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Preclinical development of PR006, a gene therapy for the treatment of frontotemporal dementia with progranulin mutations

Frontotemporal dementia (FTD) caused by GRN haploinsufficiency (FTD‐GRN) represents 5‐10% of all FTD cases. GRN encodes progranulin, a glycoprotein that is essential for normal lysosomal function and neuronal survival. FTD‐GRN patients exhibit approximately 50‐70% reduction in progranulin levels. We are developing PR006, an AAV vector designed to deliver the GRN gene, for FTD‐GRN patients with a goal of slowing or stopping disease progression. We aimed to evaluate the efficacy of PR006 in preclinical models of FTD‐GRN.