FDA Grants RMAT Designation to Allogeneic iPSC-Derived Cell Therapy for Parkinson Disease

The Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to NouvNeu001 (iRegene Therapeutics, Chengdu, China), an investigational allogeneic induced pluripotent stem cell (iPSC)–derived dopaminergic progenitor cell therapy for the treatment of Parkinson disease (PD). The designation follows early clinical data suggesting motor improvement in people with PD treated with the investigational therapy. NouvNeu001 previously received FDA Fast Track designation in August 2025.

The RMAT designation was supported by results from a phase 1 clinical study evaluating the safety and preliminary efficacy of NouvNeu001 in people with PD. In the study, participants received intracerebral administration of either a low or high dose of the cell therapy and were followed longitudinally for motor outcomes using the Movement Disorder Society–Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III. According to the company, improvements were assessed in both OFF- and ON-medication states, with follow-up extending beyond 12 months in some participants.

Key findings reported from the phase 1 study include the following:

• In the low-dose cohort, MDS-UPDRS Part III motor scores improved by 30.6 points in the OFF state (52.8% improvement from baseline) and 12.9 points in the ON state (54.7% improvement) at 12 months.
• In the high-dose cohort, MDS-UPDRS Part III scores improved by 23.3 points in the OFF state and 9.7 points in the ON state at 9 months.
• Motor improvements were reported to persist through 15 months of follow-up.

NouvNeu001 is designed as an off-the-shelf, allogeneic dopaminergic progenitor cell therapy intended to replace lost dopaminergic neurons and restore dopamine signaling in PD. RMAT designation allows for enhanced FDA engagement and potential eligibility for accelerated approval pathways as development progresses.

Source: iRegene Therapeutics. FDA grants regenerative medicine advanced therapy (RMAT) designation to iRegene’s NouvNeu001, making it the world’s first iPSC therapy with both FTD and RMAT recognitions. PR Newswire. Published January 19, 2026. Accessed January 20, 2026. https://prnmedia.prnewswire.com/news-releases/fda-grants-regenerative-medicine-advanced-therapy-rmat-designation-to-iregenes-nouvneu001-making-it-the-worlds-first-ipsc-therapy-with-both-ftd-and-rmat-recognitions-302663450.html