iRegene Hits Dual Global Firsts: U.S. Patient Dosed in Phase IIa for NouvNeu001 in Parkinson's and First Enrollment in China's Randomized MSA Trial for NouvNeu004

CHENGDU, China, Feb. 23, 2026 /PRNewswire/ -- The global race to treat neurodegenerative diseases has reached a new tempo as iRegene Therapeutics pushes its cell therapy pipeline into key clinical stages in the U.S. and China. Concurrently, the company's "AI + Chemical Induction" platform saw its first U.S. patient dosed for Parkinson's disease at Weill Cornell Medical Center and its first patient enrolled for Multiple System Atrophy (MSA) at Beijing Tiantan Hospital. These parallel milestones for NouvNeu001 and NouvNeu004 represent the next phase of iRegene's global strategy, transitioning from early safety data into mid-stage clinical trials designed to rigorously evaluate therapeutic efficacy across international patient cohorts.

First Patient Dosed in U.S. Phase IIa Trial of NouvNeu001 for Parkinson's Disease

NouvNeu001 is an investigational off-the-shelf cell therapy derived from induced pluripotent stem cells (iPSCs) and chemically induced into dopaminergic progenitor cells. The therapy was administered via stereotactic neurosurgery into the bilateral putamen at Weill Cornell Medical Center in the first patient of an open-label U.S. Phase II study.

Building on positive Phase I data, the U.S. Food and Drug Administration (FDA) cleared NouvNeu001 for direct Phase II entry in the Q3 2025, following Fast Track Designation (FTD) and Regenerative Medicine Advanced Therapy (RMAT) designation. This progression validates iRegene's preclinical and clinical package and robust CMC framework, enabling closer FDA collaboration and accelerated pathways.

NouvNeu001 is designed to address the underlying pathology of Parkinson's disease by replacing lost dopaminergic neurons and modifying disease progression — a "single dose, sustained benefit" approach aimed at addressing critical unmet needs. With the first patient dosed in U.S., parallel Phase II trials continue in both the U.S. and China.

First Patient Enrolled in China for MSA Trial with NouvNeu004

In parallel, iRegene has enrolled the first patient in a randomized controlled clinical trial in China evaluating NouvNeu004, its allogeneic iPSC-derived off-the-shelf cell therapy candidate for Multiple System Atrophy (MSA). Led by Principal Investigator Professor Yilong Wang at Beijing Tiantan Hospital, Capital Medical University, the study marks a significant advancement for MSA – a rapidly progressive neurodegenerative disorder with no approved disease-modifying therapies.

NouvNeu004 employs a dual "neurotrophic support + neural reconstruction" strategy, providing trophic support to affected brain regions while promoting functional neural differentiation to potentially halt or reverse progression. China's NMPA approved the integrated Phases I–III trial application in October 2025, while the U.S. FDA has granted a special exemption and cleared an international Phase I trial, enabling efficient simultaneous development in both countries.

"Dosing the first U.S. patient for Parkinson's disease and enrolling the first MSA patient in China are pivotal milestones in our mission to deliver transformative, disease-modifying therapies to patients worldwide," said Dr. Meng Cai, Chief Medical Officer of iRegene Therapeutics.

"These parallel advances reflect the productivity of our 'AI + Chemical Induction' platform, yielding two pioneering 'off-the-shelf' cell therapies: NouvNeu001 for Parkinson's disease and NouvNeu004 for Multiple System Atrophy. Together, they demonstrate our commitment to a robust global development pathway and our continued contribution to the advancement of cell therapy for neurodegenerative diseases."

The "AI + Chemical Induction" Advantage: From Proof-of-Concept to Industrial Validation

iRegene's core strategy centers on its proprietary "AI + Chemical Induction" platform, engineered to bring scalability and consistency to cell therapy manufacturing. While traditional differentiation methods frequently rely on complex growth factors and commercial media cocktails – which are often prone to batch-to-batch variability and high costs – iRegene utilizes algorithm-optimized small molecule combinations to direct cellular differentiation toward specific lineages.

This chemical induction approach is designed to reduce batch-to-batch variability and support consistent manufacturing under a stable CMC framework, facilitating true off-the-shelf production at lower costs with reduced supply chain complexity compared to personalized or growth factor-dependent therapies. The platform's foundational technology has been granted patents across China, Japan, and the U.S.

Building on this foundation, iRegene is developing a pipeline of universal, iPSC-derived cell therapies for currently incurable diseases, led by its flagship program and several novel candidates:

• NouvNeu001 for Parkinson's disease – The world's first clinical-stage, allogeneic iPSC-derived, chemically induced dopaminergic progenitor cell therapy for PD; holds dual FDA FTD (August 2025) and RMAT (December 2025) designations; IND cleared in both China and the U.S.; now in Phase II trials in both China and the U.S.
• NouvNeu003 for early-onset Parkinson's disease – The company's second product candidate. IND approved by China's NMPA (December 2023); Phase I completed in China.
• NouvNeu004 for Multiple System Atrophy – World's first "AI + Chemical Induction"-derived cell therapy to enter clinical development for MSA; IND approved by China NMPA covering Phases I–III; U.S. FDA special exemption and international Phase cleared; first patient enrolled in China randomized trial.
• NouvSight001 for Retinal Degenerative Diseases (RDDs) – U.S. FDA Orphan Drug Designation granted (March 2024); advancing as an innovative ophthalmology therapy.

This expanding pipeline underscores the versatility of iRegene's underlying technology, demonstrating the platform's capacity to deliver a new generation of scalable cell therapies for complex neurodegenerative disorders. As these candidates move into advanced clinical stages, they represent a tangible shift toward establishing chemically induced iPSC-derived treatments as a safe and effective pillar of global regenerative medicine.

About iRegene Therapeutics

Founded in 2017 by a team of international professionals, iRegene Therapeutics is a pioneer in applying "Chemical Induction" to precisely reprogram cell fate and optimize cellular functions for innovative cell therapies. Leveraging its proprietary "AI + Chemical Induction" platform, iRegene has built a robust pipeline targeting currently incurable diseases, including Parkinson's disease and Multiple System Atrophy.

SOURCE iRegene Therapeutics Co., Ltd.