The cell therapy field has long held a transformative ambition: to create “off-the-shelf” treatments that can reach not just dozens, but thousands of patients. However, translating brilliant science into a commercially viable medicine has been the field’s paramount challenge. Many platforms, reliant on complex genetic engineering or laborious processes, face a ‘scalability gap’—where the very steps that make a therapy potent in the lab become barriers to producing it at scale affordably.
This gap is precisely what iRegene was founded to bridge, and the company’s progress is already being recognized. Its lead product, NouvNeu001, is the first iPSC-derived therapy to receive IND approval from both the NMPA and the FDA for Parkinson’s disease. This pioneering status was further solidified when, on August 15, 2025, NouvNeu001 was granted Fast Track Designation (FTD) by the U.S. FDA, making it the first allogeneic iPSC-derived PD cell therapy worldwide to earn this recognition.
From its inception, iRegene built its company on chemical induction not only for its biological elegance but because the team believed it was the only viable path to a therapy that could one day treat millions.
Inherent Scalability: Engineered for the Many
At the heart of iRegene’s platform is chemical induction, where proprietary compounds regulate transcription to guide cells to their new identity. This process enables highly efficient, synchronous conversion. The operational workflow is streamlined and inherently scalable, as it avoids the complex cloning and viral steps of genetic engineering, relying instead on a controlled biochemical process.
This scalable foundation isn’t a feature iRegene added later; it’s the core the company started with. It enables a streamlined, cost-effective path from the lab to commercial manufacturing, dramatically shortening production timelines and reducing the cost of goods—ultimately paving the way for “off-the-shelf” therapies to become accessible to millions.
Safety by Design: A Holistic Approach from Vial to Operating Room
iRegene’s commitment to safety is evident in the recent Phase I clinical data for NouvNeu001, presented at the International Congress of Parkinson’s Disease and Movement Disorders (MDS). The therapy demonstrated excellent safety and tolerability for up to 15 months post-transplantation, notably without the use of immunosuppressants after the sixth month. PET imaging confirmed the long-term engraftment, survival, and maturation of the transplanted cells.
This safety profile is by design. While powerful, the common use of permanent genomic edits (GMO) introduces long-term theoretical risks. iRegene’s non-GMO approach uses small molecules to guide cell transformation, which are then thoroughly washed away. This eliminates a lifetime of theoretical risks by leaving no permanent genetic footprint, thereby reducing potential immunogenicity and tumorigenicity risk.
The benefits of this high-purity, functionally enhanced cell product extend beyond the vial and into the operating room.
Because iRegene generates high-purity A9 dopaminergic progenitor cells, a minimally invasive surgical procedure is possible. NouvNeu001 was administered via a stereotactically guided procedure, with bilateral injections into the posterior putamen through a single injection trajectory per hemisphere.
This elegant approach stands in contrast to more complex multi-trajectory procedures. By significantly reducing the number of times the surgical needle passes through brain tissue, iRegene’s protocol directly minimizes potential surgical risks, trauma, and procedure-related adverse events, contributing to the outstanding safety profile observed in the trial.
Compelling Efficacy: Translating Cellular Precision into Clinical Improvement
The ultimate validation of iRegene’s platform lies in its ability to meaningfully improve patients’ lives. The Phase I data revealed significant clinical efficacy, including marked improvements in MDS-UPDRS Part III motor scores, the gold standard for assessing motor function in PD.
The results were robust: the low-dose cohort showed improvements of 30.6 points (OFF, a 52.82% improvement from baseline) and 12.9 points (ON, a 54.67% improvement from baseline) at 12 months; similarly, the high-dose cohort showed improvements of 23.3 points (OFF) and 9.67 points (ON) at 9 months.
These improvements were statistically significant, and most encouragingly, continued improvement was observed through 15 months post-treatment, suggesting the sustained benefit of the engrafted cells.
A Growing Pipeline: A Commitment to Patients Across Disease States
iRegene’s vision extends beyond a single product. The company is committed to leveraging its unique “AI + Chemical Induction” platform to build a robust pipeline of universal iPSC-derived therapies for currently “incurable” diseases.
Building on the success of NouvNeu001, iRegene is advancing a growing pipeline featuring:
- NouvNeu004: Recently received I-III all-stage IND approval from the NMPA, making it the world’s first cell therapy product for Multiple System Atrophy (MSA) and iRegene’s third self-developed product to enter clinical trials.
- NouvNeu003: The company’s second product targeting early-onset Parkinson’s disease, which entered Phase I trials in December 2023.
- NouvSight001: A breakthrough ophthalmology product that was granted Orphan Drug Designation (ODD) by the U.S. FDA in March 2024 for retinal degenerative diseases.
These expanding pipelines underscore iRegene’s long-term commitment to patients and the company’s confidence in its platform to generate a new generation of transformative therapies – bringing scalable, safe, and effective cell treatments to those who need them most.