iRegene Therapeutics Presents Groundbreaking iPSC-Derived Cell Therapy NouvNeu001 as Opening Report at 2025 MDS Congress Clinical Trials Session

From October 5 to 9, 2025, the International Parkinson's Disease and Movement Disorders Congress (MDS Congress) will be held in the United States. As a top international conference in the field of Parkinson's disease and movement disorders, the 2025 MDS Congress will focus on cutting-edge research findings related to Parkinson's disease, movement disorders, and associated neurodegenerative diseases. Covering core topics such as basic research, clinical treatment, and multidisciplinary collaboration, the event provides an excellent platform for global researchers and industry professionals to showcase and exchange the latest advancements in the field.

As a global event, the 2025 MDS Congress has attracted participants from around the world, promoting international scientific research cooperation and experience sharing. The research achievements of Chinese companies in this field have also reached the international stage through this conference, enhancing China's international influence in Parkinson's disease research. On October 6, 2025, iRegene Therapeutics was invited to give an oral presentation at the 2025 MDS Congress on the groundbreaking research involving NouvNeu001, the world’s first iPSC-derived chemically induced universal dopaminergic neural progenitor cell therapy for moderate to severe Parkinson’s disease.

iRegene's unique chemical induction platform, along with the significant therapeutic data from Phase I clinical trials derived from its distinct advantages, has garnered substantial attention from numerous attending clinical experts, investment institutions, international pharmaceutical giants, and research institutes. More importantly, the presentation of iRegene's NouvNeu001-related research as the opening report in the clinical trial session at the International Parkinson and Movement Disorder Conference not only signifies the recognition of iRegene’s work by the International Parkinson and Movement Disorder Society but also highlights the attention and encouragement from international professional bodies towards iRegene's groundbreaking efforts in the field of Parkinson's disease treatment.

During the clinical trial session, Dr. Jun Wei, CEO of iRegene, represented the iRegene team and the clinical trial team in reporting the unique advantages of iRegene’s chemical induction platform. Based on an AI + chemical induction approach to regulate cell differentiation and development, the platform precisely identifies and utilizes specific "chemical small molecule switches" to initiate standardized control processes at each critical node of cell differentiation, achieving accurate control and multiple refined modifications of cell functions.

Notably, although the platform also targets the "cell fate determinants" involved in intercellular transformation, it employs a mechanism similar to chemically targeted drugs, directly regulating the transcription or function of core target genes through compounds. This allows iPSCs to achieve highly efficient and uniform large-scale precise cell conversion in a safer and more convenient manner. This R&D strategy has significantly improved the production efficiency of cell-based therapeutics. For instance, iRegene's Parkinson’s disease pipeline has already achieved an industrial production capacity of 360,000 doses.

Based on iRegene's unique chemical induction platform and optimized clinical strategies, Dr. Jun Wei further introduced the significant breakthrough data from Phase I clinical trials of NouvNeu001. As the world’s first chemically induced, universal cell therapy product to enter clinical trials, NouvNeu001 is an innovative, high-purity, functionally enhanced dopaminergic subtype precursor cell derived from iPSC using iRegene’s proprietary chemical induction system. Not only does NouvNeu001 feature a groundbreaking induction mechanism, but its method of administration is also noteworthy. For the first time internationally, it adopts a "patient benefit prioritized" single-needle stereotactic drug delivery method, significantly reducing surgical risks and related adverse events.

The chemical induction platform has not only demonstrated significant advantages in preclinical research, but its therapeutic benefits have also been further validated in Phase I clinical trials: iRegene's chemical induction platform has safely optimized the immunogenicity of universal cell products. As a result, patients did not experience immune rejection or related adverse events after discontinuing immunosuppressants six months post-surgery. Additionally, mature neuron PET imaging results at the large cohort level further confirmed the excellent long-term survival and stable differentiation integration of NouvNeu001 in the brains of Phase I trial participants.

More importantly, iRegene's chemical induction platform has achieved directional modification and enhancement of the therapeutic function of dopamine neurons. As a result, the MDS-UPDRS Part III scores (motor function scores) of NouvNeu001 subjects showed significant improvement after transplantation: 12 months post-treatment, the UPDRS-III scores improved by an average of 30.6 points in the OFF state (an average improvement of 52.82% from baseline) and by an average of 12.9 points in the ON state (an average improvement of 54.67% from baseline). The improvements in multiple indicators were statistically significant, surpassing international competitors and other treatment options, with a sustained improvement effect observed even after 15 months. Additionally, several non-motor symptoms, such as standardized Good ON Time, also showed significant improvement post-treatment. These statistically significant Phase I clinical data further confirm the unique technical advantages of iRegene's pioneering chemical induction platform.

Notably, as a special session for pharmaceutical companies' clinical trial reports, iRegene shared the stage with heavyweight international pharmaceutical giants such as BlueRock, Roche, and Biogen to present clinical progress. These companies’ innovative products represent the strongest lineup in the field of Parkinson's disease new drug development.

Dr. Meng Cai, Chief Medical Officer of iRegene, also attended the conference during the same period. He discussed various topics related to clinical trials for the development of new Parkinson's disease drugs with many international experts, and explored the global progress and research strategies of well-known pharmaceutical companies in China and abroad regarding disease-modifying therapies for Parkinson's disease.

In terms of global progress, iRegene's NouvNeu001 pipeline is among the first tier of similar pipelines. Globally, NouvNeu001 has received multiple designations from the FDA. First, in March 2024, it received special exemption from the FDA, and on June 2024, its IND was approved by the FDA to initiate an international multicenter Phase I clinical trial in the United States and other countries. On August 15, 2025, NouvNeu001 further received Fast Track Designation (FTD) from the FDA. This is the world’s first iPSC-derived off-the-shelf Parkinson’s cell therapy product to receive FDA Fast Track Designation (FTD).

As NouvNeu001 gains global recognition, iRegene has also embarked on its international expansion. Its innovation center in Denmark, operational office in Singapore, and overseas clinical center in Australia were put into use in 2022. Additionally, iRegene has established a joint innovation center with Danaher Corporation to advance the research and clinical application of AI platform-based chemical-induced cell therapies on a global scale. To prepare for global clinical trials, iRegene has also built a large-scale industrial production base capable of meeting the demand for allogeneic cell drugs across global markets, while possessing logistics capabilities covering multiple regions, including China, the United States, and Australia, ensuring autonomous control over the drug production supply chain.

Multiple advancements combined have made iRegene one of the leading companies in the international pluripotent stem cell field, gaining the most regulatory approvals, having the fastest pipeline progress, and treating the most patients, attracting significant attention from numerous institutions. Just this September, iRegene announced that the company had rapidly completed multiple large financing rounds within a few months, securing over 300 million RMB in total, setting a record for the largest financing amount in China's iPSC sector in recent years. This fully demonstrates investors' high level of recognition and trust in iRegene as an industry leader.

In addition, iRegene is also exploring the other therapeutic potentials of iPSC cell drugs. Its breakthrough ophthalmic product NouvSight001 was granted Orphan Drug Designation (ODD) by the U.S. FDA in March 2024. The product is aimed at treating "a series of retinitis pigmentosa indications" and is the first universal ophthalmic cell therapy product derived from induced pluripotent stem cells (iPSC) developed by iRegene.

Dr. Cai Meng summarized, "This exchange with many international peers on several core topics in the clinical trials of cell therapy for Parkinson's disease has been highly beneficial, and we have also received affirmation from our international counterparts. This is an important milestone event for the iRegene team in advancing international industry progress. International peers and clinical principal investigators (PIs) are very much looking forward to the clinical performance of NouvNeu001 overseas. We will continue to accelerate the completion of Phase II clinical trials and international multicenter Phase I clinical trials for NouvNeu001, steadfastly committed to bringing breakthrough therapies to patients with Parkinson's disease."

With the rapid progress of iRegene's NouvNeu001 pipeline, we look forward to a standardized, freeze-thaw stable, and transportable universal iPSC cell therapy solution for Parkinson’s disease—a condition that has troubled the industry for over two centuries since its discovery. Patients with Parkinson’s disease worldwide will be able to dance the dances they wish to and finish the paintings they long to create. We also anticipate that the "AI + chemical induction" technology platform behind NouvNeu001 will further reshape the landscape of neurodegenerative disease treatment in the future.