|Headquarters||Illinois, United States|
|Key people||Sean P. Nolan (CEO), Thomas Joseph Dee (CFO), Sukumar Nagendran (Chief Medical Officer) |
|Investors||Adage Capital Management, Boxer Capital, Deerfield, Foresite Capital, Janus Capital Group, QVT Financial, RA Capital Management, Roche Venture Fund, Rock Springs Capital, RTW Investments LLC, T. Rowe Price, Venrock |
|Number of employees||18 |
AveXis, Inc., a clinical-stage gene therapy company, engages in developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. 
It has strategic collaboration and license agreements with Nationwide Children's Hospital, The Research Institute at Nationwide Children's Hospital, REGENXBIO Inc. and Asklepios Biopharmaceutical, Inc. The company was formerly known as BioLife Cell Bank, Inc. and changed its name to AveXis, Inc. in January 2014. AveXis, Inc. was founded in 2010 and is headquartered in Bannockburn, Illinois.
- Their initial product candidate,AVXS-101, is their proprietary gene therapy product candidate currently in a Phase 1 clinical trial for the treatment of spinal muscular atrophy, or SMA,Type 1, the leading genetic cause of infant mortality.
- SMA Type 1 is a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, resulting in mortality or the need for permanent ventilation support before the age of two for greater than 90% of patients.
- The survival motor neuron, or SMN, is a critical protein for normal motor neuron signaling and function.
- Patients with SMA Type 1 either carry a mutation in their SMN1 gene or their SMN1 genes have been deleted, which prevents them from producing adequate levels of functional SMN protein.
- AVXS-101 is designed to deliver a fully functional human SMN gene into the nuclei of motor neurons that then generates an increase in SMN protein levels. This will result in improved motor neuron function and patient outcomes.
- They have completed enrollment in our ongoing Phase 1 clinical trial, in which we have treated 15 SMA Type 1 patients as of December 31, 2015, and have observed a favorable safety profile that is generally well-tolerated and have also observed preliminary signs of improved motor function.
- The U.S. Food and Drug Administration, or FDA, has granted AVXS-101 orphan drug designation for the treatment of all types of SMA and fast track designation for the treatment of SMA Type 1. 
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