December 8, 2025Blood

An engineered AAVrh10 vector containing an optimized factor IX transgene improves therapeutic response during hemophilia b gene therapy

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Increased factor IX levels observed with optimized AAVrh10 vector in hemophilia B mice, indicating potential therapeutic benefits.
The engineered transgene demonstrated significant enhancement in FIX activity, reaching levels between 529% and 1435% after treatment.
Assessment via immunohistochemistry confirmed high transgene expression in liver following gene therapy with both AAVrh10 mutants.
These findings highlight the potential of AAVrh10 as a viable option for long-term hemophilia B treatment without immune responses.

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Gene therapy improves factor IX levels in hemophilia B, indicating the role of engineered vectors in treatment efficacy.

Sarangi et al. (2025) studied this question.

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