Sentences with phrase «stage gene therapy company»
AveXis is a clinical ‐ stage gene therapy company developing treatments for patients suffering from rare and life ‐ threatening neurological genetic diseases.
http://www.smafoundation.org/
Chicago, Ill., July 20, 2016 — AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS - 101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients.
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.
Not exact matches
• Myonexus Therapeutics, a New Albany, Oh. - based clinical - stage biotechnology company developing transformative gene therapies for limb - girdle muscular dystrophies, raised $ 2.5 million in seed funding.
US - based pharmaceutical company Gilead Sciences entered the chimeric antigen receptor (CAR) T - cell therapy business through its acquisition of Kite Pharma, and Australian biopharma company CSL Behring acquired US - based Calimmune, a company that develops clinical - stage gene therapy solutions.
The company is also seeking deeper inroads into rare diseases, with a number of assets in late - stage development, and last year it scored a major regulatory win when Europe approved its «bubble boy syndrome» gene therapy Strimvelis.
After reporting second - quarter results that contained upbeat clinical news, Spark Therapeutics (NASDAQ: ONCE), a clinical - stage company focused on gene therapy, rose 18 % as of 12:30 p.m. EDT on Wednesday.
Chris Mason is a Co-Founder and Chief Science Officer at AvroBio, a clinical - stage, gene therapy company based in Cambridge, Massachusetts.
Spark Therapeutics Gene therapy clinical stage company, spin out of Children's Hospital of Philadelphia, founded in 2013.
Alachua, Fla. - based AGTC is a clinical - stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe inherited orphan diseases in ophthalmology.