AXGT - Axovant on go with pivotal study of gene therapy for rare inherited lipid disorders

The FDA has signed off on a registrational study evaluating Axovant Gene Therapies' (AXGT) AXO-AAV-GM2 gene therapy for patients with Tay-Sachs disease and Sandhoff disease, two rare inherited disorders characterized by the buildup of certain kinds of fats in the brain and other organs caused by the absence of a key enzyme.The study will enroll both infantile and juvenile subjects with GM2 gangliosidosis (Tay-Sachs).Shares up 8% premarket on modest volume.

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Axovant on go with pivotal study of gene therapy for rare inherited lipid disorders