Gene Transfer Therapy Updates

Yesterday, 10-28-25, we reported BioMarin’s press release announcing divestiture of their hemophilia A gene transfer therapy, valoctocogene roxaparvovec (Roctavian®). Previously, on February 25, 2025, Pfizer announced their intention to discontinue hemophilia B gene transfer therapy, fidanacogene elaparvovec-dzkt (Becvez®). Press releases from both indicate underperformance in sales. The remaining hemophilia B therapy, etranacogene dezaparvovec-drlb (HEMGENIX®), CSL Behring, is featured in a success story featuring Brian O’Mahoney, CEO of the Irish Haemophilia Society. We look forward to a time when gene transfer therapy becomes a standard, well-tolerated therapeutic approach. A second shout-out to Dean Willett, Precision BioLogic Inc., for the Brian O’Mahoney story.