A 2nd Patient Death Reported in Novartis’ Gene Therapy Trial

biospace | April 22, 2019

A 2nd Patient Death Reported in Novartis’ Gene Therapy Trial
Just as Novartis and AveXis’ gene therapy, Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) is progressing toward approval, a second patient death was reported. Last week, AveXis, a Novartis  company, announced interim data from its Phase III STR1VE clinical trial of Zolgensma in SMA Type 1. SMA is a severe neuromuscular disease caused by a mutation in the SMN1 gene, which codes for SMN, a protein necessary for motor neuron function. The disease is noted by the loss of motor neurons, which leads to progressive muscle weakness and paralysis. It is rare, occurring in about one in 10,000 live births. Type 1 is lethal and typically results in death by the age of two years. Zolgensma is a gene therapy in development as a one-time infusion for SMA Type 1. It uses a virus vector to deliver a copy of the human SMN gene. In December, the Food and Drug Administration (FDA) accepted AveXis’ Biologics License Application (BLA) for Zolgensma, with a target action date in May 2019. It is also expected to be approved in Japan and the European Union later this year.

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The University of Alabama at Birmingham's Master of Science in Biotechnology Program is designed to train you for a career in a sector that is adding new jobs at nearly twice the rate of the overall national private sector. You will learn the practices and principles of both science and business that are necessary for you to succeed in technology and product development. The biotechnology curriculum allows you to adapt your coursework based on your interests including drug development, regulatory affairs, technology transfer and marketing.

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