This disease is caused by mutations in the SMN1 gene, which lead to low levels of the critical SMN protein in specialized cells called motor neurons. Zolgensma, believed to be a one-time treatment, provides a normal copy of SMN1 to enable durable protein production in these nerve cells. The functional gene is delivered in a harmless and modified viral vector called AAV9 via an intravenous infusion.
Through this partnership, AveXis — now a subsidiary of Novartis — will have dedicated space at a commercial manufacturing center established by Catalent’s unit Paragon Gene Therapy and located near Baltimore-Washington International Airport.
Along with access to Catalent’s gene therapy development and manufacturing expertise, this additional capacity will enable a secure market supply of Zolgensma, a press release states. Paragon will also support the clinical supply of other viral therapies being developed by AveXis.
“We are pleased to add additional manufacturing capacity and technical expertise through this collaboration as we rapidly scale up product production,” Andy Stober, AveXis’ senior vice president of technical operations, said in the release. “We look forward to working with Catalent in continued service of the SMA community.”
Paragon focuses on newer technologies such as viral vectors, next-generation vaccines, and cancer immunotherapies. Its two facilities in Baltimore are dedicated to commercial manufacturing of most scalable AAV platforms. The company reports that it has completed more than 100 clinical GMP (Good Manufacturing Practices) AAV batches across 40 programs since 2016.
“We are honored to be a partner in this transformational journey,” said Pete Buzy, Catalent’s president, gene therapy.
AveXis and Novartis previously announced the purchase of a production facility in Longmont, Colorado, and the opening or expansion of manufacturing plants in California, Illinois, and North Carolina.