#China #Chengdu #Therapeutics #NouvNeu001 #iRegene

iRegene Therapeutics Presents Groundbreaking Results from NouvNeu001 Trials

On October 13, 2025, iRegene Therapeutics Co., Ltd. revealed promising findings from the Phase I clinical trials of their innovative product, NouvNeu001, at the International Congress of Parkinson's Disease and Movement Disorders (MDS). As a leading player in the biotech landscape, iRegene is pioneering chemically induced allogeneic cell therapies, specifically targeting Parkinson's disease (PD).

NouvNeu001 holds the distinction of being the first induced pluripotent stem cell (iPSC) therapy to obtain Investigational New Drug (IND) approval from both the National Medical Products Administration (NMPA) in China and the Food and Drug Administration (FDA) in the United States for the treatment of PD. The data presented demonstrates notable safety and tolerability, with results showcasing excellent outcomes over a follow-up period of 15 months post-transplantation.

One of the most significant aspects of the study was the lack of need for immunosuppressants after six months, which marks a pivotal advancement in treatment protocols. NouvNeu001 was administered through a stereotactically guided procedure, involving bilateral injections into the posterior putamen, which allowed for a single trajectory per hemisphere. This methodological approach ensures a reduction in surgical risks while maintaining efficacy.

PET imaging further validated the long-term engraftment, survival, and maturation of the transplanted cells. The Phase I trial results highlight impressive clinical efficacy, especially in motor function improvements, as measured by the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III.

In the low-dose cohort, participants experienced an average reduction of 30.6 points in the OFF state, illustrating a 52.82% improvement from baseline measures, and a 12.9-point reduction in the ON state, corresponding to a 54.67% enhancement. Similarly, the high-dose cohort also demonstrated significant improvements with reductions of 23.3 points (OFF state) and 9.67 points (ON state) observed at nine months after treatment. Notably, these improvements were both statistically significant and sustained over the 15-month assessment.

Dr. Meng Cai, Chief Medical Officer at iRegene Therapeutics, expressed his enthusiasm regarding the Phase I outcomes. He stated, "The encouraging results from the Phase I trial of NouvNeu001 can be attributed to iRegene's unique chemical induction platform and optimized clinical strategies. We utilize a proprietary chemical inducer cocktail to generate high-purity A9 dopaminergic progenitor cells with enhanced functionality."

Dr. Cai emphasized the significance of the surgical method, designed specifically to minimize potential risks and enhance patient safety. The innovative approach underscores iRegene's commitment to developing effective treatment protocols for complex neurological disorders.

The iRegene CEO, Dr. Jun Wei, echoed Dr. Cai's sentiments and underscored the company's dedication to advancing its research. He noted, "The iRegene team is greatly encouraged by this important milestone. We remain fully committed to progressing through the initial clinical phases and undertaking the international multi-center Phase I trial, striving to deliver transformative therapies to those affected by Parkinson's disease."

NouvNeu001's development narrative highlights its milestones: in August 2023, the product received IND clearance from China to initiate Phase I/II trials, followed by FDA clearance in June 2024. Further emphasizing its innovative nature, the product was designated Fast Track Status by the U.S. FDA on August 15, 2025, marking NouvNeu001 as the first allogeneic iPSC-derived PD cell therapy to receive such recognition. This achievement follows the FDA's Special Exemption granted in March 2024, affirming the integrity of iRegene's clinical data, product quality, and revolutionary approach to treatment.

Founded in 2017 by a diverse group of international professionals, iRegene Therapeutics is at the forefront of biotechnology, with a focus on leveraging chemical induction techniques to optimize cellular functions and reprogram cell fate. As a pioneer in this emerging field, iRegene is dedicated to building a robust pipeline aimed at addressing currently incurable diseases, including Parkinson's disease and vision impairment. The successful outcomes from NouvNeu001 indicate a bright future for patients seeking effective treatment options against Parkinson's disease.