Clinical Trial RGX-111 for MPS I Halted

Nov 9, 2023 | Clinical Trials, News

November 8, 2023

Dear MPS I community, 

This afternoon REGENXBIO Press Release 8Nov2023, we announced a corporate restructuring that impacts our development of potential AAV gene therapies for MPS I. We are disappointed to share that REGENXBIO will no longer be moving forward with the development of RGX-111 for the treatment of MPS I.  This was a very difficult decision, but due to the challenging economic market facing biotechnology companies REGENXBIO is no longer in a position to continue this program on our own. 

Ken Mills, President and Chief Executive Officer of REGENXBIO, said, “We believe that gene therapy has the potential to impact the course of this disease and we are committed to pursuing alternative paths forward for RGX-111, including finding a potential partner.”

Patients who have been dosed in the clinical trials of RGX-111 will continue to be followed, but new patients will not be enrolled at this time. Families of patients currently enrolled in these trials will be contacted through their study site and can reach out to their study team for more information.

It has been a privilege to be part of your community and to have experienced first-hand the amazing work of the MPS organizations and families to advocate for change for patients. Please know that we are committed to doing everything we can for the MPS I community to identify a partner to advance this investigational therapy to patients.