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

Designation underscores potential of chemically induced, off-the-shelf cell therapy to address high unmet need in Parkinson’s disease, following compelling Phase I clinical data

CHENGDU, China, Jan. 19, 2026 /PRNewswire/ — iRegene Therapeutics Co., Ltd. (“iRegene” or the “Company”), a biotechnology company pioneering chemically induced allogeneic cell therapy, today announced that its lead product, NouvNeu001, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of Parkinson’s disease (PD).

This milestone makes NouvNeu001 the first allogeneic iPSC-derived cell therapy globally to hold both FDA Fast Track Designation (FTD) and RMAT designations, following the FTD granted in August 2025. These regulatory recognitions validate the promising clinical data, innovative chemical induction platform, and the therapy’s potential to modify the course of Parkinson’s disease.

The RMAT designation is a special regulatory pathway established by the U.S. FDA under the 21st Century Cures Act, to accelerate the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure serious or life-threatening diseases. Products granted RMAT designation may receive benefits including early, frequent, and close interaction with the FDA, and potential eligibility for accelerated approval pathways and priority review. This collaborative regulatory framework is expected to further shorten the timeline from clinical development to market, supporting iRegene’s mission to bring transformative therapies to patients worldwide.

Dr. Meng Cai, Chief Medical Officer of iRegene Therapeutics, stated:

“The RMAT designation is a pivotal regulatory advancement for NouvNeu001 and a strong endorsement of its clinical value. This designation reflects the FDA’s recognition of NouvNeu001’s potential to address a serious unmet medical need in Parkinson’s disease and provides a structured framework for deep, efficient collaboration with the agency.  We are committed to leveraging this opportunity to accelerate our global clinical development program and bring this transformative therapy to patients as early as possible.”

Addressing a Fundamental Unmet Need in Parkinson’s Disease

Parkinson’s disease is the second most prevalent neurodegenerative disorder globally. Existing therapies primarily focus on symptomatic relief or complication management, rather than modifying or reversing disease progression.

NouvNeu001 was developed to directly address this fundamental treatment gap. As an iPSC-derived, allogeneic dopaminergic progenitor cell therapy, it is designed to replace lost dopaminergic neurons, restore disrupted neural circuits, and enable patients to regain endogenous dopamine production. By targeting the root cause of neuronal degeneration, NouvNeu001 represents a first-in-class, disease-modifying approach with the potential to fundamentally change the PD treatment standard.

Compelling Efficacy: Translating Cellular Precision into Clinical Improvement

The ultimate validation of iRegene’s platform lies in its ability to meaningfully improve patients’ lives. The Phase I data revealed significant clinical efficacy, including marked improvements in MDS-UPDRS Part III motor scores, the gold standard for assessing motor function in PD.

The results were robust: the low-dose cohort showed improvements of 30.6 points (OFF, a 52.82% improvement from baseline) and 12.9 points (ON, a 54.67% improvement from baseline) at 12 months; similarly, the high-dose cohort showed improvements of 23.3 points (OFF) and 9.67 points (ON) at 9 months.

These improvements were statistically significant, and most encouragingly, continued improvement was observed through 15 months post-treatment, suggesting the sustained benefit of the engrafted cells.

A Growing Pipeline: A Commitment to Patients Across Disease States

iRegene’s vision extends beyond a single product. The company is committed to leveraging its unique “AI + Chemical Induction” platform to build a robust pipeline of universal iPSC-derived therapies for currently “incurable” diseases.

Building on the success of NouvNeu001, iRegene is advancing a growing pipeline featuring:

• NouvNeu004: Recently received I-III all-stage IND approval from the NMPA, making it the world’s first cell therapy product for Multiple System Atrophy (MSA) and iRegene’s third self-developed product to enter clinical trials.
• NouvNeu003: The company’s second product targeting early-onset Parkinson’s disease, which entered Phase I trials in December 2023.
• NouvSight001: A breakthrough ophthalmology product that was granted Orphan Drug Designation (ODD) by the U.S. FDA in March 2024 for retinal degenerative diseases.

These expanding pipelines underscore iRegene’s long-term commitment to patients and the company’s confidence in its platform to generate a new generation of transformative therapies – bringing scalable, safe, and effective cell treatments to those who need them most. 

About iRegene Therapeutics

Founded in 2017 by a team of international professionals, iRegene Therapeutics is the world’s earliest biotech to apply ” Chemical induction” to precisely reprogram cell fate and optimize cellular functions for their innovative cell therapy products. Leveraging this breakthrough platform, iRegene has built a robust pipeline targeting currently “incurable” diseases including Parkinson’s disease (PD) and blindness. As a leader in “chemical induction”, iRegene’s proprietary system enables the efficient easy generation of human specific cell types with high purity, and enhanced cellular functionality, pioneering the next generation of chemically derived cell therapies.

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SOURCE iRegene Therapeutics Co., Ltd