RESEARCH TRIANGLE PARK Dont call it AveXis anymore. Its now Novartis Gene Therapies.

Novartis has announced the renaming of its previously acquired AveXis gene therapy arm because of the growing importance of gene therapy to building a leading, focused medicines company with advanced therapy platforms.

AveXis, headquartered in Bannockburn, Illinois, was purchased in 2018 for $8.7 billion by the Swiss drugmakerNovartis.

The company announced early last year that it would put $60 million more into its then-new $55 million Durham County manufacturing facility, doubling the workforce here from 200 to 400. Now, according to a Novartis spokesperson, there are more than 400 people working at the Durham site, and more are being hired while the site is anticipating final FDA operational approval in 2021.

The company makes gene therapies for neurological genetic diseases. Its initial product is Zolgensma (onasemnogene abeparvovec-xioi), a gene replacement therapy to treat spinal muscular atrophy (SMA) Type 1, a deadly neuromuscular disease with limited treatment options.

AveXispurchased rightsto its gene therapy technology in 2015 for an undisclosed sum fromAsklepios BioPharmaceutical, a Chapel Hill gene therapy platform company commonly called AskBio.

AskBio said it received an upfront payment and stands to gain milestone payments and royalties based on AveXis successful development and commercialization of its treatment.

AskBios gene therapy platform is based on the work of Jude Samulski, Ph.D., a pioneering scientist in gene therapy who was recruited to the University of North Carolina School of Medicine from the University of Pittsburgh in 1993 with the help of about $250,000 in grant funding from the North Carolina Biotechnology Center. Samulski, renowned for his development of the harmless adeno-associated virus (AAV) as a vector, or delivery tool, for genes, directed UNCs Gene Therapy Center for many years.

Building on the success of Zolgensma, Novartis Gene Therapies said it 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.

DavidLennon, Ph.D., previously president of AveXis, is now president of Novartis Gene Therapies and will continue reporting to Vas Narasimhan, CEO of Novartis.

Novartis sees tremendous potential in the future of gene therapy, and weve seen the impact gene therapy can have on so many lives, said Narasimhan. 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.

The most widely used gene therapy in the world, Zolgensma treats 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 2 in more than 90% 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., its 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.

Novartis said 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 Friedreichs 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 Lennon.

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. With nearly 1 million square feet of gene therapy manufacturing capacity, Novartis Gene Therapies is the worlds largest gene therapy manufacturer.

(C) N.C. Biotech Center

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Novartis Gene Therapies is new name for gene therapy firm with growing RTP presence - WRAL Tech Wire

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