About Rett Syndrome
Rett syndrome (RTT) is a devastating, rare neurological disorder characterized by slowed growth, loss of normal movement and coordination and loss of communication skills. RTT is caused by an X-linked dominant mutation in the methyl CpG binding protein 2 (MECP2) gene, which results in problems with the protein production critical for brain development. RTT occurs in approximately one of every 10,000 female births and usually begins to show signs and symptoms in infants between six and 18 months of age. Current treatments only offer symptomatic relief and do not target the genetic cause of the disease, leaving a significant unmet need.
About Genetic Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Familial or inherited forms of ALS reflect five to 10 percent of ALS cases, or approximately one to two thousand people in the U.S., and can be caused by mutations in several genes known to be associated with ALS. Approximately 20 percent of these cases are caused by mutations in the gene that produces the copper zinc superoxide dismutase 1 (SOD1) enzyme, which leads to a progressive degeneration of motor neurons affecting movement and muscle control. ALS usually occurs in people between the ages of 40 and 70. Current treatments only offer modest benefits and do not target the genetic cause of the disease, leaving a significant unmet need.
About AveXis, Inc.
AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The companys initial proprietary gene therapy candidate, AVXS-101, is in an ongoing Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit www.avexis.com.
This press release contains forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis research, development and regulatory plans for AVXS-101, including the potential of AVXS-101 to positively impact quality of life and alter the course of disease in children with SMA Type 1, expectations regarding AveXis research, development and regulatory plans for its programs for treatment of RTT and genetic ALS, its expectations regarding initiation of IND-enabling studies for these programs and timing of providing an update on these programs, the overall clinical development of AVXS-101, AveXis research, development and regulatory plans for AVXS-101, including AveXis commercial manufacturing process, AveXis potency assay development and the expected timing of the submission of data regarding the potency assay to the FDA, the expected timing of the initiation of AveXis planned future clinical trials, the expected timing of future meetings with the FDA, AveXis ability to meet future commercial demand for AVXS-101 through its manufacturing facility and the possibility that AveXis may be able to utilize data from its Phase 1 clinical trial in the regulatory pathway of AVXS-101. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the scope, progress, expansion, and costs of developing and commercializing AveXis product candidates; regulatory developments in the U.S. and EU, as well as other factors discussed in the Risk Factors and the Managements Discussion and Analysis of Financial Condition and Results of Operations section of AveXis Annual Report on Form 10-K for the year ended December 31, 2016, filed with the SEC on March 16, 2017. In addition to the risks described above and