First-in-Class Domestic Parkinson's Cell Therapy NouvNeu001 Receives FDA Fast Track and RMAT Dual Designations

On December 24, iRegene announced that its independently developed NouvNeu001 injection had been granted by the FDA on December 22.Advanced Regenerative Medicine Therapies（RMAT）Recognition. In August this year, the FDA granted the drug Fast Track Designation (FTD) for the treatment of Parkinson's disease. According to the news release from iRegene, NouvNeu001 is so far...The World's First IPSC Product to Receive Dual FDA FTD and RMAT Designations。

RMAT stands for Regenerative Medicine Advanced Therapy, an accelerated approval pathway established by the 21st Century Cures Act of 2016. It is specifically designed for regenerative medicine therapies, focusing on significant clinical value to facilitate their rapid market entry and address unmet medical needs.

Screenshot source: Official WeChat account of the company

As the world's second-largest neurodegenerative disease, the current therapeutic positioning for Parkinson's disease focuses on symptomatic relief or "complication management," rather than "disease-modifying" treatments. The development of NouvNeu001 directly addresses this fundamental treatment gap.Aiming to fundamentally repair the loss of dopaminergic neurons through cell replacement therapy and reverse the progression of Parkinson's disease., bringing clinical benefits that may alter the course of the disease for patients.

On October 6, 2025, iRegene presented an oral report at the 2025 International Parkinson's Disease and Movement Disorders Congress (MDS Congress) on the groundbreaking research of NouvNeu001, the world’s first iPSC-derived chemically induced universal dopaminergic neural progenitor cell therapy for moderate to severe Parkinson's disease.

NouvNeu001 Subjects Show Significant Improvement in MDS-UPDRS Part III Scores (Motor Function Scores) Post-Transplantation: At 12 Months Post-Dosing, UPDRS-III Scores Improved by an Average of 30.6 Points in the OFF State (Representing a 52.82% Improvement from Baseline) and by an Average of 12.9 Points in the ON State (Representing a 54.67% Improvement from Baseline). Improvements in Multiple Indicators Were Statistically Significant, Outperforming International Competitors and Other Treatment Options, with a Sustained Improvement Effect Observed Even After 15 Months.

In addition, standardized Good ON Time and multiple other non-motor symptoms also showed significant improvement after treatment.

In addition to iRegene's NouvNeu001, there are other stem cell therapies with relatively rapid global progress currently.Bayer's BRT-DA01, Shize Bio's XS411, and Zhongsheng Suyuan's NCR201。

Sina Medicine Comprehensive

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