Benzinga | Sep 30, 2020 07:05AM EDT

REGENXBIO Announces Expansion Of Clinical Development Program For RGX-121 For The Treatment Of Mucopolysaccharidosis Type II

ROCKVILLE, Md., Sept. 30, 2020 /PRNewswire/ --REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV(r)Technology Platform, today announced the expansion of the RGX-121 program for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, to gain additional insight into the neurodegenerative manifestations of the disease and evaluate RGX-121 in a broader patient population. RGX-121 is an investigational one-time gene therapy, designed to use the AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) through intracisternal administration.