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

Summary

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.

Timeline

Start

2021-03-11

Primary completion

2023-05-09

Completion

2024-05-23

Drugs

Indications