World's first MSA cell therapy product clears clinical trial hurdle, a breakthrough for a disease long lacking effective treatments

On October 23, 2025, China's National Medical Products Administration (NMPA) officially approved the I-III phase full-cycle clinical trial application for iRegene's NouvNeu004 injection.

This marks the world's first cell therapy product for Multiple System Atrophy (MSA) to enter the clinical stage, offering new hope for this neurodegenerative disease that has long lacked effective treatments.

Multiple System Atrophy (MSA) is a rapidly progressive neurodegenerative disorder in adults, whose clinical challenge lies in its simultaneous attack on multiple systems including autonomic function and motor coordination. Patients typically present with varying combinations of autonomic failure, parkinsonism, and cerebellar ataxia, resulting in complex clinical manifestations and extremely high diagnostic and therapeutic difficulty.

The disease advances aggressively, with most patients facing life-threatening risks within 6 to 10 years after diagnosis. Currently, there are no approved disease-modifying therapies globally that can delay or halt its progression.

The pathological core of MSA involves abnormal aggregation of α-synuclein in oligodendrocytes, leading to myelin damage and impaired neural transmission. This mechanism fundamentally distinguishes MSA from other neurodegenerative diseases like Parkinson's disease, which explains why conventional neurological treatment strategies often show limited efficacy against MSA.

Current clinical management of MSA primarily relies on symptomatic support measures. However, these approaches often provide limited benefit and can be counteractive: treatments for orthostatic hypotension typically involve plasma volume expansion, wearing compression stockings, and using vasopressors; for parkinsonian symptoms, levodopa-based medications are largely ineffective in most MSA patients; while drugs used to address bladder dysfunction may induce or worsen orthostatic hypotension. This current treatment paradigm, which addresses symptoms without altering the underlying disease progression, highlights the urgent need for novel therapies in the MSA field.

In this context, iRegene's NouvNeu004, as the first cell therapy product for MSA globally to enter the clinical stage, adopts a fundamentally different technical pathway, offering new therapeutic hope for this condition.

Dr. Cai Meng, Chief Medical Officer of iRegene, stated that the clinical approval of NouvNeu004 not only consolidates the company's research and development leadership in neurodegenerative diseases but, more importantly, provides MSA patients with unprecedented treatment options.

iRegene's NouvNeu004 employs an innovative therapeutic strategy combining "neurotrophic support + neural reconstruction." The product comprises chemically induced functionally enhanced neural precursor cells capable of highly efficient secretion of neurotrophic factors. Its innovative dual mechanism of action forms the therapeutic core: on one hand, it provides trophic support to endangered cells in the lesion area, preventing further cell death; on the other hand, it differentiates into functional neural cells through microenvironment induction across multiple lesion sites, achieving systematic neural repair and functional reconstruction.

The clinical trial will be conducted in collaboration with the Rare Neurological Disease Clinical and Translational Center of Beijing Tiantan Hospital, with Professor Wang Yilong, a renowned expert in China's neurological disease field and Executive Vice President of Beijing Tiantan Hospital, serving as the Principal Investigator. This collaboration adheres to a "patient-centered" philosophy, aiming to accelerate new drug development and commercialization while striving to bring new therapeutic hope to MSA patients.

This collaboration represents not only a powerful alliance between clinical and industrial forces, but also a deep integration of medical humanistic care and scientific innovation. With the clinical approval of NouvNeu004, the research center is intensively preparing for trial initiation, with patient recruitment expected to commence nationwide this December.

Underpinning this innovative therapy's rapid transition to clinical application is iRegene's independently developed "AI + Chemical Induction" platform. This platform utilizes artificial intelligence to screen small molecule compounds as inducing components, enabling highly efficient and precise directional cell differentiation with significant technical advantages.

Notably, the platform employs chemical induction rather than gene editing to obtain functionally enhanced cells. This technical approach proves more efficient, safer, lower in cost, and better suited for large-scale industrial production. Taking iRegene's Parkinson's disease pipeline as an example, the company has already achieved an industrial production capacity of 360,000 doses.

The Parkinson's disease treatment product NouvNeu001, developed based on this platform, has provided preliminary proof of concept for this technical pathway through its early clinical data. Phase I clinical data of NouvNeu001 demonstrated that patients exhibited no immune rejection or related adverse events after discontinuing immunosuppressants at the sixth month post-operation. Furthermore, PET imaging results of mature neurons in the large subject cohort confirmed NouvNeu001's excellent capability for long-term survival, stable differentiation, and integration within the brains of Phase I participants.

These positive signals regarding NouvNeu001's safety and feasibility validate the unique advantages of iRegene's pioneering chemical induction platform, while also providing theoretical foundation and research confidence for the therapeutic mechanism of same-platform product NouvNeu004 in the MSA field.

From a broader perspective, the clinical approval of NouvNeu004 is not an isolated incident, but rather a demonstration of iRegene's systematic strategic layout in the field of central nervous system (CNS) diseases.

The company has built a robust product pipeline for Parkinson's disease, underscoring its strong R&D execution capabilities. Its core product, NouvNeu001, is positioned in the global first echelon of comparable pipelines. Progressing from an FDA special waiver in March 2024, to FDA approval in June 2024 for initiating an international multi-center Phase I clinical trial across the United States and other countries, and further to the grant of Fast Track Designation (FTD) in August 2025, it has become the world's first iPSC-derived, off-the-shelf cell therapy product for Parkinson's disease to receive this designation.

Furthermore, addressing the increasing prevalence of Parkinson's disease in younger populations, the company's self-developed NouvNeu003 received approval in China to enter Phase I clinical trials in December 2023. It has become the world's first iPSC-derived, off-the-shelf cell therapy product for early-onset Parkinson's disease to enter the registered clinical stage.

iRegene continues to expand its exploration into ophthalmic diseases. Its breakthrough ocular product, NouvSight001, was granted Orphan Drug Designation by the U.S. FDA in March 2024. Targeting the treatment of "retinitis pigmentosa series indications," this product represents the first iPSC-derived, off-the-shelf cell therapy developed by iRegene for ophthalmic diseases.

Regarding its global footprint, iRegene's Innovation Center in Denmark, operational office in Singapore, and overseas clinical center in Australia commenced operations in 2022. The company has also established a Joint Innovation Center with Danaher Corporation to jointly advance the global development and clinical application of AI-platform-driven chemical induction cell therapies. To support global clinical trials, iRegene has built large-scale industrial production facilities capable of meeting allogeneic cell therapy manufacturing demands for global markets. The company has also established logistics distribution capabilities covering China, the United States, and Australia, achieving autonomous control over its drug production supply chain.

The company's innovative capabilities continue to gain international recognition. At the 2025 International Congress of Parkinson's Disease and Movement Disorders (MDS Congress), iRegene was invited to deliver an oral presentation on the groundbreaking research of NouvNeu001, the world's first iPSC-derived, chemically induced, off-the-shelf dopaminergic neural precursor cell therapy for moderate-to-severe Parkinson's disease. Notably, iRegene shared the presentation session with innovative products from internationally renowned pharmaceutical companies including BlueRock, Roche, and Biogen, signifying that Chinese enterprises have now joined the global forefront in innovative therapies for neurological diseases.

The clinical approval of iRegene's NouvNeu004 represents not only a major breakthrough by a Chinese company in the field of advanced therapies but also signals a turning point in addressing the long-standing lack of effective treatments for MSA patients.

As the clinical trials advance, this product is expected to generate valuable research data and clinical experience for MSA treatment, providing critical references for future development. We anticipate that this innovative therapy may inaugurate a new chapter in the treatment of neurodegenerative diseases, illustrating a clear pathway for Chinese cell therapies to advance toward a more innovative future.