RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

key information

study id #: NCT04571970

condition: Mucopolysaccharidosis Type II (MPS II)

status: Recruiting


RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.

intervention: RGX-121


last updated: February 26, 2022