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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：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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公开(公告)号：US20210002433A1
公开(公告)日：2021-01-07
申请号：US16977735
申请日：2019-03-01
摘要： Described herein are compositions and methods for preparing hydrogel capsules using a cross-linking solution comprising a process additive. The process additive improves the quality of the resulting hydrogel capsules, such as increasing the number of defect-free capsules.
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