Daily Archives: February 1, 2019

REGENXBIO Receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy

“REGENXBIO announced the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to RGX-181. RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene. In addition to the Rare