AveXis, Inc. is a United States-based clinical-stage gene therapy company, which develops and commercializes treatments for patients suffering from rare and life-threatening neurological genetic diseases. The Company’s initial product candidate, AVXS-101, is a gene therapy product candidate, which is in Phase I clinical trial for the treatment of spinal muscular atrophy (SMA) Type 1, the genetic cause of infant mortality. Its AVXS-101 is designed to deliver functional human survival motor neuron (SMN) gene into the nuclei of motor neurons. Its AVXS-101 utilizes a non-replicating adeno-associated virus (AAV) capsid to deliver a functional copy of a human SMN gene to the patient’s own cells without modifying the existing deoxyribonucleic acid (DNA) of the patient. It utilizes, the AAV9 capsid, which crosses the blood-brain barrier, a tight protective barrier that regulates the passage of substances between the bloodstream and the brain, thus allowing for intravenous administration.
