Novartis’ AveXis to Build Gene Therapy Factory in Durham, North Carolina

AveXis, a Novartis company based in Illinois, is investing $55 million to build a new manufacturing facility in Durham, North Carolina. The facility will create 200 jobs.AveXis will use the new plant to make its first product candidate, AVXS-101, a gene therapy to treat three types of spinal muscular atrophy (SMA). SMA Type 1, also known as Werdnig Hoffmann disease, is a severe form of SMA. It usually is seen before six months of age and includes worsening muscle weakness and hypotonia (poor muscle tone) because of the loss of lower motor neurons in the spinal cord and brain stem. There are also feeding and breathing problems. The disease is caused by mutations in the SMN1 gene. Currently the only treatment approved by the U.S. Food and Drug Administration (FDA) is Biogen’s Spinraza (nusinersen).