Wolfgang Miesbach: Award-Winning Poster on Long-Term AAV Gene Therapy Outcomes in Hemophilia B
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“Delighted that this poster by Ulrike Reiss was awarded the first poster prize at EAHAD 2026 in Dublin. ‘9‑year outcomes of scAAV2/8‑LP1‑hFIXco enriched for full AAV capsids in adults with severe haemophilia B (PO041)’.
The study builds on the pioneering UCL/St Jude AAV8 haemophilia B programme, comparing a new vector prep enriched for full AAV capsids (full:empty 1:3) with the original 1:10 preparation to see whether this strategy improves FIX expression or liver safety over the long term:
FIX activity rose in the higher‑dose cohort, confirming robust liver transduction, but levels gradually declined over time instead of remaining fully stable.
Rates and patterns of vector‑associated transaminitis were similar to the earlier 1:10 study, suggesting that full‑capsid enrichment alone does not mitigate this immunogenicity signal.
Across 9 years of follow‑up there were no FIX inhibitors, no thrombotic complications and no late liver toxicity, reinforcing the long‑term safety of systemic AAV‑mediated FIX gene transfer in this cohort.
Overall, there was no clear advantage of the 1:3 full‑capsid preparation in terms of reducing transaminitis, enhancing transduction, or stabilising FIX expression compared with the original vector batch.
For me, this award‑winning poster is a great example of how meticulous vector characterisation plus truly long‑term clinical follow‑up can refine our understanding of AAV biology and guide the next generation of safer, more durablegene therapies for haemophilia B”
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