AVXS-401, by Novartis Gene Therapies

25/01/2021. AVXS-401 is a gene therapy developed by Novartis Gene Therapies (AveXis, now is part of Novartis). It uses the AAV9 adeno-associated virus (AAV) vector to deliver a functional copy of the FXN gene to restore the production of frataxin in tissues that are severely affected by FA. Early toxicity studies conducted in healthy mice showed that AVXS-401 has good safety and tolerability. Efficacy studies have shown that in a FA mouse model lacking frataxin in the central nervous system, a single intracerebroventricular (ICV) injection of low-dose AVXS-401 can improve behavior and promote glial cell proliferation. In a mouse model lacking frataxin in the heart, AVXS-401 can restore heart function, prolong the median survival by more than 3 times, and prevent the onset of cardiovascular disease. When the therapeutic dose is increased proportionally and administered in non-human primates, AVXS-401 can achieve sustained expression of frataxin in the central nervous system and heart, and the effect is still obvious after 6 months of treatment.