iRegene Therapeutics Co., Ltd. has announced its investigational cell therapy NouvSight001 has received clearance from China’s National Medical Products Administration (NMPA) for a phase I/III clinical trial in patients with retinitis pigmentosa (RP) to evaluate its safety, tolerability and preliminary efficacy.
The U.S. Food and Drug Administration (FDA) has also cleared NouvSight001 for a phase I/II clinical trial for the same indication and granted a special exemption.
NouvSight001 is an allogeneic, off-the-shelf photoreceptor cell therapy developed using iRegene’s proprietary “AI + Chemical Induction” (“AI + Chem”) platform. As a cell replacement therapy, NouvSight001 is not limited to patients with a specific disease-causing genotype. The regulatory clearances in the US and China mark another milestone in iRegene’s global product development.
The company has built a regenerative medicine pipeline focused on major diseases associated with the structural burden of an aging global population, including Parkinson’s disease (PD), multiple system atrophy (MSA), retinal degenerative diseases and ischemic stroke. Its pipeline now spans the central nervous system, visual system and neurovascular system.
iRegene’s “AI + Chem” platform is designed to identify and characterise key biological networks that determine cell fate and function, and to precisely regulate these networks using small molecules. Through this approach, iRegene aims to make complex cell differentiation processes more predictable, controllable, reproducible and scalable for therapeutic manufacturing.
As multiple programmes generated from the same platform move into international clinical development, iRegene is beginning to show the platform’s applicability across different cell types and disease areas.
NouvSight001 expands iRegene’s pipeline into retinal regenerative medicine, an area with significant unmet medical need. The programme marks another step toward iRegene’s “one platform, multiple products, global development” model.
Following FDA Orphan Drug Designation in 2024, NouvSight001 has now received clinical trial clearances from both the NMPA and FDA, as well as a special exemption from the FDA.
Although iRegene’s programmes address different diseases, target tissues and clinical settings, they share a common set of underlying capabilities: global regulatory strategy; CMC and quality systems established to meet FDA and NMPA requirements; manufacturing capabilities for allogeneic, off-the-shelf cell therapies; cross-border cold-chain and supply-chain capabilities; clinical development capabilities in the US and China; and an R&D platform designed to continuously generate new therapeutic candidates.
As populations age worldwide and healthcare needs evolve, iRegene said it is focused on the fundamental challenge of restoring cells and tissue functions lost to disease and aging. Rather than developing a single cell therapy, iRegene aims to build a technology platform capable of continuously generating new regenerative medicine products. The company has built a pipeline across several major disease areas. Its programs seek to restore functions lost to disease, including neural circuits and vision, and to repair damaged neural tissue.
Through these programmes, iRegene is building a regenerative medicine portfolio around key functions lost to aging and disease. The company aims to move more of these therapies into global development while building toward commercial scalability.
“Globalisation has been embedded in our R&D system since the company was founded,” said Tianshi Ruan, chief financial officer of iRegene Therapeutics.
“NouvNeu001 has given us hands-on experience advancing an iPSC-derived cell therapy across both the NMPA and FDA regulatory frameworks. NouvSight001 allows us to apply that experience to ophthalmology, a very different disease and tissue setting. The same underlying technology platform allows us to advance our pipeline globally and more efficiently. Going forward, we will continue to advance our core programs in clinical development in the US and China, while actively exploring global multicentre studies and international commercial partnerships.”