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Condition update published on

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 additional insight into the potential treatment effects of RGX-121 in more patients. REGENXBIO is committed to advancing potential gene therapy treatment options for MPS II, as there remains a significant unmet medical need to address the neurological manifestations and prevent or stabilize cognitive decline for patients" Steve Pakola, M.D., Chief Medical Officer of REGENXBIO

You can read the full statement on the REGENXBIO website here.

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