Wolfgang Miesbach: New Editorial Highlights – 6 Year Outcomes of Hemophilia B Gene Therapy
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about a recent article he and his colleague co-authored, published in Blood Advances, adding:
“Happy to share that our editorial is now online in Blood Advances.
We discuss the 6‑year follow‑up of a phase 1/2 study in 14 adults with severe or moderately severe haemophilia B who received a single IV infusion of fidanacogene elaparvovec (5 × 10¹¹ vg/kg), reporting durable FIX levels in the mild range at year 6, sustained bleed reduction, and clinically meaningful improvements in patient‑reported quality of life.
We also highlight safety and methodological nuances, including ALT elevations in 8/14 participants during years 2–6, hepatic steatosis in those with elevated baseline BMI, interindividual variability in FIX expression, and the implications of Pfizer’s withdrawal of Beqvez despite these encouraging long‑term outcomes.
Many thanks to my co‑author Roland W Herzog for this excellent collaboration.”
Title: Six-year FIX-padua gene therapy in hemophilia B
Authors: Wolfgang Miesbach, Roland Herzog
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