New Company Formed to Focus on New Technology for Disease Treatment

Dallas, TX, July 27, 2013 --(PR.com)-- AveXis Inc. (avexisinc.com) announced its formation as a company focused on research and development of technology specifically for disease treatment.

A synthetic biology platform company, AveXis has, at its core, a desire to establish unique industry alliances which will bring innovative treatments to people with unmet medical needs. Spinal muscular atrophy (SMA), a rare/orphan disease, is the first focus.

SMA is the most common genetic cause of infant mortality in the world, and is the second most common inherited autosomal recessive disorder. Currently, there is no medical treatment for SMA.

Located in Dallas, AveXis was developed by the leadership of BioLife Cell Bank and is being financed by White Rock Capital Texas, Inc. and others. Current Chief Executive Officer, John A. Carbona retains his position and now also acts in a similar position for AveXis. BioLife founders, Dr. David G. Genecov and John D. Harkey Jr., are pleased to see AveXis continue their original vision for BioLife (empowering researchers as they develop new therapies) with a focus on treatments that are disease-specific.

“This past year has been non-stop for our team. Last year we announced our BioLife-Intrexon partnership working with stem cells, and now the formation of AveXis will herald our entry into the gene therapy field—more specifically, gene replacement therapy,” said John A. Carbona, CEO. “In SMA, I anticipate making announcements over the coming months that will bring everyone up to date on our accelerated plans to enter Phase I clinical trials.”

The company’s name has both scientific and mythological meaning. AveXis is derived from: Av = adeno-associated virus (AAV) serotype 9; ve = vector; X = DNA helix; is = Isis, the goddess of children, nature and magic.

For more information about AveXis: 1-855-9-AVEXIS or avexisinc.com.

About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons. SMA is caused by a genetic defect in the SMN1 gene which codes SMN, a protein necessary for survival of motor neurons. SMA kills more infants than any other genetic disease in today's world.

About AveXis Inc.
Based in Dallas, Texas, AveXis is a synthetic biology platform company establishing unique industry alliances to create innovative treatments for people with unmet medical needs. Spinal muscular atrophy (SMA) is the company’s first focus.