucleotide sequence
nucleic acid
encoding
vitro method
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TW111100145A
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Chinese (zh)
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阿里瑞薩 里薩尼亞
瓦倫廷 史拉許
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瑞士商克里斯珀醫療股份公司
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TW111100145A 2020-12-31 2022-01-03 通用供體細胞 TW202241472A (zh)

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US202063132890P 2020-12-31 2020-12-31
US63/132,890 2020-12-31
US202163234997P 2021-08-19 2021-08-19
US63/234,997 2021-08-19
US202163288356P 2021-12-10 2021-12-10
US63/288,356 2021-12-10

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AU (2) AU2021414617A1 (enExample)
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US12378572B2 (en) 2018-09-07 2025-08-05 Crispr Therapeutics Ag Universal donor cells
US11116798B2 (en) 2019-09-05 2021-09-14 Crispr Therapeutics Ag Universal donor cells
US11566230B2 (en) 2020-12-31 2023-01-31 Crispr Therapeutics Ag Universal donor cells
CN116179495B (zh) * 2022-11-28 2025-06-06 上海恩凯细胞技术有限公司 转基因免疫细胞及其应用
WO2025202874A1 (en) 2024-03-25 2025-10-02 Crispr Therapeutics Ag Genetically modified cells expressing glp-1 receptor agonist
WO2025259836A1 (en) * 2024-06-13 2025-12-18 Beth Israel Deaconess Medical Center, Inc. Therapy for ocular neovascularization
WO2026069163A1 (en) 2024-09-25 2026-04-02 Crispr Therapeutics Ag Differentiation method for producing immature beta cells

Family Cites Families (114)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1724052A (en) 1928-06-13 1929-08-13 Luther S Swearengin Fire-door construction
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5283058A (en) 1990-08-30 1994-02-01 The General Hospital Corporation Methods for inhibiting rejection of transplanted tissue
EP0555428A1 (en) 1991-07-30 1993-08-18 Baxter International Inc. Foramenous implant
US7101543B2 (en) 2000-03-31 2006-09-05 Novarx Genetically modified cells expressing a TGFβ inhibitor, the cells being lung cancer cells
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
US20060222633A1 (en) 2000-05-11 2006-10-05 Yale University Prevention, decrease, and/or treatment of immunoreactivity by depleting and/or inactivating antigen presenting cells in the host
US6635750B1 (en) 2000-07-20 2003-10-21 Millennium Pharmaceuticals, Inc. B7-H2 nucleic acids, members of the B7 family
US7432104B2 (en) 2001-08-06 2008-10-07 Bresgen Inc. Methods for the culture of human embryonic stem cells on human feeder cells
EP2722387B1 (en) 2003-12-23 2019-12-11 Viacyte, Inc. Definitive endoderm
US8586357B2 (en) 2003-12-23 2013-11-19 Viacyte, Inc. Markers of definitive endoderm
US20050266554A1 (en) 2004-04-27 2005-12-01 D Amour Kevin A PDX1 expressing endoderm
US7985585B2 (en) 2004-07-09 2011-07-26 Viacyte, Inc. Preprimitive streak and mesendoderm cells
US7541185B2 (en) 2003-12-23 2009-06-02 Cythera, Inc. Methods for identifying factors for differentiating definitive endoderm
US8187878B2 (en) 2004-08-13 2012-05-29 University Of Georgia Research Foundation, Inc. Methods for increasing definitive endoderm differentiation of pluripotent human embryonic stem cells with PI-3 kinase inhibitors
EP2110667A1 (en) 2005-01-20 2009-10-21 University Of Rochester Thoredoxin interacting protein (TXNIP) as regulator of vascular function
ES2687233T3 (es) 2005-10-27 2018-10-24 Viacyte, Inc. Endodermo de intestino proximal dorsal y ventral que expresa PDX-1
EP2420565B1 (en) 2006-02-23 2017-08-30 ViaCyte, Inc. Compositions and methods useful for culturing differentiable cells
NZ571427A (en) 2006-03-02 2012-07-27 Viacyte Inc Endocrine precursor cells, pancreatic hormone-expressing cells and methods of production
US7695965B2 (en) 2006-03-02 2010-04-13 Cythera, Inc. Methods of producing pancreatic hormones
WO2007139929A2 (en) 2006-05-25 2007-12-06 The Burnham Institute For Medical Research Methods for culture and production of single cell populations of human embryonic stem cells
RU2473685C2 (ru) 2007-07-31 2013-01-27 Лайфскен, Инк. Дифференцировка человеческих эмбриональных стволовых клеток
US7695963B2 (en) 2007-09-24 2010-04-13 Cythera, Inc. Methods for increasing definitive endoderm production
US8623650B2 (en) 2007-10-19 2014-01-07 Viacyte, Inc. Methods and compositions for feeder-free pluripotent stem cell media containing human serum
CN107574142B (zh) 2007-11-27 2021-07-06 生命扫描有限公司 人胚胎干细胞的分化
US8338170B2 (en) 2008-04-21 2012-12-25 Viacyte, Inc. Methods for purifying endoderm and pancreatic endoderm cells derived from human embryonic stem cells
US7939322B2 (en) 2008-04-24 2011-05-10 Centocor Ortho Biotech Inc. Cells expressing pluripotency markers and expressing markers characteristic of the definitive endoderm
US20120058105A1 (en) 2008-06-27 2012-03-08 Martin Kean Chong Ng Method of treatment of vascular complications
EP2346988B1 (en) 2008-10-31 2017-05-31 Janssen Biotech, Inc. Differentiation of human embryonic stem cells to the pancreatic endocrine lineage
JP2012507289A (ja) 2008-10-31 2012-03-29 ヤンセン バイオテツク，インコーポレーテツド ヒト胚性幹細胞の膵内分泌系への分化
US8008075B2 (en) 2008-11-04 2011-08-30 Viacyte, Inc. Stem cell aggregate suspension compositions and methods of differentiation thereof
US8895300B2 (en) 2008-11-04 2014-11-25 Viacyte, Inc. Scalable primate pluripotent stem cell aggregate suspension culture and differentiation thereof
EP2356227B1 (en) 2008-11-14 2018-03-28 Viacyte, Inc. Encapsulation of pancreatic cells derived from human pluripotent stem cells
US20100272710A1 (en) 2009-03-26 2010-10-28 Abdelhadi Rebbaa Compositions and methods for treating and preventing aging-associated diseases
ES2849181T3 (es) 2009-04-22 2021-08-16 Viacyte Inc Composiciones celulares derivadas de células reprogramadas desdiferenciadas
US9109245B2 (en) 2009-04-22 2015-08-18 Viacyte, Inc. Cell compositions derived from dedifferentiated reprogrammed cells
WO2010129294A2 (en) 2009-04-27 2010-11-11 Schulz Thomas C Small molecules supporting pluripotent cell growth and methods thereof
EP2456858B1 (en) 2009-07-20 2018-08-29 Janssen Biotech, Inc. Differentiation of human embryonic stem cells
RU2540016C2 (ru) 2009-07-20 2015-01-27 Янссен Байотек, Инк. Дифференцировка эмбриональных стволовых клеток человека
RU2664864C1 (ru) 2009-12-23 2018-08-23 Янссен Байотек, Инк. Способы увеличения экспрессии ngn3 и nkx6.1 в эндокринных клетках поджелудочной железы
DK2516625T3 (da) 2009-12-23 2024-09-09 Janssen Biotech Inc Differentiering af humane embryonale stamceller
KR101836850B1 (ko) 2010-08-31 2018-03-09 얀센 바이오테크 인코포레이티드 인간 배아 줄기 세포의 분화
WO2012030540A2 (en) 2010-08-31 2012-03-08 Janssen Biotech, Inc. Differentiation of pluripotent stem cells
JP5875597B2 (ja) 2010-12-08 2016-03-02 ヴィアサイト，インコーポレイテッド ヒト万能性幹細胞の成長を阻害する薬剤および方法
DK2699593T4 (da) 2011-04-20 2021-02-08 Univ Washington Through Its Center For Commercialization Beta-2 mikroglobulin-deficiente celler
USD726306S1 (en) 2011-12-12 2015-04-07 Viacyte, Inc. Cell encapsulation device
USD728095S1 (en) 2011-12-12 2015-04-28 Viacyte, Inc. Cell encapsulation device
USD726307S1 (en) 2011-12-12 2015-04-07 Viacyte Inc. Cell encapsulation device
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
RU2705001C2 (ru) 2011-12-22 2019-11-01 Янссен Байотек, Инк. Дифференцировка эмбриональных стволовых клеток человека в одногормональные инсулинположительные клетки
DK2657340T3 (en) 2012-04-25 2017-02-27 Deutsches Krebsforsch Thioredoxin-interacting protein (TXNIP) inhibitors for therapy
US20140162359A1 (en) 2012-05-07 2014-06-12 Janssen Biotech, Inc. Differentiation of Human Embryonic Stem Cells into Pancreatic Endoderm
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
USD734847S1 (en) 2012-05-31 2015-07-21 Viacyte, Inc. Cell encapsulation device
CN104334719B (zh) 2012-06-08 2018-02-13 詹森生物科技公司 人胚胎干细胞向胰腺内分泌细胞的分化
RU2015100900A (ru) 2012-06-14 2016-08-10 Янссен Байотек, Инк. Обработка плюрипотентных клеток
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
US10370644B2 (en) 2012-12-31 2019-08-06 Janssen Biotech, Inc. Method for making human pluripotent suspension cultures and cells derived therefrom
DK2938722T3 (da) 2012-12-31 2022-02-14 Janssen Biotech Inc Suspension og klyngedannelse af humane pluripotente celler til differentiering til endokrine pankreasceller
MX2015008578A (es) 2012-12-31 2015-09-07 Janssen Biotech Inc Diferenciacion de celulas madre embrionarias humanas en celulas endocrinas pancreaticas mediante el uso de relugadores de hb9.
KR102084561B1 (ko) 2012-12-31 2020-03-04 얀센 바이오테크 인코포레이티드 췌장 내분비 세포로의 분화를 위한 공기-액체 계면에서의 인간 배아 줄기세포의 배양
USD720469S1 (en) 2013-03-07 2014-12-30 Viacyte, Inc. Cell encapsulation device
JP6234485B2 (ja) 2013-03-07 2017-11-22 ヴィアサイト インコーポレイテッド ３次元大収容力細胞封入デバイスアセンブリー
US8859286B2 (en) 2013-03-14 2014-10-14 Viacyte, Inc. In vitro differentiation of pluripotent stem cells to pancreatic endoderm cells (PEC) and endocrine cells
US9101597B2 (en) 2013-03-14 2015-08-11 The Administration Of The Tulane Educational Fund Immunoprotective primary mesenchymal stem cells and methods
WO2014195159A1 (en) 2013-06-03 2014-12-11 Theravectys Lentiviral vectors containing an mhc class i, mhc class ii, or beta-2 microglobulin upstream promoter sequence
AU2014278178B2 (en) 2013-06-11 2021-01-21 President And Fellows Of Harvard College SC-beta cells and compositions and methods for generating the same
KR20140144964A (ko) 2013-06-12 2014-12-22 한국생명공학연구원 티오레독신-결합 단백질을 유효성분으로 포함하는 약학적 조성물 및 이의 용도
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
RU2689710C2 (ru) 2013-11-01 2019-05-29 Янссен Байотек, Инк. Суспендирование и кластеризация плюрипотентных стволовых клеток человека с целью их дифференцировки в панкреатические эндокринные клетки
USD718472S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718469S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718471S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718466S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-Dimensional large capacity cell encapsulation device
USD718467S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718468S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718470S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD714956S1 (en) 2014-03-07 2014-10-07 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
RU2752933C2 (ru) 2014-03-11 2021-08-11 Селлектис Способ создания т-клеток, пригодных для аллогенной трансплантации
EP3122370A1 (en) 2014-03-28 2017-02-01 Buck Institute for Research on Aging Methods and compositions for modulating the immune system
USD761424S1 (en) 2014-04-16 2016-07-12 Viacyte, Inc. Deployment tool for an encapsulation device
USD761423S1 (en) 2014-04-16 2016-07-12 Viacyte, Inc. Sizing tool for an encapsulation device
USD760399S1 (en) 2014-04-16 2016-06-28 Viacyte, Inc. Case for an encapsulation device
USD734166S1 (en) 2014-04-16 2015-07-14 Viacyte, Inc. Fill pouch assembly for encapsulation device
SG11201609473XA (en) 2014-05-16 2016-12-29 Janssen Biotech Inc Use of small molecules to enhance mafa expression in pancreatic endocrine cells
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
CN107250349A (zh) 2014-12-19 2017-10-13 詹森生物科技公司 多能干细胞的悬浮培养
EP3294342A4 (en) * 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
EP4339287A3 (en) 2015-07-31 2024-05-22 Regents Of The University Of Minnesota Modified cells and methods of therapy
US12642852B1 (en) * 2015-11-04 2026-06-02 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells
CA3027879A1 (en) 2016-06-21 2017-12-28 Janssen Biotech, Inc. Generation of human pluripotent stem cell derived functional beta cells showing a glucose-dependent mitochondrial respiration and two-phase insulin secretion response
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
US9982267B2 (en) 2016-10-12 2018-05-29 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
EP4595951A3 (en) 2016-11-10 2025-08-27 ViaCyte, Inc. Pdx1 pancreatic endoderm cells in cell delivery devices
KR20240095477A (ko) 2017-01-13 2024-06-25 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 면역조작된 만능 세포
EP3596117A4 (en) 2017-03-17 2021-01-13 The Johns Hopkins University TARGETED EPIGENETIC THERAPY AGAINST THE DISTAL EXPRESSION REGULATORY ELEMENT OF TGFB2
CN110869494B (zh) 2017-06-12 2025-02-18 西奈卫生系统公司 无需全身性免疫遏制的同种异体移植物耐受
US10391156B2 (en) 2017-07-12 2019-08-27 Viacyte, Inc. University donor cells and related methods
JP7621795B2 (ja) 2017-10-19 2025-01-27 セレクティス 改善された免疫細胞療法のためのｎｋ阻害物質遺伝子の標的指向遺伝子組み込み
CN107723275B (zh) 2017-10-20 2020-09-04 重庆精准生物技术有限公司 通用型car-t细胞及其制备方法和应用
SG11202004529XA (en) 2017-11-16 2020-06-29 Mogam Inst Biomedical Res Transformed human cell and use thereof
CN111556892A (zh) 2017-12-08 2020-08-18 菲特治疗公司 使用增强的iPSC衍生的效应细胞的免疫疗法
US20190223416A1 (en) 2018-01-23 2019-07-25 Cotyledon, LLC Canine genome editing
EP3754018A4 (en) 2018-02-16 2021-11-24 Kyoto University METHOD OF MANUFACTURING A WEAK ANTIGENT CELL
US12378572B2 (en) 2018-09-07 2025-08-05 Crispr Therapeutics Ag Universal donor cells
CA3120576A1 (en) 2018-11-26 2020-06-04 Massachusetts Institute Of Technology Compositions and methods for immune tolerance
CA3123102A1 (en) 2018-12-13 2020-06-18 Sinai Health System Immunomodulatory cells and uses thereof
WO2020206055A1 (en) 2019-04-02 2020-10-08 Yale University Methods of treatment of diseases and disorders associated with increased tet level, increased h19 level, increased level of tgf signaling, or any combination thereof
US20220213434A1 (en) * 2019-05-10 2022-07-07 The Regents Of The University Of California Modified pluripotent cells
WO2020228039A1 (en) 2019-05-16 2020-11-19 Egenesis, Inc. Cells, tissues, organs, and/or animals having one or more modified genes for enhanced xenograft survival and/or tolerance
CN110373389A (zh) * 2019-06-10 2019-10-25 云南农业大学 一种适合胰岛细胞及皮肤等组织异种移植供体猪的构建方法
US11116798B2 (en) 2019-09-05 2021-09-14 Crispr Therapeutics Ag Universal donor cells
US11104918B2 (en) 2019-09-05 2021-08-31 Crispr Therapeutics Ag Universal donor cells
US11566230B2 (en) 2020-12-31 2023-01-31 Crispr Therapeutics Ag Universal donor cells

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