A world first for MSA? iRegene's cell therapy NouvNeu004 enters global trials after FDA clearance

On November 26, 2025, the U.S. Food and Drug Administration (FDA) formally granted a Special Exemption to the NouvNeu004 injection developed by iRegene, and further approved the international Phase I clinical trial of NouvNeu004 injection for the treatment of Multiple System Atrophy (MSA).

Just one month prior, on October 23, 2025, China's National Medical Products Administration (NMPA) officially approved the full-cycle (Phase I-III) clinical trial application for the NouvNeu004 injection. Achieving concurrent approvals from both the Chinese and U.S. regulatory authorities marks a significant acceleration in iRegene's international development strategy.

The rapid advancement of NouvNeu004 injection into clinical trials brings new hope to MSA patients who have long faced a severe lack of treatment options.

Multiple System Atrophy (MSA) is a rapidly progressive neurodegenerative disorder in adults. The disease advances aggressively, with most patients facing life-threatening risks within 6 to 10 years after diagnosis. Clinically, patients typically present with varying combinations of autonomic failure, parkinsonism, and cerebellar ataxia, resulting in a complex condition that poses significant diagnostic and therapeutic challenges.

Currently, there are no approved disease-modifying therapies available worldwide capable of slowing or halting the progression of MSA.

The pathological hallmark of MSA is the abnormal aggregation of α-synuclein within oligodendrocytes, leading to myelin damage and impaired neural transmission, which progressively disrupts multiple critical brain regions. This mechanism fundamentally differs from that of other neurodegenerative diseases, such as Parkinson's disease. Consequently, conventional therapeutic strategies for neurological disorders are often minimally effective against MSA.

To date, there is no cure for MSA. Clinical management focuses on alleviating symptoms and improving quality of life through a multidisciplinary approach that includes pharmacological intervention, rehabilitation, lifestyle management, and psychological support.

Drug therapy for MSA primarily follows individualized regimens targeting specific symptoms, yet these approaches often yield limited efficacy. For orthostatic hypotension, treatment typically involves volume expansion, compression stockings, and vasopressor agents. For parkinsonian symptoms, levodopa-based medications are poorly responsive in most patients, with diminishing benefits over time. Medications for bladder dysfunction may induce or exacerbate orthostatic hypotension. The therapeutic challenges faced by MSA patients highlight an urgent need for novel treatments.

Against this backdrop, iRegene's NouvNeu004 injection—an innovative cell therapy product for MSA developed through a novel multi-target mechanism—brings new hope to this field.

iRegene's NouvNeu004 injection is a chemically induced, functionally enhanced neural precursor cell product that employs an innovative therapeutic strategy combining "neurotrophic support" and "neural reconstruction."

Specifically, the novel multi-target mechanism of NouvNeu004 injection encompasses two key aspects. First, the product exhibits high-efficiency secretion of neurotrophic factors, providing nutritional support to endangered cells in lesion areas to prevent further cell death. Second, it can be induced by the microenvironment to differentiate into functional neural cells at multiple lesion sites, enabling systematic neural repair and functional reconstruction.

The clinical trial of NouvNeu004 injection in China will be conducted at the Clinical and Translational Center for Rare Neurological Diseases at Beijing Tiantan Hospital. The Principal Investigator is Professor Wang Yilong, a renowned expert in the field of neurological diseases in China and the Executive Vice President of Beijing Tiantan Hospital, Capital Medical University. The research center has already initiated nationwide patient recruitment for MSA in China. Additionally, following the approval of its international Phase I clinical trial, the overseas clinical development of NouvNeu004 will also commence.

Supporting the rapid translation of this innovative therapy into clinical application is iRegene's proprietary "AI + Chemical Induction" platform. This platform utilizes AI to screen small-molecule compounds as inducing agents, enabling efficient and precise directional differentiation of cells, offering significant technological advantages.

From its inception, based on differentiated market analysis and the scientific background of its founding team, iRegene established an R&D strategy centered on "AI + Chemical Induction" for precise cellular reprogramming. The chemical induction platform holds broad application potential, not only addressing multiple practical challenges in cell therapy manufacturing but also demonstrating extensive scalability for developing diverse therapeutic modalities. To this end, iRegene independently developed a universal iPSC-derived product development platform based on AI-selected small-molecule compounds as core inducing agents, enabling efficient conversion from iPSCs to functional cell therapies.

This process involves three critical steps. First, leveraging a proprietary computational biology platform, iRegene identifies core driver genes. Second, after determining the key nodes in the cellular conversion network, iRegene employs AI screening to obtain compounds that induce cellular transformation. Third, these compounds are used directly to achieve precise cellular functional reprogramming, allowing for a safer, more convenient, efficient, and uniform large-scale conversion of iPSCs.

Through this approach, using only a few selected compounds combined with a chemically defined basal medium, iRegene can achieve highly efficient and precise differentiation from iPSCs to universal functional cell therapies at an industrial scale, along with targeted cellular functional enhancement.

Notably, this platform obtains functionally enhanced cells through chemical induction rather than gene editing. This technological approach is more efficient, safer, lower in cost, and better suited for large-scale industrial manufacturing. This strategy has significantly enhanced cell therapy production efficiency. For example, iRegene's Parkinson's disease pipeline has achieved an industrial-scale production capacity of 360,000 doses.

Early clinical data from NouvNeu001 injection, a Parkinson's disease treatment product developed on this platform, provide validation for this technological pathway. Regarding safety, 15-month Phase I clinical data for NouvNeu001 showed no immune rejection or related adverse events in patients after discontinuing immunosuppressants six months post-transplantation. In terms of efficacy, the MDS-UPDRS Part III scores demonstrated a faster onset of improvement and a more pronounced magnitude of effect. Additionally, positron emission tomography (PET) imaging of mature neurons in a large cohort of subjects indicated long-term survival and stable differentiation of the transplanted NouvNeu001 cells in patients' brains.

Currently, NouvNeu001 injection has received Fast Track Designation (FTD) from the U.S. FDA, indicating that the clinical value of this product has gained recognition from global regulatory authorities. Moreover, due to its demonstrated breakthrough efficacy in treating Parkinson's disease, the FDA has not only provided tailored regulatory and market approval support for NouvNeu001 but has also agreed to expand its use to other compassionate use scenarios. This significantly extends the product's clinical application value and commercial potential.

The positive clinical data from NouvNeu001 validate the unique advantages of iRegene's pioneering "AI + Chemical Induction" platform while also strengthening the team's confidence in the clinical efficacy of NouvNeu004.

According to data from Pharnexcloud, there are currently no iPSC-derived products in the global clinical pipeline for the indication of Multiple System Atrophy (MSA). The NouvNeu004 injection thus represents the world's first off-the-shelf, iPSC-derived cell therapy to enter clinical development for MSA.

Dr. Wei Jun, CEO of iRegene, stated that the consecutive approvals for clinical trials of NouvNeu004 injection in both China and the U.S., along with the Special Exemption granted by the FDA, not only reinforce iRegene's R&D leadership in cell therapy for central nervous system diseases but also provide an unprecedented therapeutic option for MSA patients.

Beyond NouvNeu004, another key product from iRegene, NouvNeu001, is also highly notable. This product is intended for the treatment of Parkinson's disease and currently holds the most advanced global clinical development status at Phase II, positioning it firmly within the top tier of similar pipelines worldwide. Following the FDA's Special Exemption granted in March 2024, the product was further awarded Fast Track Designation (FTD) by the FDA in August 2025, making it the world's first iPSC-derived, off-the-shelf cell therapy for Parkinson's disease to receive this FTD recognition.

At the International Congress of Parkinson's Disease and Movement Disorders (MDS Congress) in 2025, iRegene presented an oral report on its groundbreaking research into NouvNeu001 for treating moderate-to-severe Parkinson's disease. This report served as the opening presentation in the clinical trials section of the congress, signifying international recognition from professional organizations of iRegene's pioneering work in Parkinson's treatment. It is noteworthy that presentations scheduled alongside iRegene's included innovative products from internationally renowned pharmaceutical companies such as BlueRock, Roche, and Biogen, marking a milestone where a Chinese enterprise has joined the global forefront in the field of innovative neurological disease therapies.

Furthermore, addressing the trend of increasing early-onset Parkinson's disease, iRegene's NouvNeu003 has successfully completed Phase I clinical trials, becoming the world's first iPSC-derived, off-the-shelf cell therapy product for early-onset Parkinson's disease to enter the registration-directed clinical stage. In the ophthalmology field, iRegene's breakthrough product, NouvSight001, received Orphan Drug Designation from the U.S. FDA in March 2024 for retinitis pigmentosa and related indications.

To support its international expansion, iRegene has established an innovation center in Denmark, an operational office in Singapore, and has put its overseas clinical center in Australia into operation. Additionally, iRegene has co-founded a Joint Innovation Center with Danaher to jointly advance the development and clinical application of new products.

iRegene's sustained investment in R&D is now yielding consistent results. The recent approval for the international clinical trial of the NouvNeu004 injection further validates the technological advantages of iRegene's core "AI + Chemical Induction" platform and solidifies its leading position in the field of central nervous system diseases. As clinical trials progress, researchers will gather more clinical data, accumulating invaluable experience for the treatment of MSA.