HETANG Portfolio Company Ruijian Medicine Receives FDA RMAT Designation for iPSC-Derived Therapy NouvNeu001 in Parkinson’s Disease
iRegene Therapeutics
Cell Therapy Product Developer
On December 22, 2025, NouvNeu001 injection, independently developed by iRegene (Chengdu) Pharmaceutical Technology Co., Ltd. ("iRegene"), an investment company of Hetang Ventures, was granted RMAT designation by the FDA.NouvNeu001 Becomes the World's First iPSC Chemically Induced Universal Cell Therapy Product to Receive RMAT Designation in the Field of Neurological Diseases.
FDA's RMATThe full name is Regenerative Medicine Advanced Therapy (RMAT), an accelerated approval pathway established by the 21st Century Cures Act in 2016. It specifically targets regenerative medicine therapies, focusing on significant clinical value to facilitate their rapid market entry and address unmet medical needs.
NouvNeu001Following the FDA's granting of FTD in August 2025, it quickly obtained RMAT designation,Become the world's first IPSC product to date to receive both FDA FTD and RMAT designations, reflecting the FDA's high recognition of NouvNeu001 clinical data.iRegene's NouvNeu001 Demonstrates Complete Data Integrity, Rigorous Clinical Trials, and Superior Safety and Efficacy in Phase I Clinical Trials for Parkinson’s Disease, Earning High Praise from the FDA. The RMAT designation underscores the significant therapeutic value of this product in the international Parkinson’s disease market. The FDA will further engage in close communication with iRegene to provide customized guidance for subsequent clinical development, including discussions on trial design, selection of clinical endpoints, and accelerated approval pathways, thereby reducing the time from clinical trials to market. The acquisition of RMAT also signifies iRegene's innovative strength in the iPSC field, with the clinical value of its pipeline gaining high recognition from international authoritative regulatory agencies, fully reflecting iRegene's important breakthrough and continued leadership in the iPSC field from "0 to 1".
As the world's second-largest neurodegenerative disease, the therapeutic positioning of existing treatments for Parkinson's disease is either symptomatic or"Complication Management," rather than "disease-modifying" treatment methods. The development of NouvNeu001 directly targets this fundamental therapeutic gap, aiming to fundamentally repair the loss of dopaminergic neurons through cell replacement therapy, reverse the progression of Parkinson's disease, and bring potential clinical benefits that may alter the course of the disease for patients.
iRegene's proprietary intellectual property"AI + Chemical Induction" technology can achieve the directional differentiation of high-purity specific dopaminergic neuron subtypes and simultaneously complete various functional modifications during the induction process, including cellular biochemistry, to meet genuine clinical needs. After transplantation, the cells fill the gap of missing functional cells at the lesion site, ultimately repairing the circuit loss caused by Parkinson's disease in patients and enabling them to regain dopamine self-secretion function. This represents a disease-modifying potential that traditional symptomatic treatments cannot achieve. It belongs to a true First-In-Class technological approach.
iRegene's team adhered to the principle of prioritizing patient benefits from the project's inception. Compared with global peers, iRegene adopts the world's first single-needle stereotactic drug delivery method, achieving maximum safety in the drug delivery process and enabling long-term survival and integration of transplanted cells. Its efficacy data also demonstrate international leadership, with subjects receiving a single dose.After 12 months, the motor function score (MDS-UPDRS III) improved by an average of 30.6 points (52.82% improvement) in the OFF state and by an average of 13.6 points (54.67% improvement) in the ON state, significantly outperforming existing therapies. These statistically significant clinical outcomes demonstrate the substantial therapeutic value of NouvNeu001 in the international Parkinson's disease market.
Regulatory Collaboration Throughout the Entire R&D Cycle
Dr. Meng Cai, Chief Medical Officer of iRegene, stated:"Receiving RMAT designation marks a significant regulatory milestone in the development of NouvNeu001, fully demonstrating its clinical value and innovation. This not only signifies the FDA's recognition of the product’s therapeutic potential for addressing serious unmet medical needs but also provides us with a structured pathway for deeper and more efficient collaboration with regulatory authorities. We will fully leverage this qualification to accelerate global multicenter clinical trials, striving to bring this revolutionary therapy to patients worldwide as soon as possible."
As of now,The R&D progress of NouvNeu001 continues to lead:
On August 3, 2023, NouvNeu001 received approval from China's NMPA to conduct Phase I/II combined clinical trials in China for moderate to severe Parkinson's disease.
In December 2023, NouvNeu001 was officially launched in Phase I clinical trials at the National Center for Geriatric Medicine, Beijing Hospital, Ministry of Health.
In March 2024, the U.S. FDA granted NouvNeu001 special exemption status, recognizing iRegene's breakthrough innovation platform and comprehensive quality system, and exempting NouvNeu001 from related U.S. Pharmacopeia requirements.
In June 2024, the U.S. FDA approved its IND to initiate an international multicenter Phase I clinical trial in the United States and other countries;
In November 2024, NouvNeu001 completed the enrollment for Phase I clinical trial in China;
In April 2025, NouvNeu001 officially launched the Phase II clinical trial in China;
From April to June 2025, iRegene launched the multinational multi-center Phase I clinical trial for NouvNeu001 overseas.
In July 2025, the first group of patients in the Phase II clinical trial of NouvNeu001 completed dosing;
On August 15, 2025, NouvNeu001 was granted Fast Track Designation (FTD) by the FDA.
On December 22, 2025, NouvNeu001 further received FDA Regenerative Medicine Advanced Therapy (RMAT) designation.
Regarding Advanced Regenerative Medicine Therapies (RMAT) Designation
RMAT (Regenerative Medicine Advanced Therapy) is a specialized review pathway established by the U.S. FDA under the framework of the 21st Century Cures Act. It aims to accelerate the development and review of regenerative medicine products used for treating, delaying, reversing, or curing serious or life-threatening diseases. Products granted RMAT designation can benefit from accelerated support measures similar to Breakthrough Therapy Designation (BTD), including earlier and more frequent communication with the FDA, eligibility for rolling review, and the potential for accelerated approval and priority review.
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The content of this article is reproduced fromiRegene Pharmaceutical TechnologyOfficial Account
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