Research
Durham, NC — April 15, 2025 — The National MPS Society welcomes the news that Spruce Biosciences is now leading the development of the TA-ERT program (formerly AX 250), bringing renewed hope and momentum to the MPS IIIB (Sanfilippo) community.
Spruce Biosciences has made clear its intention to move swiftly and responsibly, acknowledging the long-standing advocacy and involvement of families, researchers, and clinicians. The company recognizes the challenges and uncertainty caused by the previous pause in development and is committed to rebuilding trust through open, consistent engagement.
“We are encouraged and deeply grateful to see Spruce Biosciences advancing the TA-ERT program for individuals living with MPS IIIB, also known as Sanfilippo Syndrome,” said National MPS Society President & CEO Terri Klein. “This marks an important step forward in addressing an urgent, unmet need for this patient community, and we commend the renewed commitment to those impacted by MPS IIIB. The National MPS Society remains hopeful and supportive as this promising therapy moves toward clinical development and, ultimately, to the families who are waiting.”
Manufacturing of TA-ERT has resumed to ensure the availability of products, and plans have been made to pursue accelerated approval in the U.S., initiate a registrational clinical study, and expand global early access opportunities by early 2026.
Community partnership is a central pillar of Spruce Bioscience’s approach. A dedicated Patient Engagement function has been established, and additional opportunities for communication, feedback, and collaboration will be available to the patient community.
The National MPS Society is encouraged by Spruce Biosciences’ renewed dedication to this program, and we look forward to continuing to work together on behalf of families living with MPS III.
To read Spruce Bioscience’s full statement, please click here.
