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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
Applicant: SIGILON THERAPEUTICS, INC.
Inventor: 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
Abstract: 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.