iRegene Secures Historic RMAT Designation for Parkinson’s iPSC Therapy

January 16, 2026-iRegene Therapeutics has reached a historic regulatory milestone as its lead candidate, NouvNeu001, became the first allogeneic induced pluripotent stem cell (iPSC)-derived therapy to receive both Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. FDA. This “regulatory double-play” underscores the transformative potential of iRegene’s “AI + Chemical Induction” platform, which bypasses traditional genetic engineering to create high-purity dopaminergic progenitor cells. Designed to replace the neurons lost to Parkinson’s disease, NouvNeu001 aims to restore the brain’s natural dopamine production and reconstruct disrupted neural circuits, offering a potential disease-modifying cure rather than a mere symptomatic fix.

The FDA’s decision was fueled by compelling Phase I clinical data demonstrating significant and durable motor recovery. In the trial’s low-dose cohort, patients experienced an average improvement of 30.6 points in their MDS-UPDRS Part III motor scores during “OFF” states (a 52.82% reduction in symptom severity) at the 12-month mark. High-dose participants showed similar robust gains, with benefits observed to be sustained or even continuing to improve through 15 months post-transplantation. These results were achieved through a minimally invasive, stereotactically guided procedure that injects the cells into the bilateral putamen, ensuring deep integration with minimal surgical trauma.

With RMAT status now secured, iRegene will benefit from intensive FDA guidance and eligibility for priority review and accelerated approval, potentially bringing this “off-the-shelf” regenerative therapy to the millions of Parkinson’s patients worldwide much sooner than traditional timelines allow. As the company advances into Phase II trials, NouvNeu001 stands as a pivotal proof-of-concept for a new generation of chemically induced, non-genetically modified cell therapies targeting previously “incurable” neurodegenerative conditions.