iRegene's NouvNeu001 granted FDA RMAT designation, poised as breakthrough therapy for Parkinson's disease
On December 22, iRegene's self-developed NouvNeu001 injection was granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA. Established by the 21st Century Cures Act in 2016, RMAT provides an expedited approval pathway in the U.S. for regenerative medicine therapies with significant clinical potential.
NouvNeu001 is now the first iPSC product globally to hold both RMAT and Fast Track Designation (FTD) status, the latter having been granted on August 15, 2025. This rapid sequential recognition highlights the FDA's strong confidence in its clinical data.
The core pathological feature of Parkinson's disease is the degeneration and death of dopaminergic neurons in the substantia nigra of the midbrain, leading to motor dysfunctions such as tremor, rigidity, bradykinesia, and gait abnormalities. The condition progressively worsens until patient death. Due to unclear pathogenic mechanisms, Parkinson’s disease remains incurable and irreversible.
As the second most common neurodegenerative disorder globally, current treatments for Parkinson’s disease are merely symptomatic or focused on "complication management." Whether through the temporary relief provided by levodopa or the limited improvement from deep brain stimulation, these approaches can only alleviate symptoms to some extent—they cannot regenerate lost neurons, nor halt or reverse the neurodegenerative progression of the disease. Patients inevitably face functional decline. Long-term medication may also lead to significant side effects; for example, prolonged use of levodopa can cause motor complications and psychiatric hallucinations.
As the disease advances, patients ultimately confront increasingly severe loss of motor function and a sharp decline in quality of life. There is therefore an urgent need for a "disease-modifying" treatment for Parkinson's. The development of NouvNeu001 directly addresses this fundamental therapeutic gap.
NouvNeu001 is the world's first chemically induced allogeneic dopaminergic neuronal precursor cell derived from induced pluripotent stem cells (iPSCs). iPSCs possess self-renewal capacity and the potential to differentiate into nearly all human cell types, enabling the replacement of failing organs and the promotion of tissue regeneration. Through cell replacement therapy, NouvNeu001 fundamentally repairs the loss of dopaminergic neurons and reverses the progression of Parkinson's disease. This therapy directly targets the core pathological mechanism of Parkinson's disease—the deficiency of dopaminergic neurons—thus holding significant therapeutic potential.
Following its FDA Fast Track Designation (FTD) in August 2025, NouvNeu001 promptly received RMAT designation, reflecting the FDA's strong recognition of its Phase I clinical data and its potential value in treating Parkinson's disease. Currently, NouvNeu001 has advanced to Phase II clinical trials globally, placing its development progress at the forefront of similar pipelines worldwide. This achievement further consolidates iRegene's leading position in the field of central nervous system diseases.
Behind the rapid advancement of this innovative therapy lies iRegene's proprietary AI-driven Chemical Induction platform. This platform utilizes AI to screen small-molecule compounds as inducing agents, enabling highly efficient and precise directional differentiation of cells.
iRegene's AI-driven Chemical Induction platform achieves the directed differentiation of highly specific dopaminergic neuronal subtypes with high purity. It concurrently implements various functional modifications—including cellular biochemical properties—during the induction process to meet clinical requirements.
Notably, the platform generates functionally enhanced cells through chemical induction rather than genetic editing. This approach is more efficient, safe, cost-effective, and better suited for large-scale industrial production. This R&D strategy has significantly enhanced cell therapy manufacturing efficiency, and iRegene has already established an industrial production capacity of 360,000 doses.
Following transplantation of NouvNeu001, the iPSC-derived cells replenish the loss of functional cells at the lesion site, ultimately restoring the neural circuitry impaired by Parkinson's disease within the patient's body and enabling the restoration of endogenous dopamine secretion. This treatment represents a disease-modifying therapy unattainable through conventional symptomatic management, qualifying as a truly First-in-Class product.
Furthermore, iRegene has innovated the administration method for NouvNeu001. Distinct from other approaches in the field, the company employs a globally pioneered single-tract stereotactic delivery technique, maximizing procedural safety. This novel administration method also supports the long-term survival and integration of the transplanted cells.
The combination of iRegene's AI-driven Chemical Induction technology platform and its proprietary delivery approach enables the targeted modification and functional enhancement of dopaminergic neuronal therapy. In clinical trials, subjects receiving a single administration of NouvNeu001 demonstrated significant motor function improvement at 12 months post-treatment, as measured by the MDS-UPDRS Part III score: an average reduction of 30.6 points (52.82% improvement) in the OFF state and 13.6 points (54.67% improvement) in the ON state—results that substantially surpass those achievable with existing therapies.
These statistically significant clinical outcomes further validate the technological advantages of iRegene's pioneering chemical induction platform and underscore the potential value of NouvNeu001 in the treatment of Parkinson's disease.
NouvNeu001, developed based on iRegene's unique chemical induction platform, has attracted sustained interest from investment institutions, international pharmaceutical companies, and research organizations.
In October 2025, iRegene delivered an oral presentation on the groundbreaking study of NouvNeu001 for moderate-to-severe Parkinson's disease at the International Congress of Parkinson's Disease and Movement Disorders (MDS Congress). The Congress served as a premier global platform for showcasing frontier research and clinical advances in Parkinson's and related neurodegenerative disorders.
Notably, iRegene's NouvNeu001-related study was selected as the opening presentation of the clinical trials session at the MDS Congress, reflecting the international professional community's recognition of NouvNeu001's pioneering work in Parkinson's disease treatment. Sharing the session with iRegene were innovative pipelines from leading global pharmaceutical companies such as BlueRock, Roche, and Biogen, signifying that Chinese companies have now entered the global forefront in the field of innovative neurological disease therapies.
It is reported that since the inception of NouvNeu001's development, iRegene has established a regular and forward-looking collaborative working mechanism with regulatory agencies, consistently translating key R&D milestones into regulatory recognition. Supported by robust non-clinical data and a comprehensive CMC (Chemistry, Manufacturing, and Controls) system, NouvNeu001 obtained a special exemption from the FDA in March 2024, followed by IND approval in June of the same year to initiate overseas Phase I clinical trials. Subsequently, based on positive clinical progress, the program was granted Fast Track Designation (FTD) in August 2025 and Regenerative Medicine Advanced Therapy (RMAT) designation in December 2025. With this, NouvNeu001 has become the first iPSC-derived product globally to receive both FTD and RMAT designations from the FDA.
The data integrity demonstrated in iRegene's NouvNeu001 clinical studies, the rigor of the trial conduct, and the safety and efficacy outcomes have earned sustained recognition from the FDA. This continuous series of regulatory breakthroughs not only reflects the FDA's acknowledgment of NouvNeu001's therapeutic potential but will also further support iRegene's R&D team in areas such as clinical trial design, advancing Phase III trials, and optimizing the regulatory approval pathway.
Dr. Cai Meng, Chief Medical Officer of iRegene, informed VCBeat that obtaining RMAT designation represents a significant regulatory milestone in the development of NouvNeu001, fully validating its clinical value and innovativeness. Moving forward, iRegene will fully leverage this designation to accelerate global multicenter clinical trials and expedite the product's commercialization process.
An international strategy will further support this goal. It has been revealed that iRegene's Innovation Center in Denmark, Singapore operations office, and overseas clinical center in Australia are already operational. Additionally, the company has established a joint innovation center with Danaher Corporation to collaboratively advance the development and clinical application of novel cell therapies. In preparation for global clinical trials, iRegene has also constructed a large-scale industrial production facility and established logistics capabilities covering China, the United States, and Australia, ensuring a self-controlled supply chain for therapeutic manufacturing.
As sustained long-term investments continue to translate into R&D achievements, iRegene's development pipeline is also steadily expanding.
NouvNeu004, targeting multiple system atrophy (MSA), received a special exemption from the FDA in November of this year. This marks another significant breakthrough following the approval of its Phase I-III full-cycle clinical trial application by the NMPA.
Addressing the trend of earlier-onset Parkinson's disease, iRegene's NouvNeu003 has successfully completed its Phase I clinical trial. It has now become the world's first iPSC-derived universal cell therapy for early-onset Parkinson's disease to advance into pivotal clinical trials
Meanwhile, the breakthrough ophthalmic product NouvSight001 was granted Orphan Drug Designation by the U.S. FDA in March 2024 for its indication targeting retinitis pigmentosa and related conditions.
This ongoing pipeline expansion not only underscores the technological advantages of iRegene's core AI-driven Chemical Induction platform but also further solidifies its leading position in the field of central nervous system diseases. As the flagship product in the pipeline, the FDA RMAT designation for NouvNeu001 marks a major stride toward bringing this first-in-class cell therapy — originated from China — to Parkinson's disease patients worldwide.