iRegene Therapeutics said its investigational cell therapy NouvSight001 has received clearance from China's National Medical Products Administration (NMPA) for a phase 1/3 clinical trial in retinitis pigmentosa (RP) and separately won FDA clearance for a phase 1/2 trial in the same indication plus a special exemption, the company announced September 29, 2026.¹
Key facts
- Drug: NouvSight001 (iRegene Therapeutics)
- Class: Allogeneic, off-the-shelf photoreceptor cell therapy (AI + Chemical Induction platform)
- Indication studied: Retinitis pigmentosa
- Regulatory status: NMPA-cleared phase 1/3 trial in China; FDA-cleared phase 1/2 trial plus special exemption in the US
- Prior designation: FDA orphan drug designation, granted 2024
- Platform validation: NouvNeu001 (Parkinson disease) showed a 52.8% OFF-state UPDRS-III improvement at 12 months in phase 1 data
- Geography: Clinical development underway in the US and China
- Unmet need: Retinitis pigmentosa affects an estimated 1.5 million people worldwide; the only approved gene therapy is limited to RPE65 mutations
The NMPA-cleared trial will evaluate NouvSight001's safety, tolerability, and preliminary efficacy in patients with RP.¹ The FDA clearance authorizes a phase 1/2 study for the same indication, and the agency separately granted NouvSight001 a special exemption.¹ iRegene said the parallel US and China clearances mark another milestone in its global product-development strategy.¹
"Globalization has been embedded in our R&D system since the company was founded," said Tianshi Ruan, chief financial officer of iRegene Therapeutics, in a company press release.¹ "NouvNeu001 has given us hands-on experience advancing an iPSC [induced pluripotent stem cell]-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,” Ruan added in the release. “Going forward, we will continue to advance our core programs in clinical development in the US and China, while actively exploring global multicenter studies and international commercial partnerships."
What is NouvSight001 and how was it developed?
NouvSight001 is an allogeneic, off-the-shelf photoreceptor cell therapy built on iRegene's "AI + Chemical Induction" platform, which identifies biological networks that determine cell fate and uses small molecules to regulate them so that cell differentiation becomes more predictable, controllable, and scalable for manufacturing.¹ As a cell-replacement therapy, it is not restricted to patients with a specific disease-causing genotype, unlike gene therapies that target a single mutation.¹ NouvSight001 extends iRegene's pipeline, which already spans Parkinson disease, multiple system atrophy, and ischemic stroke, into retinal regenerative medicine, giving the Chengdu, China-based company programs across the central nervous, visual, and neurovascular systems.¹
What clinical track record supports the platform?
NouvSight001 follows NouvNeu001, iRegene's iPSC-derived dopaminergic progenitor cell therapy for Parkinson disease, which holds FDA and NMPA clearances of its own.¹ In phase 1 data iRegene reported in October 2025, NouvNeu001's low-dose cohort showed a mean 30.6-point improvement in OFF-state Unified Parkinson's Disease Rating Scale Part III (UPDRS-III) motor scores (52.8%) at 12 months, with the therapy well tolerated and no immunosuppressants required after month 6.² NouvSight001 also follows FDA orphan drug designation the agency granted in 2024.¹
