Wolfgang Miesbach: What the Latest Gene Therapy Setbacks Mean for Haemophilia Care
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“Another setback — and what it means.
Be Biopharma has terminated its Phase 1/2 BeCoMe-9 trial of BE-101, an autologous B-cell therapy for severe haemophilia B, after enrolling just 5 of 24 planned patients – a ‘strategic business decision’ not a safety issue.
BE-101 edited patients’ own B cells ex vivo to produce FIX, with stable activity and re-dosing potential.
It was one of the very few B-cell therapies so far.
This comes on top of:
- Pfizer cutting all funding and global rollout of its approved haemophilia B gene therapy Beqvez, citing limited interest from patients and physicians
- BioMarin withdrawing its haemophilia A gene therapy Roctavian after slow uptake and modest sales, despite regulatory success
So what does this mean for gene therapy in haemophilia?
Approval and elegant biology are not equivalent to real-world adoption.
Durability, re-dosing, eligibility, cost and system readiness are what decide.
Today’s ‘one-and-done’ liver-directed AAV approaches may stay niche while highly effective factor and non-factor prophylaxis remain widely available.
In my view, this is less a verdict against gene therapy than a call to align the next generation of trials and products with what truly matters to people with hemophilia: durable bleed protection, full reimbursement, and sustainable integration into care.”
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