Novartis has renamed the previously acquired AveXis to Novartis Gene Therapies. The change signifies the growing importance of gene therapy to building a leading, focused medicines company with advanced therapy platforms.

Building on the success of Zolgensma (onasemnogene abeparvovec), Novartis Gene Therapies will be responsible for the research, development, manufacturing and commercialization of the next wave of AAV-based innovative gene therapies. Novartis Gene Therapies will also provide manufacturing support for gene therapy work conducted by other Novartis units. Dave Lennon, previously president of AveXis, becomes president of Novartis Gene Therapies and will continue reporting into Vas Narasimhan, CEO of Novartis.

“Novartis sees tremendous potential in the future of gene therapy, and we’ve seen the impact gene therapy can have on so many lives,” said Vas Narasimhan, CEO of Novartis. “With the creation of Novartis Gene Therapies, we will continue to advance our gene therapy pipeline for rare genetic diseases, to accelerate the delivery of transformative innovation in areas of high unmet need, and to reimagine medicine for patients all around the world.”

Since its acquisition in May 2018, AveXis has been a high-profile and extremely successful investment for Novartis, evolving from a clinical-stage to a commercial organization. The most widely used gene therapy in the world, Zolgensma treats spinal muscular atrophy (SMA), the leading genetic cause of infant death. If left untreated in its most common form, SMA leads to death or the need for permanent ventilation by the age of two in more than 90 percent of cases.

To date, more than 600 patients have been treated with Zolgensma, including in clinical trials, commercially and through managed access programs. In addition to Zolgensma being approved in the U.S., it’s approved in Japan, Europe and Brazil. Novartis Gene Therapies is pursuing Zolgensma registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Australia, Argentina and South Korea in late 2020 or early 2021.

The change to Novartis Gene Therapies is the natural evolution as the company scales up to deliver Zolgensma globally and expand its reach via a robust pipeline of AAV-based gene therapies for rare genetic diseases including investigational treatments for Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Novartis Gene Therapies also establishes a seamless global presence for Zolgensma and the gene therapies to come. Instead of alternating between the AveXis and Novartis umbrella brands by market, the company comes together under one banner as a unified entity.

“Our patients and their families are the motivation for everything that we do, and under the banner of Novartis Gene Therapies, our dedicated team will continue to create a lifetime of possibilities to people suffering from rare genetic diseases,” said David J. Lennon, president of Novartis Gene Therapies. “Becoming Novartis Gene Therapies symbolizes the importance of our gene therapy advances for the future of Novartis and our industry leadership at large.”

Novartis Gene Therapies comprises more than 2,000 employees across corporate, manufacturing and research facilities in the U.S. (Illinois, North Carolina, Colorado, California); Zurich, Switzerland; and Tokyo, Japan. With nearly 1 million square feet of gene therapy manufacturing capacity, Novartis Gene Therapies is the world’s largest gene therapy manufacturer. For more information, visit the Novartis Gene Therapies section.