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🔎 Fund Overview

AveXis, Inc. is a clinical-stage gene therapy company. The Company operates through the developing and commercializing gene therapy treatments for patients suffering from neurological genetic diseases segment. The Company's product candidate, AVXS-101, is its gene therapy product candidate that is in a Phase I clinical trial for the treatment of spinal muscular atrophy (SMA) Type 1, which is a genetic disorder characterized by motor neuron loss and associated muscle deterioration. The survival motor neuron (SMN) is a protein for normal motor neuron signaling and function. Patients with SMA Type 1 either have experienced a deletion of their SMN1 genes, which prevents them from producing adequate levels of functional SMN protein, or carry a mutation in their SMN1 gene. AVXS-101 is designed to deliver a functional human SMN gene into the nuclei of motor neurons that then generates an increase in SMN protein levels.