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Avigbagene parvec

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Engineered glucocerebrosidase gene therapy in a recruiting pivotal Phase 3 study for Gaucher disease type 1.

Modality
liver-directed AAV gene therapy encoding engineered glucocerebrosidase
Availability (US)
Pivotal Phase 3 GALILEO-3 study Recruiting
Biomarkers
glucosylsphingosine; hemoglobin; platelet count; liver and spleen volume; bone-disease measures; adverse events
Hallmark targeted
lysosomal dysfunction, impaired substrate clearance, sphingolipid homeostasis
Kind
investigational gene therapy
Clinical trials
NCT07223944 — Phase 3, Recruiting on September 3, 2026 update; NCT05324943 — Phase 1, Completed on June 2, 2026 update