Sep 02, 2020

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.


These materials contain forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “growing,” “strategy,” “building,” “becomes,” “anticipated,” “next wave,” “to date,” “evolving,” “pursuing,” “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “seek,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding our strategy and future business plans, or regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in these materials, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that our strategy or business plans will be executed as planned, or achieve any or all of their intended goals and objectives, in any particular time frame, or at all. Neither can be any guarantee that the investigational or approved products described in these materials will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding our strategy, business plans, and products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in these materials as of this date and does not undertake any obligation to update any forward-looking statements contained in these materials as a result of new information, future events or otherwise.