Posted by Michael Wonder on 13 Jun 2018
RegenxBio receives FDA fast track designation for RGX-111 gene therapy for the treatment of mucopolysaccharidosis type I
12 June 2018 - Phase I clinical trial expected to enroll children and adults with MPS I.
RegenxBio today announced that the U.S. FDA has granted fast track designation for RGX-111. RGX-111 is a novel, one-time investigational treatment for mucopolysaccharidosis Type I (MPS I), that is designed to deliver the human iduronidase gene directly to the central nervous system using the NAV AAV9 vector.
Leading international gene therapy and lysosomal storage disease centers will participate in the Phase I clinical trial for RGX-111 for the treatment of MPS I.
