Method And Compositions For The Treatment Of Fabry Disease

NZ793215BActive Publication Date: 2026-09-01SANGAMO THERAPEUTICS INC
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Patent Information

Application Number
NZ793215
Authority / Receiving Office
NZ · NZ
Patent Type
Patents
Current Assignee / Owner
Priority Date
2017-08-31
Filing Date
2017-10-19
Publication Date
2026-09-01
Estimated Expiration
2037-10-19

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Abstract

The disclosure provides nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic alpha galactosidase A (alpha-Gal A) protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and / or prevention of Fabry disease. Further provided are compositions for expressing at least one alpha-Gal A protein.
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