AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXS - 101. (sciencedaily.com)
• Nightstar Therapeutics, a London - based gene therapy company focused on retinal diseases, set the terms of its IPO. (fortune.com)
• Krystal Biotech, a Pittsburgh, Penn. - based gene therapy company focused on dermatological diseases, filed for an IPO Monday. (fortune.com)