REGENXBIO announces expansion of gene therapy programme for the treatment of MPS II

On Wednesday 30 Septemeber, REGENXBIO released an update on the progress of their investigational gene therapy programme for MPS II Hunters disease.


Key highlights from the update are:

  • The ongoing Phase I/II trial plans to enroll up to six additional patients.

  • REGENXBIO are initiating a new study to evaluate RGX-121 in patients with severe MPS II ages 5-18 years old. Participating sites will be informed as soon as possible.

  • A new prospective observational study is expected to start. This non-interventional study is critically important, as it provides detailed characterisation of neurocognitive development and key biomarkers in patients with severe MPS II. 

Expansion of the ongoing Phase I/II trial, and start-up of an observational study, have been communicated to families. What is new for families is the announcement of a protocol to treat children 5-18 years of age



Today's update reflects significant forward progress in our clinical program for the treatment of MPS II, as we expand the program to gain