https://www.PR.com/ Latest news releases from PR.com for AveXis Inc. Wed, 14 Oct 2026 02:59:36 -0400 © 2026 PR.com 60 en-us https://www.PR.com/images/logo_white.jpg https://www.PR.com/ 68 69 PR.com provides press release distribution, and is a unique directory of businesses, products and services. Find full company profiles. https://www.pr.com/press-release/611255 Sat, 21 Mar 2015 03:00:00 -0400 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, AveXis EU Ltd., SME (Small and Medium Sized Business Entities) status. The EMA, recognizing the need to encourage drug [PR.com] https://www.pr.com/press-release/611255 AveXis Inc. https://www.pr.com/press-release/607604 Fri, 27 Feb 2015 03:00:00 -0500 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 to move forward with its plans to open clinical trials in the European Union and leverage European Medicines Agency’s [PR.com] https://www.pr.com/press-release/607604 AveXis Inc. https://www.pr.com/press-release/599871 Thu, 08 Jan 2015 03:00:00 -0500 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 conjunction with this financing round, Jonathan Leff of Deerfield and Carole Nuechterlein of Roche Venture Fund will join the [PR.com] https://www.pr.com/press-release/599871 AveXis Inc. https://www.pr.com/press-release/596176 Wed, 10 Dec 2014 03:00:00 -0500 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 been dosed. The world’s first human gene therapy trial for the treatment of infants with SMA (NCT02122952) evaluates [PR.com] https://www.pr.com/press-release/596176 AveXis Inc. https://www.pr.com/press-release/595291 Thu, 04 Dec 2014 03:00:00 -0500 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. The December 2014 unveiling of the website is an integral element to AveXis’ multi-pronged effort to grow awareness of [PR.com] https://www.pr.com/press-release/595291 AveXis Inc. https://www.pr.com/press-release/593288 Fri, 21 Nov 2014 03:00:00 -0500 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 Healthcare Conference on Tuesday, December 2, 2014. AveXis will present at 1:50 p.m. EDT in the Kennedy 2 room on the fourth [PR.com] https://www.pr.com/press-release/593288 AveXis Inc. https://www.pr.com/press-release/585596 Wed, 08 Oct 2014 03:00:00 -0400 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 2014. “History in gene transfer therapy was made on May 13th, when an infant was successfully injected with about 400 trillion viral [PR.com] https://www.pr.com/press-release/585596 AveXis Inc. https://www.pr.com/press-release/585132 Sat, 04 Oct 2014 03:00:00 -0400 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 patients. SMA is an autosomal recessive birth defect, and the largest genetic killer of infants. It is also a disease that [PR.com] https://www.pr.com/press-release/585132 AveXis Inc. https://www.pr.com/press-release/566494 Thu, 26 Jun 2014 03:00:00 -0400 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 by Dr. Brian Kaspar, of Nationwide Children’s Hospital, and licensed to AveXis for development and commercialization. [PR.com] https://www.pr.com/press-release/566494 AveXis Inc. https://www.pr.com/press-release/565389 Fri, 20 Jun 2014 03:00:00 -0400 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 GenomeRX Symposium on Monday, June 23, 2014. The panel is scheduled to begin at 10:40 a.m. EDT and will take place at The Yale [PR.com] https://www.pr.com/press-release/565389 AveXis Inc. https://www.pr.com/press-release/563008 Sun, 08 Jun 2014 16:47:50 -0400 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 Harbor, Maryland. The session will take place from 4-6 p.m. in the Prince George Exhibit Hall E at the Gaylord National Resort [PR.com] https://www.pr.com/press-release/563008 AveXis Inc. https://www.pr.com/press-release/558399 Fri, 16 May 2014 03:00:00 -0400 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 by Dr. Brian Kaspar, of Nationwide Children’s Hospital, and licensed to AveXis for development and commercialization. [PR.com] https://www.pr.com/press-release/558399 AveXis Inc. https://www.pr.com/press-release/554182 Wed, 23 Apr 2014 03:00:00 -0400 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 product, chariSMA, is a unique approach to the disease which affects more infants than any other genetic disease in the world today. [PR.com] https://www.pr.com/press-release/554182 AveXis Inc. https://www.pr.com/press-release/525031 Fri, 01 Nov 2013 03:00:00 -0400 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 (SMA). The new website offers a contemporary look and more user friendly experience. It features resources and functionality [PR.com] https://www.pr.com/press-release/525031 AveXis Inc. https://www.pr.com/press-release/522600 Mon, 21 Oct 2013 14:35:22 -0400 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 [PR.com] https://www.pr.com/press-release/522600 AveXis Inc. https://www.pr.com/press-release/522276 Fri, 18 Oct 2013 03:00:00 -0400 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 atrophy (SMA). This new gene therapy product created by scientists at The Research Institute was granted Fast Track status after [PR.com] https://www.pr.com/press-release/522276 AveXis Inc. https://www.pr.com/press-release/515582 Fri, 13 Sep 2013 03:00:00 -0400 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 treatment. While Ms. Du stays involved in AveXis’ first focus in spinal muscular atrophy (SMA), she will continuously [PR.com] https://www.pr.com/press-release/515582 AveXis Inc. https://www.pr.com/press-release/510919 Wed, 21 Aug 2013 09:00:00 -0400 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. Specifically, AveXis’ first focus is spinal muscular atrophy (SMA), the most common genetic cause of infant mortality in the [PR.com] https://www.pr.com/press-release/510919 AveXis Inc. https://www.pr.com/press-release/506911 Thu, 01 Aug 2013 05:00:00 -0400 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 biotech companies—notably, in early-stage drug development. He joins the AveXis team, bringing over 12 years of [PR.com] https://www.pr.com/press-release/506911 AveXis Inc. https://www.pr.com/press-release/505812 Sat, 27 Jul 2013 03:00:00 -0400 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 treatments to people with unmet medical needs. Spinal muscular atrophy (SMA), a rare/orphan disease, is the first focus. SMA is the [PR.com] https://www.pr.com/press-release/505812 AveXis Inc.