NouvSight001 – First Cell Therapy for Retinitis Pigmentosa Approved in China and U.S.

NouvSight001 — a human iPSC-derived retinal progenitor cell therapy for retinitis pigmentosa — has received both NMPA IND approval in China and FDA IND clearance, marking the first cell therapy for RP with dual regulatory authorization.

Introduction

Retinitis pigmentosa (RP) is a group of inherited retinal dystrophies characterized by progressive degeneration of photoreceptors, leading to night blindness, visual field constriction, and ultimately legal blindness. With an estimated prevalence of 1 in 4,000 worldwide, RP is one of the most common inherited retinal diseases and a leading cause of blindness in working-age adults.

Despite decades of research, no approved regenerative therapy exists for RP. Current treatments are limited to symptomatic management, vitamin A supplementation, and, in some cases, retinal implants for end-stage disease.

NouvSight001, a human iPSC-derived retinal progenitor cell therapy developed by Regenerative Medicine (睿健医药), has received both NMPA IND approval and FDA IND clearance, making it the first cell therapy for RP with dual regulatory authorization in China and the United States.

The NouvSight001 Therapy

NouvSight001 consists of retinal progenitor cells derived from clinical-grade human induced pluripotent stem cells (iPSCs). These cells are designed to:

• Differentiate into photoreceptors — replacing degenerated photoreceptors in the retina
• Support surviving retinal cells — secreting neurotrophic factors that promote survival and function
• Modulate the retinal microenvironment — reducing inflammation and oxidative stress

The cells are surgically transplanted into the subretinal space, where they integrate with the host retina and restore photoreceptor function.

Regulatory Milestones

NouvSight001 has achieved two significant regulatory milestones:

1. NMPA IND Approval

China's Center for Drug Evaluation (CDE) approved the IND application for NouvSight001, authorizing a Phase I/IIa clinical trial for the treatment of retinitis pigmentosa. This is the first cell therapy for RP to receive clinical trial approval in China.

2. FDA IND Clearance

Following the NMPA approval, the U.S. Food and Drug Administration (FDA) also cleared the IND application, allowing the same Phase I/IIa trial to be conducted in the United States. This makes NouvSight001 the first iPSC-derived retinal progenitor cell therapy with regulatory authorization from both China and the U.S.

This dual regulatory strategy enables:

• Accelerated global development — simultaneous clinical trials in two major markets
• Regulatory validation — FDA clearance confirms the quality and safety of China-developed manufacturing
• Broader patient access — patients in both China and the U.S. can participate in clinical trials

Significance for RP Patients

For patients with retinitis pigmentosa, NouvSight001 offers:

• First regenerative therapy — no approved regenerative therapies currently exist for RP
• Potential for disease modification — not just symptom management but actual photoreceptor replacement
• Global accessibility — clinical trials in both China and the U.S.
• China-developed innovation — demonstrates the quality of China's iPSC and cell therapy platforms