Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

17 March 2022

The search for novel and innovative treatments in Haemophilia has seen significant progress in the last few years, thanks in large part to the work done by our very own Professor Johnny Mahlangu. In this open-label, single-group, multicenter, phase 3 study, Valoctocogene roxaparvovec (an adeno-associated virus 5 (AAV5)-based gene-therapy) was investigated to see if it had any impact on bleeding and the use of factor VIII concentrate in men with severe hemophilia A.

The treatment provided endogenous factor VIII production with no development of factor VIII inhibitors or thrombosis .

We congratulate Prof Mahlangu on an excellent publication.

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