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