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公开(公告)号：US20210145759A1
公开(公告)日：2021-05-20
申请号：US17045127
申请日：2019-03-27
发明人： Lauren Emily Barney , Michael Beauregard , Guillaume Carmona , Francisco Caballero Gonzalez , Richard Heidebrecht , Erika Ellen Johnston , Robert James Miller , Matthias Alexander Oberli , Owen O'Connor , David Peritt , Jared A. Sewell , Devyn McKinley Smith , Omid Veiseh , Paul Kevin Wotton
摘要： Described herein are particles comprising a first compartment, a second compartment, and a compound of Formula (I), as well as compositions and methods of making and using the same. The particles may comprise a cell capable of expressing a therapeutic agent useful for the treatment of a disease, disorder, or condition described herein.
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公开(公告)号：US20200263196A1
公开(公告)日：2020-08-20
申请号：US16651892
申请日：2018-09-27
发明人： Guillaume Carmona , Francisco Caballero Gonzalez , Richard Heidebrecht , Robert James Miller , Matthias Alexander Oberli , David Peritt , Jared A. Sewell , Devyn McKinley Smith , Omid Veiseh , Paul Kevin Wotton
IPC分类号： C12N15/85 , C12N5/079 , A61K35/30 , C07K14/755 , C07K14/55 , C07K14/635 , A61K9/00 , A61K9/48 , A61K47/54
摘要： Described herein are cell compositions comprising an active cell (e.g., an engineered active cell, e.g., an engineered RPE cell) or derivatives thereof, as well as compositions, pharmaceutical products, and implantable elements comprising an active cell, and methods of making and using the same. The cells and compositions may express a therapeutic agent useful for the treatment of a disease, disorder, or condition described herein.
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公开(公告)号：US20230123802A1
公开(公告)日：2023-04-20
申请号：US17598154
申请日：2020-03-27
发明人： Lauren Emily Barney , Michael Beauregard , Guillaume Carmona , Francisco Caballero Gonzalez , Richard Heidebrecht , Erika Ellen Johnston , Robert James Miller , Owen O'Connor , Matthias Alexander Oberli , David Peritt , Jared A. Sewell , Devyn McKinley Smith , Omid Veiseh , Paul Kevin Wotton , Zoe Yin
摘要： Described herein are RPE cells engineered to secrete a FVII protein, as well as compositions, pharmaceutical preparations, and implantable devices comprising the engineered RPE cells, and methods of making and using the same for treating a patient with hemophilia or FVII deficiency.
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公开(公告)号：US20230038379A1
公开(公告)日：2023-02-09
申请号：US17778159
申请日：2020-11-20
摘要： Described herein are genetically modified cells derived from a human cell and which contain at least one exogenous transcription unit inserted into at least one of four open chromatin regions (OCRs) located on Chromosomes 2, 5, 8 and 12, as well as compositions, pharmaceutical preparations, and implantable devices comprising the genetically modified cells, and methods of using the same for preventing or treating a disease, disorder, or condition.
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公开(公告)号：US20220267794A1
公开(公告)日：2022-08-25
申请号：US17598176
申请日：2020-03-27
发明人： Lauren Emily Barney , Michael Beauregard , Guillaume Carmona , Francisco Caballero Gonzalez , Richard Heidebrecht , Erika Ellen Johnston , Robert James Miller , Owen O'Connor , Matthias Alexander Oberli , David Peritt , Jared A. Sewell , Devyn McKinley Smith , Omid Veiseh , Jeffrey Charles Way , Paul Kevin Wotton , Zoe Yin , Elina Makino , Brian Richard Fluharty , Marianthi Papakosta
摘要： Described herein are RPE cells engineered to secrete a GLA protein, as well as compositions, pharmaceutical preparations, and implantable devices comprising the engineered RPE cells, and methods of making and using the same for treating Fabry disease.
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公开(公告)号：US20220000789A1
公开(公告)日：2022-01-06
申请号：US17280802
申请日：2019-09-27
发明人： Lauren Emily Barney , Michael Beauregard , Guillaume Carmona , Francisco Caballero Gonzalez , Richard Heidebrecht , Erika Ellen Johnston , Robert James Miller , Owen O'Connor , Matthias Alexander Oberli , David Peritt , Jared A. Sewell , Devyn McKinley Smith , Omid Veiseh , Paul Kevin Wotton , Zoe Yin
摘要： Described herein are implantable devices comprising means for mitigating the foreign body response (FBR) and at least one cell-containing compartment which comprises a plurality of cells (e.g., live cells) encapsulated in a polymer composition comprising a cell-binding substance (CBS), as well as compositions and methods of making and using the same. The cells are capable of expressing a therapeutic agent useful for the treatment of a disease, disorder, or condition described herein.
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