Posted by Michael Wonder on 03 May 2018
RegenxBio receives FDA fast track designation for RGX-121 gene therapy for the treatment of mucopolysaccharidosis Type II
2 May 2018 - Phase I/II clinical trial expected to enrol children with MPS II.
RenenxBio today announced that the U.S. FDA has granted fast track designation for RGX-121. RGX-121 is a novel, one-time investigational treatment for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, that is designed to deliver the human iduronate 2-sulphatase 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/II clinical trial for RGX-121 for the treatment of MPS II.
