AveXis EU Ltd. Granted SME Status by the European Medicines Agency
AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced that the European Medicines Agency (EMA) has granted AveXis’ wholly owned European subsidiary,... - March 21, 2015
AveXis Announces the Formation of Its European Subsidiary, AveXis EU, Ltd.
AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced the formation of its EU-based wholly-owned subsidiary, AveXis EU, Ltd. The subsidiary will allow AveXis... - February 27, 2015
AveXis Closes Its Series C Financing Round with Deerfield Management and Roche Venture Fund
AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced it has completed a $10 million financing round led by Deerfield Management and Roche Venture Fund. In... - January 08, 2015
AveXis Announces Dosing of First Patient in Intermediate Cohort in Gene Transfer Trial
AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced that the first patient in the intermediate cohort in the Gene Transfer Clinical Trial for SMA Type 1 has... - December 10, 2014
AveXis Announces Launch of New Patient-Oriented Website Focused on Spinal Muscular Atrophy
AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), is pleased to announce the launch of the company’s brand new website specifically focused on SMA: www.smastudy.org. - December 04, 2014
AveXis to Present at the 26th Annual Piper Jaffray Healthcare Conference
AveXis, a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy, today announced that Mr. John Carbona, Chief Executive Officer, and Dr. Brian Kaspar will present at the 26th Annual Piper Jaffray... - November 21, 2014
AveXis Announces the Completion of Dosing of the Low Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy
AveXis, Inc., a clinic-stage gene therapy company, today announced the completion of dosing in the low dose cohort in the world's first human gene therapy trial for the treatment of infants with spinal muscular atrophy (SMA). The trial (NCT02122952) opened for enrollment in April... - October 08, 2014
AveXis Receives Orphan Drug Status from the US Food and Drug Administration (FDA) for chariSMA for the Treatment of Spinal Muscular Atrophy
AveXis Inc., a synthetic biology platform company primarily focused on developing treatments for Spinal Muscular Atrophy (SMA) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its product scAAV9, called chariSMA to treat Spinal Muscular Atrophy... - October 04, 2014
AveXis Announces Dosing of First Patient for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 at Nationwide Children’s Hospital
AveXis, Inc., a synthetic biology platform company, today announced that the first patient in the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 has now been enrolled and dosed by Nationwide Children’s Hospital. This trial utilizes chariSMA, the gene therapy product developed... - June 26, 2014
AveXis to Present at Piper Jaffray GenomeRX Symposium
AveXis, a clinical stage synthetic biology platform company, today announced that Mr. John Carbona, Chief Executive Officer and other members of the AveXis team will participate in the panel discussion “The Power Alley: Gene Therapy For Neuromuscular Disorders” at the Piper Jaffray... - June 20, 2014
AveXis to Present at Families of Spinal Muscular Atrophy Annual Conference
AveXis, a clinical stage synthetic biology platform company, today announced that Dr. Allan Kaspar, Chief Scientific Officer, will be presenting at The Family and Researcher Poster Session at The Annual Spinal Muscular Atrophy Conference, which will be held on Thursday, June 12, 2014 in National... - June 08, 2014
AveXis Announces Opening of Enrollment for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 at Nationwide Children's Hospital
AveXis, Inc., a clinic-ready synthetic biology platform company, today announced that the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 has now formally begun enrollment at Nationwide Children’s Hospital. This trial will utilize chariSMA, the gene therapy product developed... - May 16, 2014
AveXis Closes a Round of Financing from PBM Capital to Advance Its SMA Gene Therapy Program
AveXis, Inc., a clinic-ready synthetic biology platform company, today announced it has completed a financing round led by PBM Capital Group. This round of financing will help support the company’s gene therapy program targeting spinal muscular atrophy (SMA). AveXis’ gene therapy... - April 23, 2014
AveXis-BioLife Launch New Website
AveXis-BioLife is pleased to announce the launch of the company’s brand new website: www.avexisinc.com. The October 2013 unveiling of the website coincides with the company’s licensing of the gene therapy program from The Kaspar Laboratory for the treatment of Spinal Muscular Atrophy... - November 01, 2013
AveXis-BioLife Licenses Spinal Muscular Atrophy (SMA) Patent Portfolio from Nationwide Children's Hospital and The Ohio State University
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... - October 21, 2013
AveXis BioLife Announce The Research Institute at Nationwide Children’s Hospital Received Fast Track Status for Spinal Muscular Atrophy Treatment
AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children’s Hospital received Fast Track designation from the U.S. Food and Drug Administration for its scAAV9.CB.SMN gene therapy product for the treatment of spinal muscular... - October 18, 2013
AveXis Inc. Names Director, Scientific and Corporate Affairs
AveXis Inc. (avexisinc.com), a synthetic biology platform company, today announced Mindy Du as the company’s new Director of Scientific and Corporate Affairs. This is an essential position within the company, as AveXis is focused on research/development of leading-edge technology for disease... - September 13, 2013
AveXis Inc. Names Director of Clinical Operations and Research
AveXis Inc. (avexisinc.com), a synthetic biology platform company, today announced Minna Du as the company’s Director of Clinical Operations and Research. This is a critical position within the company, as AveXis is focused on research/development of technology for disease treatment. - August 21, 2013
AveXis Inc. Names Chief Scientific Officer
AveXis Inc. (avexisinc.com), a synthetic biology platform company focused on research and development of technology for disease treatment, today announced Dr. Allan A. Kaspar will serve as the company’s first Chief Scientific Officer (CSO). Dr. Kaspar has held research positions at multiple... - August 01, 2013
New Company Formed to Focus on New Technology for Disease Treatment
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... - July 27, 2013