AveXis

AveXis, now operating as Novartis Gene Therapies, is a biotechnology company focused on developing treatments for rare and life-threatening neurological genetic disorders. The company was originally founded as BioLife Cell Bank in 2010, then reorganized and established as AveXis in 2012 by John D. Harkey Jr. and John Carbona. Harkey, a seasoned investor and executive with a background in business and law from the University of Texas and Stanford, co-founded the company to pioneer impactful technologies in gene therapy. The scientific foundation of AveXis was built upon research licensed in 2013 from Nationwide Children's Hospital, specifically from the laboratory of Brian Kaspar, who became a key scientific advisor.

The company's business model centers on the research, development, and commercialization of AAV-based gene therapies. Its landmark achievement was the U.S. FDA approval in May 2019 for its first product, Zolgensma (onasemnogene abeparvovec), a one-time treatment for spinal muscular atrophy (SMA), the leading genetic cause of infant mortality. Zolgensma addresses the genetic root cause of SMA by delivering a functional copy of the SMN1 gene. Revenue is generated from the sale of this high-value therapy. Zolgensma's launch price was set at $2.125 million, making it one of the most expensive drugs in the world, with Novartis offering payment plans, such as an annuity-like model of $425,000 per year for five years.

A pivotal moment in the company's history was its acquisition by Novartis in mid-2018 for approximately $8.7 billion. This acquisition integrated AveXis's AAV9 gene therapy platform and manufacturing capabilities into Novartis's broader strategy, aiming to establish a leadership position in neuroscience and gene therapies. In September 2020, Novartis rebranded AveXis to Novartis Gene Therapies to signify the growing strategic importance of this therapeutic area. Beyond SMA, the company's pipeline includes investigational treatments for other rare genetic diseases like Rett syndrome and a genetic form of amyotrophic lateral sclerosis (ALS).

Keywords: gene therapy, spinal muscular atrophy, SMA, Zolgensma, Novartis Gene Therapies, AAV9 vector, rare genetic diseases, neurological disorders, John D. Harkey Jr., Brian Kaspar, AVXS-101, one-time treatment, SMN1 gene, Rett syndrome, amyotrophic lateral sclerosis, neurodegenerative disease, pediatric genetic diseases, biopharmaceutical, clinical trials, FDA approval, orphan drugs