Mechanism of Action Adeno-associated virus-5 (AAV5)-based gene therapy designed to deliver a copy of a gene encoding the Padua variant of human coagulation Factor IX (hFIX598 Padua) Single IV infusion results in cell transduction and increase in circulating Factor IX activity in patients with...
FDA’s request was also acknowledged. To ensure linkage between the FIX expression and potency release assays, the sample to be analyzed for FIX expression will be taken from the cell supernatant of the potency release assay. Due to the cell-based nature of this bioassay, the absolute con...
Mechanism Of Action HEMGENIX is an adeno-associated virus serotype 5 (AAV5) based gene therapy designed to deliver a copy of a gene encoding the Padua variant of human coagulation Factor IX (hFIX-Padua). Single intravenous infusion of HEMGENIX results in cell transduction and increase in circu...