Regenxbio Results from RGX-121 for MPSII
Regenxbio (RGNX) announced interim data from Phase I/II trial of RGX-121 for Mucopolysaccharidosis Type II (MPS II). RGX-121 is designed to deliver the gene that encodes iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) using the AAV9 vector.
About Mucopolysaccharidosis Type . . .