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Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease - Gene Therapy

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Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease - Gene Therapy

Fabry disease is an X-linked lysosomal storage disorder caused by loss of alpha-galactosidase A (α-Gal A) activity and is characterized by progressive accumulation of glycosphingolipids in multiple cells and tissues. FLT190, an investigational gene therapy, is currently being evaluated in a Phase 1/2 clinical trial in patients with Fabry disease (NCT04040049).

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Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease - Gene Therapy

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Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease - Gene Therapy

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