AAVrh10K169Q-FIX Padua

AAVrh10K169Q-FIX Padua is an engineered adeno-associated virus (AAV) gene therapy candidate designed for the treatment of hemophilia B. It utilizes a non-human primate-derived AAVrh10 capsid featuring a K169Q mutation at a predicted neddylation site, which is intended to enhance vector stability and transduction efficiency by avoiding post-translational modification-mediated degradation. The vector carries a codon-optimized transgene encoding the hyperactive human coagulation Factor IX (FIX) Padua variant (R338L) under the control of a novel Kozak sequence to maximize protein expression. In preclinical studies using hemophilia B mouse models, the therapy demonstrated supraphysiological FIX activity levels (ranging from 529% to 1435%) and a favorable safety profile with no significant vector-induced immune activation or inhibitor development. The program was developed by researchers at the Indian Institute of Technology Kanpur and the Sanjay Gandhi Post Graduate Institute of Medical Sciences, with support from the Pfizer ASPIRE program.

Other names

AAVrh10K169Q-FIX-PaduaAAVrh-10K169Q-FIX-PaduaAAVrh 10K169Q-FIX-PaduaAAVrh10K169Q-LP1-hFIX-PaduaAAVrh-10K169Q-LP1-hFIX-PaduaAAVrh 10K169Q-LP1-hFIX-Padua