iRegene's NouvSight001 Cleared for US and China RP Trials

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

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

What is the unmet need in retinitis pigmentosa?

RP is a group of inherited retinal degenerative diseases that iRegene estimates affects approximately 1.5 million people worldwide, beginning with night blindness and progressing to peripheral vision loss and, in some cases, blindness.¹ A peer-reviewed literature review puts global prevalence at more than 1 million people, or roughly 1 in 4000.³ The only approved gene therapy is restricted to patients with biallelic RPE65 mutations, a small fraction of the RP population,¹ and that review notes no definitive cure for RP yet exists.³ Most patients still have no disease-modifying treatment option.¹

What are the limitations?

Regulatory clearance to begin a trial is not evidence of safety or efficacy; NouvSight001 has not yet been dosed in RP patients, and outcomes from NouvNeu001 in a different disease and tissue do not guarantee similar results in the eye. iRegene did not disclose the scope of the FDA's special exemption or a timeline for enrollment. Orphan drug designation does not guarantee approval, and iRegene, a privately held company, did not disclose trial size, sites, or a data readout date.

References

1. iRegene Therapeutics. iRegene Therapeutics's NouvSight001 cleared for clinical trials in the US and China for retinitis pigmentosa. Press release. Published September 29, 2026. Accessed September 29, 2026. https://www.prnewswire.com/news-releases/iregene-therapeuticss-nouvsight001-cleared-for-clinical-trials-in-the-us-and-china-for-retinitis-pigmentosa-302892416.html
2. iRegene Therapeutics. iRegene Therapeutics announced promising NouvNeu001 Phase I clinical data. Press release. Published October 14, 2025. Accessed September 29, 2026. https://www.iregene.com/#page5
3. Suleman N. Current understanding on retinitis pigmentosa: a literature review. Front Ophthalmol (Lausanne). 2025;5:1600283. doi:10.3389/fopht.2025.1600283