iRegene Doses First U.S. Patient with NouvNeu001 for Parkinson's, Enrolls First MSA Patient in China

iRegene Therapeutics has dosed the first U.S. patient in a Phase IIa trial of NouvNeu001 for Parkinson's disease at Weill Cornell Medical Center and enrolled the first patient in a randomized controlled clinical trial in China evaluating NouvNeu004 for Multiple System Atrophy (MSA). 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.

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 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.

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 the Chief Medical Officer of iRegene Therapeutics.

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.