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Gene therapy firm to make medicines for rare genetic diseases
February 26, 2019
By: Tim Wright
Editor-in-Chief, Contract Pharma
Gene therapy firm Avexis has unveiled plans to invest $60 million to expand is manufacturing center in Durham, NC. The new center was first announced in May of last year and once complete will create 200 jobs. A Novartis company headquartered in Bannockburn, IL, AveXis’ initial product candidate, AVXS-101, now known as Zolgensma (onasemnogene abeparvovec-xioi), is an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. Zolgensma is designed to address the genetic root cause of SMA Type 1, a deadly neuromuscular disease with limited treatment options. ZOLGENSMA previously received Breakthrough Therapy designation and has been granted Priority Review by the FDA, with regulatory action anticipated in May 2019. “Our primary focus is to bring gene therapies to patients suffering from devastating rare neurological genetic diseases, such as SMA, genetic amyotrophic lateral sclerosis and Rett syndrome. Continued investment in our infrastructure in North Carolina will allow us to manufacture multiple gene therapies simultaneously, helping us reach more patients, faster,” said Andy Stober, senior vice president of technical operations and chief technical officer, AveXis. “Gene therapy manufacturing requires a highly skilled team, and Research Triangle Park is an ideal location for our continued expansion as it enables us to recruit top talent, including through partnership with local schools and colleges.”
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