iRegene Therapeutics' Innovative Ophthalmic Therapy NouvSight001 Receives IND Approvals from Both NMPA and FDA, Validating Global Pipeline Strategy

Chengdu, China / United States, 2026 YearsSeptember 7——iRegene Therapeutics Co., Ltd.(iRegene Therapeutics, hereinafter referred to as "iRegene")Announced Today: The Company’s Independently Developed Innovative Cell Therapy ProductNouvSight001 InjectionApproved by the National Medical Products Administration of China（NMPA）Approval to ConductRetinitis Pigmentosa（Retinitis Pigmentosa, RP）A combined Phase I/III clinical study in patients to systematically evaluate the safety, tolerability, and preliminary efficacy of NouvSight001. Meanwhile,NouvSight001 Injectionalso approved by the U.S. Food and Drug Administration（FDA）Clinical Trial Authorization and Special Waiver（Special Exemption）, approve the Phase I/II overseas joint clinical study for the same indication.

NouvSight001 is iRegene Therapeutics' self-developed“AI + Chemical Induction（AI + Chem）」A universal, off-the-shelf photoreceptor cell therapy product developed by the platform. The successive approvals of clinical trial applications for NouvSight001 by regulatory authorities in China and the United States mark another significant milestone in iRegene Therapeutics’ global product development. As a cell replacement therapy, NouvSight001 is not dependent on specific pathogenic genotypes.

Currently, iRegene Therapeutics has established a long-term strategic layout centered on the structural disease burden brought about by global population aging:# Coverage of Parkinson's Disease（PD）, Multiple System Atrophy（MSA）, major disease areas such as retinal degenerative diseases and ischemic stroke, forming a regenerative medicine product portfolio covering the central nervous system, visual system, and neurovascular system.

The true value of global biotech companies lies not merely in possessing a product capable of entering U.S. clinical trials, but in establishing a system that can consistently advance diverse products through global development.

The core objective of iRegene Therapeutics in establishing its “AI + Chemical Induction” platform is to identify and decipher the key biological networks that determine cell fate and functional formation, and to precisely modulate these networks using small-molecule compounds. Through this technological approach, the company aims to gradually transform the complex process of cell differentiation into a more predictable, controllable, reproducible, and scalable drug manufacturing process. The progression of multiple diverse product candidates into international clinical trials is progressively demonstrating the extensibility of iRegene’s underlying technology platform.

NouvSight001 officially marks iRegene Therapeutics’ entry into the field of retinal regenerative medicine, a disease area with significant unmet clinical needs. Consequently, the approval of NouvSight001 for clinical trials in both China and the United States signifies that iRegene Therapeutics is building its global capability centered on the strategy of “one platform, multiple products, global development.” This represents a core competency essential for a true global Platform Biotech company.

For innovative biotechnology companies, global capability entails more than merely securing overseas Investigational New Drug (IND) applications; it critically depends on the ability to navigate diverse regulatory frameworks and advance products through clinical development. Following its designation as an Orphan Drug by the U.S. Food and Drug Administration (FDA) in 2024, NouvSight001 has subsequently obtained clinical trial approvals from both the National Medical Products Administration (NMPA) and the FDA, along with a special exemption from the FDA. This further demonstrates that iRegene Therapeutics possesses not only the capacity to discover and develop innovative drugs in China, but also the organizational infrastructure to propel its innovative products directly into U.S. clinical development in accordance with global standards.

iRegene Therapeutics’ diverse product portfolio addresses entirely different diseases, target tissues, and clinical application scenarios, yet shares the same foundational capabilities: a global regulatory strategy; CMC and quality systems established in accordance with FDA and NMPA standards; a universal off-the-shelf cell therapy manufacturing platform; cross-border cold chain and supply chain capabilities; clinical execution capacity at sites in both China and the United States; and R&D capabilities for continuously generating new products based on a unified technology platform. iRegene’s globalization has entered the phase of true global clinical execution.（global clinical execution）stage. The ability to continuously supply the same off-the-shelf cell therapy product to clinical centers in both China and the United States, while simultaneously advancing clinical development under two distinct regulatory frameworks, serves as a comprehensive test of a cell therapy company’s capabilities in research and development, manufacturing, quality control, regulatory affairs, and organizational execution.

Global population aging is driving profound and long-term shifts in healthcare needs. Neurodegenerative diseases, retinal degeneration, and cerebrovascular disorders are becoming significant disease burdens affecting the healthspan of the elderly population. iRegene Therapeutics focuses on a more fundamental challenge: how to restore cellular and tissue functions lost during disease progression and aging.

iRegene Therapeutics aims not merely to develop a single cell therapy product, but to establish a technological platform capable of continuously generating novel regenerative medicine products. Currently, iRegene has built a regenerative medicine pipeline covering multiple major disease areas, ranging from the reconstruction of dopaminergic neural circuits and restoration of visual function to the repair of neural and tissue injuries. By focusing on key functions progressively lost during human aging, iRegene is constructing a next-generation portfolio of regenerative medicine products and advancing more candidates with clinical translation potential and scalable commercial viability into global development.

Retinitis pigmentosa (RP) is a group of inherited retinal degenerative diseases, affecting approximately 1.5 million patients worldwide. It typically presents with night blindness and progressively leads to peripheral visual field loss and ultimately blindness. Currently, there is only one approved therapy globally, indicated for patients with biallelic RPE65 gene mutations, which covers a very small proportion of the total RP patient population. The vast majority of patients still lack disease-modifying treatment options.

iRegene Therapeutics（iRegene Therapeutics）iRegene Therapeutics is a platform-based biotechnology company dedicated to developing innovative regenerative medicine products for major age-related diseases worldwide. Centered on its proprietary “AI + Chemically Induced” technology platform, the company integrates artificial intelligence, systems biology, and chemical biology to identify and control key biological regulatory networks that determine cell fate and function, translating these insights into standardized, scalable, off-the-shelf universal cell therapy products.

Mr. Ruan Tianshi, CFO of iRegene Therapeutics, stated: “Globalization is not merely an attempt focused on a single product for iRegene, but rather a long-term strategy embedded in our R&D system since the company’s inception. NouvNeu001 has already helped us establish practical experience in advancing iPSC cell therapy products through both the NMPA and FDA regulatory frameworks, while NouvSight001 further replicates this capability in ophthalmology, a completely different disease and tissue context. From a resource allocation perspective, the ability of the same underlying technology platform to continuously generate candidate products that meet global development standards means that the development and regulatory pathways for each pipeline asset are replicable. Looking ahead, we will continue to advance the clinical development of our core pipelines in China and the United States, while actively exploring global multi-center studies and international commercial collaborations.”

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