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AveXis- BioLife Licenses Spinal Muscular Atrophy (SMA) Patent Portfolio from Nationwide Children’s Hospital and The Ohio State University

Columbus, OH - 10/18/2013

BioLife, a synthetic biology platform company soon to be renamed AveXis, has been granted exclusive rights to the spinal muscular atrophy gene therapy program developed at The Research Institute at Nationwide Children’s Hospital and The Ohio State University in Columbus, Ohio.

The license enables the company’s focus on the treatment of children with spinal muscular atrophy (SMA), a motor neuron disease which is the leading genetic killer of children under the age of two. SMA is the second most common pediatric autosomal recessive disorder with an incidence of one in 6,000 live births.  SMA is known to be caused by lack of expression of survival motor neuron (SMN) protein due to a genetic loss of the SMN1 gene.

The licensed technology encompasses the unique ability to deliver the SMN gene across the blood brain barrier or through the cerebrospinal fluid to effectively target motor neurons throughout the brain and spinal cord.  In pre-clinical efficacy studies in the severe rodent model of SMA, a one-time treatment of the gene therapeutic improved survival from the average lifespan of 14 days to over 400 days.  Additionally, the one-time treatment was safe and well tolerated.

AveXis-BioLife has secured the key intellectual property developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University.  The findings have been led by Brian K. Kaspar, Ph.D. a Principal Investigator at The Research Institute at Nationwide Children’s Hospital and Associate Professor at The Ohio State University College of Medicine along with collaborator Arthur Burghes, Ph.D. Professor at The Ohio State University.   Dr Kaspar stated, “We are thrilled to partner our SMA program with AveXis-BioLife to rapidly move our findings to SMA patients and look forward to initiating human clinical trials in early 2014.”

A clinical trial has been planned with Jerry R. Mendell, M.D., director of the Center for Gene Therapy andPrincipal Investigator in The Research Institute at Nationwide Children’s Hospital and the team recently received approval for an investigational new drug application in September.

“Being in partnership with this brilliant team of people, who are not only compassionate but driven to eradicate this disease, is an honor for our company,” said John A. Carbona, Chief Executive Officer.

For more information about Avexis-BioLife: 214-233-4240 or

About AveXis

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.

About Nationwide Children’s Hospital

Ranked in all 10 specialties in “U.S. News & World Report’s 2013-14 Best Children’s Hospitals” and among the Top 10 on Parents magazine’s 2013 “Best Children’s Hospitals” lists, Nationwide Children’s Hospital is one of the nation’s largest not-for-profit freestanding pediatric healthcare networks providing care for infants, children, adolescents and adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s Hospital faculty train the next generation of pediatricians, scientists and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational and health services research at Nationwide Children’s Hospital.

About The Ohio State University:

Ohio State is a dynamic community of diverse resources, where opportunity thrives and where individuals transform themselves and the world. Founded in 1870, The Ohio State University is a world-class public research university and the leading comprehensive teaching and research institution in the state of Ohio. With more than 63,000 students (including 57,000 in Columbus), the Wexner Medical Center, 14 colleges, 80 centers, and 175 majors, the university offers its students tremendous breadth and depth of opportunity in the liberal arts, the sciences, and the professions.

Corporate Contact:

John A. Carbona, Chief Executive Officer
972-331-1905 or

Media Contact:

Jillian Bowman, Administrative Specialist
972-331-9626 or

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