Ruijian Medicine's First-in-Class iPSC-Derived Therapy NouvNeu001 Achieves First Patient Dosing in U.S. Phase II Trial for Parkinson’s Disease

iRegene's NouvNeu001 Injection Achieves Significant Global Clinical Progress.

On February 11, 2026, iRegene completed the first patient dosing in the U.S. Phase II clinical trial of NouvNeu001 injection, a product independently developed by iRegene. NouvNeu001 is the world's first iPSC (induced pluripotent stem cell)-based chemically induced off-the-shelf universal cell therapy product to receive both FDA FTD and RMAT designations, aimed at treating Parkinson's disease.

For Parkinson's disease, the world's second-largest neurodegenerative disease, the current therapeutic positioning is symptomatic treatment or "complication management." These treatments can only alleviate symptoms to a certain extent, unable to regenerate lost neurons, and certainly unable to stop or reverse the neurodegenerative process of the disease. As the condition progresses, patients will eventually face the dilemma of increasingly severe loss of motor function and a sharp decline in quality of life. NouvNeu001 fundamentally repairs the loss of dopaminergic neurons through cell replacement therapy, reversing the progression of Parkinson's disease, thus...Fill the fundamental treatment gap in "disease-reversing" Parkinson's drugs。

With the successful dosing of the first patient in the U.S. Phase II clinical trial, NouvNeu001's global development has entered a critical stage. In the field of neurological diseases, NouvNeu001 has also become the first iPSC-derived off-the-shelf allogeneic cell therapy product to enter an international multicenter Phase II clinical trial. This not only signifies an important breakthrough in the iPSC field, achieving "from 0 to 1," but also further solidifies iRegene's leading international position in this domain.

The global clinical development of NouvNeu001 has seen continuous positive progress, closely related to its tremendous potential in treating Parkinson's disease and recognition from regulatory authorities.

NouvNeu001 is the world's first iPSC-derived, chemically induced universal dopaminergic neural progenitor cell. After transplantation of NouvNeu001 into patients, it restores the missing functional cells at the lesion site, ultimately repairing the circuit loss caused by Parkinson’s disease in the patient’s body and enabling the patient to regain dopamine secretion function.

NouvNeu001 fundamentally repairs the loss of dopaminergic neurons through cell replacement therapy, reversing the progression of Parkinson's disease. This therapy directly targets the core pathological mechanism of Parkinson’s disease — the loss of dopaminergic neurons — and thus holds immense therapeutic potential. This treatment approach, which traditional symptomatic treatments cannot achieve, is a true First-in-Class (FIC) product capable of reversing disease progression.

iPSC-derived neural cell therapy has long been in the early exploratory stage, and there are no projects globally that have actually entered mid-term clinical validation. The therapeutic potential and leading advantage of NouvNeu001 are very evident.

Moreover, the regulatory authorities have also given high recognition to this project.

It is understood that since the initial development of iRegene's NouvNeu001, a normalized and forward-looking collaborative working mechanism has been established with regulatory agencies to continuously transform key R&D milestones into regulatory approvals: In March 2024, NouvNeu001 was the first to receive FDA special exemption, and in June of the same year, it was approved for IND to conduct Phase I clinical trials overseas. Thereafter, based on positive clinical progress, the project received FTD designation in August 2025 and RMAT designation in December 2025.NouvNeu001 has also become the world's first iPSC product to receive both FDA FTD and RMAT designations.

Notably, the NouvNeu001 project received strong support from the U.S. FDA in the third quarter of 2025, allowing it to directly enter Phase II clinical trials in the United States. The first patient dosing for the Phase II trial in the U.S. was conducted at Weill Cornell Medicine, a top-tier medical school under Cornell University, which maintains close collaborations with over 20 international partners across six continents. The support from leading global medical institutions will accelerate the progress of the international multicenter clinical trial for NouvNeu001.

In addition, solid non-clinical data and a comprehensive and robust CMC system are also important reasons for the rapid advancement of the NouvNeu001 project's clinical trials.

NouvNeu001 precisely targets the unmet clinical needs of Parkinson's disease, aiming to fundamentally repair the loss of dopaminergic neurons through cell replacement therapy and reverse the progression of Parkinson's disease. The FDA’s support for NouvNeu001 to directly enter Phase II clinical trials in the United States reflects the regulatory agency's recognition of the quality of the preliminary data and the maturity of the CMC system.

The "AI + Chemical Induction" platform independently developed by iRegene, which possesses independent intellectual property rights, is the key to the successful implementation of this therapy. The platform uses AI to screen small molecule compounds as inducing components, enabling efficient and precise induction of targeted cell differentiation.

iRegene's "AI + Chemical Induction" Platform Has the Following Three Core Advantages: 1)Directional Induction, which can optimize small molecule combinations through algorithms to achieve direct targeted induction of specific midbrain dopaminergic neuron subtypes; 2)Function Optimization, which can synchronously optimize and modify cell functions during the induction process; 3)Quality Controllable, which can establish a more controllable and standardized chemical system, reducing batch-to-batch fluctuations caused by biological factors.

Notably, iRegene's "AI + Chemical Induction" platform acquires functionally enhanced cells through chemical induction rather than gene editing. This technical approach is more efficient, safer, and cost-effective, making it more suitable for large-scale industrial production. Therefore, iRegene's proprietary "AI + Chemical Induction" platform is more conducive to establishing a stable CMC system, enabling both scalable off-the-shelf production and reducing the costs and supply chain pressures associated with personalized customization.

The underlying design of the "AI + chemical induction" platform brings NouvNeu001 closer to the logic of "industrializable cell therapy" in terms of technical pathways. The core intellectual property of this technology platform has already been granted simultaneous authorization in multiple countries, including China, Japan, and the United States.

In addition, the data integrity, the rigor of clinical trials, and the safety and efficacy data demonstrated by iRegene's NouvNeu001 in clinical research have continued to gain recognition from the FDA. Prior to this, NouvNeu001 had already received FTD and RMAT designations. Under the RMAT framework, the project has received ongoing rolling communication with the FDA and potential support for an accelerated approval pathway. This not only signifies that the clinical value of iRegene’s pipeline has been highly recognized by international authoritative regulatory agencies but also fully reflects the strong execution capability of iRegene’s team in conducting international multicenter clinical trials.

The successful dosing of the first patient in the Phase II clinical trial in the United States also marks that the cell transplantation therapy for reversing Parkinson's disease has moved from the "technical validation" stage to the "clinical value validation" stage. With the gradual accumulation of international multicenter data, the clarity of the registration pathway and the visibility of the timeline for the NouvNeu001 project have significantly improved.

NouvNeu001 is the world's first iPSC-derived chemically induced universal dopaminergic neural progenitor cell. Globally, there are no projects that have actually entered mid-term clinical validation, and iRegene is at the forefront of R&D progress in this field.Ranking First Globally。

In fact, the NouvNeu001 project has been in a globally leading position since its initial development. The successful completion of the first international multi-center Phase II overseas dosing for NouvNeu001 further solidifies iRegene's strategic first-mover advantage. Moreover, iRegene's CMC system has passed multiple rounds of FDA reviews and verifications. The smooth dosing in the U.S. Phase II clinical trial also marks that NouvNeu001 has a clear path towards advancing to a pivotal Phase III trial.

This series of regulatory breakthroughs and clinical trial advancements not only highlights the FDA's strong recognition of NouvNeu001's therapeutic potential, iRegene's innovative CMC, and development strategy, but will also further assist iRegene's R&D team in gaining support in areas such as clinical trial design, advancing registration-based Phase III clinical trials with the FDA, and optimizing the market entry process.

Dr. Meng Cai, Chief Medical Officer of iRegene, stated: "The successful dosing of the first participant in the U.S. Phase II clinical trial of NouvNeu001 marks a significant milestone in the development of NouvNeu001, demonstrating the determination and execution capability of iRegene's R&D team in advancing overseas clinical trials. iRegene has established a globally leading strategic position in the frontier field of iPSC-derived off-the-shelf cell therapy for Parkinson's disease, and this progress also provides the company with important experience for further in-depth and efficient international cooperation with regulatory agencies."

According to iRegene, the company will fully leverage the leading and breakthrough advantages of its R&D team in regulatory pathways and global clinical research to accelerate the pace of global multicenter clinical trials, striving to make this revolutionary therapy benefit patients worldwide as soon as possible. At that time, the traditional "complication management"-style treatment model for Parkinson's disease will be fundamentally reversed.