COMPOSITIONS, DEVICES AND METHODS FOR TREATING FABRY DISEASE

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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Bibliographic Details
Main Authors BARNEY, Lauren, Emily, CARMONA, Guillaume, WOTTON, Paul, Kevin, JOHNSTON, Erika, Ellen, WAY, Jeffrey, Charles, YIN, Zoe, HEIDEBRECHT, Richard, SMITH, Devyn, McKinley, VEISEH, Omid, PAPAKOSTA, Marianthi, MAKINO, Elina, O'CONNOR, Owen, OBERLI, Matthias, Alexander, MILLER, Robert, James, BEAUREGARD, Michael, GONZALEZ, Francisco, Caballero, PERITT, David, SEWELL, Jared, A, FLUHARTY, Brian Richard
Format Patent
LanguageEnglish
French
German
Published 09.02.2022
Subjects
CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS, ORSURGICAL ARTICLES
DISINFECTION, STERILISATION, OR DEODORISATION OF AIR
HUMAN NECESSITIES
HYGIENE
MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICALARTICLES
MEDICAL OR VETERINARY SCIENCE
METHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS INGENERAL
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary: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.
Bibliography:Application Number: EP20200779673