Wolfgang Miesbach: Nonviral Gene Therapy for Haemophilia A Using Lipid Nanoparticles
Wolfgang Miesbach, Head of Hemostasis/Hemophilia Center at Medical Clinic 2 at University Hospital Frankfurt, shared a post on LinkedIn:
“Two of the hardest limits in haemophilia gene therapy are pre existing anti AAV antibodies and the fact that you only get one shot.
A poster at ESGCT2026 goes non viral instead: plasmid DNA delivered in lipid nanoparticles.
The obstacle is the cGAS STING response to cytoplasmic DNA answered here with a dexamethasone prodrug built into the LNP, plus ImmTOR nanoparticles to induce tolerance to the transgene.
DexLNP sharply reduced transaminase rise and weight loss
Therapeutically relevant FVIII activity in F8 mice after a single dose, sustained to 3 months
With ImmTOR: no anti FVIII inhibitors at week 12 (0 8 vs 4 4 without)
FVIII activity still declines over time, and whether LNP DNA can genuinely be redosed is the question that matters next.
A platform free of anti vector immunity and open to repeat administration would change the arithmetic for a great many patients to Sebastian Hook, Simon Krooss, Michael Ott and the team in Hannover.”
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