![]() 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 SMAĪVXS-101 to treat SMA Type 1, we plan to develop AVXS-101 to treat additional SMA types and develop other novel treatments for rare neurological genetic diseases. ![]() We have completed enrollment in our ongoing Phase 1 clinical trial, in which we have treatedġ5 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 motorįunction. We believe this will result in improved motor neuron function and patient outcomes. 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 Them from producing adequate levels of functional SMN protein. Patients with SMA Type 1 either carry a mutation in their SMN1 gene or their SMN1 genes have been deleted, which prevents The survival motor neuron, or SMN, is aĬritical protein for normal motor neuron signaling and function. SMA Type 1 is a lethal genetic disorder characterized by motor neuron loss andĪssociated muscle deterioration, resulting in mortality or the need for permanent ventilation support before the age of two for greater than 90% of patients. Treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality. Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in a Phase 1 clinical trial for the Rare and life-threatening neurological genetic diseases. We are a clinical-stage gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from You should read this entire prospectus carefully, especially the "Risk Factors" section beginning on page 11 and ourĬonsolidated financial statements and the related notes appearing at the end of this prospectus, before making an investment decision.Īs used in this prospectus, unless the context otherwise requires, references to "we," "us," "our," "the company" and "AveXis" refer to Of the information you should consider before investing in our common stock. This summary highlights information contained elsewhere in this prospectus. The information contained in this prospectus is current only as of its date. Offered hereby, but only under circumstances and in jurisdictions where it is lawful to do so. This prospectus is an offer to sell only the shares We take no responsibility for, and can provide no assurance as to the reliability of, any other information that others may give you. Have not authorized anyone to provide any information or to make any representations other than those contained in this prospectus or in any free writing prospectuses we have Federal Income Tax Consequences to Non-U.S. ![]() Management's Discussion and Analysis of Financial Condition and Results ofĬertain Relationships and Related Party Transactions Special Note Regarding Forward-Looking Statements Any representation to the contrary is a criminal offense. Neither the Securities and Exchange Commission nor any other regulatory body has approved or disapproved of these securities or passed upon the accuracy orĪdequacy of this prospectus. See "Risk Factors" on page 11 to read about factors you should consider before buying shares of our common stock. Reporting requirements for this prospectus and future filings. Listing on The NASDAQ Global Select Market under the symbol "AVXS."Īre an "emerging growth company" as that term is used in the Jumpstart Our Business Startups Act of 2012 and, as such, have elected to comply with certain reduced public company The initial public offering price is $20.00 per share. To this offering, there has been no public market for our common stock. All of the 4,750,000 shares of our common stock are being sold This is an initial public offering of shares of common stock of AveXis, Inc. Index to Consolidated Financial Statements Use these links to rapidly review the document
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