Dual IND Approvals in China and the US! Off-the-Shelf Cell Therapy Breaks the Deadlock in Ophthalmic Treatment

Surprises in the cell therapy sector are endless.

Just yesterday (September 7, 2026), domestic cell therapy company

iRegene Therapeutics Co., Ltd.(hereinafter referred to as “iRegene”) unveiled a highly significant industry milestone: NouvSight001, a Self-Developed Ophthalmic Cell Therapy Product, Officially Secures Dual IND Approval in China and the US.

Many people have become accustomed to news of new drug approvals and clinical trial authorizations, viewing this as just another routine pipeline update.

But having been deeply engaged in the field of medical innovation for many years, I must say that this breakthrough is entirely different.

It is not merely a simple overseas product launch, but It has thoroughly broken through the limitations of traditional ophthalmic gene therapies, opening up a novel treatment pathway for millions of patients with retinitis pigmentosa (RP).

In recent years, iRegene has secured clinical trial approvals in both China and the United States for multiple pipelines, ranging from the Parkinson’s disease candidate NouvNeu001 and the multiple system atrophy candidate NouvNeu004 to the ophthalmology candidate NouvSight001. This achievement charts a growth path characteristic of a platform-based biotech company, marking a substantial leap in core competitiveness from the internationalization of a single product to the globalization of its entire technology platform.

Let’s start by discussing a harsh reality.

Retinitis Pigmentosa (RP) is one of the most common hereditary degenerative diseases of the fundus, affecting 1.5 million patients worldwide.

The torment of this disease is a gradual, insidious erosion of hope.

Initially, patients experience night blindness and difficulty seeing in the dark. As the disease progresses, peripheral vision deteriorates continuously, and the photoreceptor cells in the fundus undergo persistent apoptosis and atrophy. In the middle to late stages, the vast majority of patients suffer a sudden and severe decline in visual acuity, ultimately resulting in complete blindness.

What is even more disempowering is that, In the face of this slow "process of blindness," there are virtually no effective curative treatments available in clinical practice.

Many people assume that the widespread adoption of gene therapy will provide a cure for this type of eye disease. However, the actual clinical reality is far more severe than public perception suggests.

Currently, the only approved gene therapy for RP worldwide is Luxturna, but its target population is extremely narrow, limited to patients carrying biallelic RPE65 gene mutations.

This group accounts for a negligible proportion of all patients with retinitis pigmentosa.

In other words, more than 90% of patients lack matching gene mutation targets and are completely excluded from gene therapy.

For a long time, these patients could only rely on conservative measures such as vitamin A preparations, nutritional management, and low-vision rehabilitation to barely maintain their condition. These approaches do not constitute true treatment; rather, they serve merely to “delay the inevitable,” failing to halt the apoptosis of photoreceptor cells or reverse the trajectory of vision loss.

Why Gene Therapy Fails to Save Most Patients? The Limitations of Its Core Logic Are Inherent from the Outset.

The essence of gene therapy is "defect correction": exogenous supplementation of normal genes to repair the lesion where a patient’s gene is defective. This means that, It must precisely match the type of genetic mutation, with an extremely low tolerance for error.

iRegene’s core breakthrough this time lies in completely breaking free from this constrained logic.

NouvSight001, as a universal off-the-shelf photoreceptor cell therapy product, follows a completely different "replacement and regeneration" approach.

It does not require screening for patients’ genetic mutations and is not limited by genotype; its core principle is straightforward: Direct transplantation of healthy, functional photoreceptor precursor cells to replace apoptotic and necrotic photoreceptor neurons in the fundus.

This represents a fundamental distinction from traditional ophthalmic treatments.

Previous retinal pigment epithelium (RPE) cell products and pharmacological interventions have focused on “protecting residual cells” to delay damage as much as possible, whereas NouvSight001 aims to “restore cellular function,” directly replenishing the lost photoreceptive capacity in the fundus.

In addition, this product boasts two particularly compelling clinical advantages:

First, Ultra-low immunogenicity, universal off-the-shelf. No need to combine with oral immunosuppressants in clinical practice, significantly reducing treatment risks and patient burden;

Second, Broad-Spectrum Adaptability. Without relying on pathogenic gene mutations, it can theoretically cover the vast majority of patients with retinitis pigmentosa.

Notably, it is the first photoreceptor precursor cell product derived from chemical induction to be accepted by China’s Center for Drug Evaluation (CDE). Globally, the only competitor in this space is OpCT‑001, owned by Bayer. Leveraging iRegene’s proprietary “AI + Chemical Induction” platform, this product boasts significant differentiated technological advantages.

The simultaneous initiation of clinical trials in China and the United States aims primarily to verify safety and tolerability, while also capturing preliminary efficacy signals.

If clinical development proceeds smoothly, the long-standing industry dilemma of “limited genotyping and lack of therapeutic options for most patients” will be completely resolved.

Notably, the domestic Phase I/III clinical trial of NouvSight001 is led by Professor Su Ruifang from Peking Union Medical College Hospital. As a leading team in the field of hereditary retinal diseases in China, Professor Su’s research group has been dedicated to this field for over three decades, accumulating nearly 10,000 family samples of hereditary retinal degeneration and completing genetic analysis for more than 5,000 patients, thereby establishing the true mutation spectrum of retinitis pigmentosa in the Chinese population.

Professor Sui Ruifang’s team has established a comprehensive clinical evaluation system for inherited eye diseases, possesses extensive practical experience in conducting multiple first-in-human clinical studies on ophthalmic gene therapy, and specializes in refined endpoint assessments of retinal structure and visual function.

For photoreceptor cell transplantation therapy, the core challenge in clinical trials lies in objectively determining whether the transplanted cells survive and whether retinal function can be restored. This assessment capability is precisely the key guarantee for detecting effective signals in the trial. Leveraging solid international academic expertise, the team is better positioned to coordinate research designs between China and the United States, providing strong support for the synergy of joint Sino-US clinical protocols.

What’s even more promising is that RP is merely its first indication.

Looking at the global market, diabetic retinopathy and age-related macular degeneration each affect a massive patient population exceeding 100 million. Currently, mainstream clinical treatments, including anti-VEGF drugs and laser therapy, can only inhibit neovascularization and slow disease progression, but cannot prevent irreversible neuronal necrosis and permanent vision loss.

The cell regeneration mechanism of NouvSight001 can, in theory, address all vision impairments caused by retinal degeneration and retinal damage, offering even broader prospects for future clinical applications and commercialization.

Many people only see the dual IND achievements of NouvSight001, but in my view, Clinical approval is merely the outcome; the true core strength lies in iRegene’s established platform capabilities.

In the current cell therapy industry, many companies adopt a model of “one technology, one product, one global expansion,” focusing on breakthroughs with a single product.

iRegene has already completed its iteration, transitioning from “single-product globalization” to “One Platform, Multi-Line Output, Global Synchronous Development” systematic globalization model.

Underpinning all of this is its independently developed AI + Chemical Induction Underlying Technology Platform.

The most remarkable aspect of this system is that it circumvents the safety risks associated with gene editing and viral vectors, relying entirely on small-molecule compounds to regulate cellular gene expression and achieve directed differentiation of iPSCs.

A simple breakdown of its core logic: AI-driven high-throughput screening identifies core targets and compound combinations, while purely chemical induction precisely reshapes cellular functions, ultimately transforming the complex and unstable process of cell differentiation into a Standardizable, Scalable, and Replicable Industrialized Production Process.

This precisely hits the biggest pain points in the cell therapy industry: batch-to-batch inconsistency, high production costs, difficulty in scaling up, and challenges in cross-indication repurposing.

Mature platform capabilities have never been about single-dimensional enablement.

This technological foundation has already been validated across multiple key pipelines:

Parkinson's Disease Pipeline NouvNeu001, secured dual designations from the FDA for Fast Track and Regenerative Medicine Advanced Therapy (RMAT), with Phase II clinical trials in China and the United States progressing steadily;

Multiple System Atrophy Pipeline NouvNeu004, achieving dual filing and dual approval in both China and the United States, it is the world’s first iPSC-derived therapeutic product for MSA to enter clinical trials;

Nowadays NouvSight001: Clinical Implementation in China and the US, formally extending this mature technology into the field of degenerative eye diseases.

From neurodegenerative diseases to ophthalmic fundus disorders, leveraging a unified underlying technology platform to continuously deliver a globally compliant pipeline of innovative therapies constitutes the core competitive moat of a platform-based enterprise.

Securing dual IND approvals in China and the US is merely a paperwork achievement. For off-the-shelf cell therapies to truly achieve global clinical implementation, they must overcome a series of practical hurdles, including regulatory compliance, CMC manufacturing, cross-border supply chains, and multi-center clinical operations, thereby testing a company’s comprehensive execution capabilities.

Cellular therapies differ from conventional small-molecule drugs, and off-the-shelf iPSC cell products impose extremely stringent requirements on production quality control.

What sets iRegene apart is its establishment of a unified CMC and quality system that complies with the dual regulatory standards of the NMPA and the FDA.

Leveraging this system, the company has established a Standardized Off-the-Shelf Cell Product Manufacturing Platform, addressing the industry pain points in the large-scale manufacturing of cell-based therapies. This also serves as a critical foundation for enabling dual regulatory submissions in both China and the United States across multiple pipelines.

Building on this foundation, iRegene has established the Supply Chain System Covering Both China and the United States, build cross-border cold-chain logistics capabilities to ensure that spot cell therapy products can be simultaneously supplied to different clinical centers in China and the United States, thereby addressing the practical challenges of transporting and delivering cell-based drugs.

On the clinical front, iRegene has already established the Clinical Center Execution Network in China and the United States.The earlier regulatory submissions and clinical development of NouvNeu001 and NouvNeu004 in China and the United States have accumulated mature experience in communicating with the regulatory authorities of both countries and designing global clinical trial protocols. This experience can be leveraged for the NouvSight001 project.

iRegene’s CFO, Ruan Tianshi, has stated that globalization is not merely an attempt focused on a single product for iRegene, but rather a long-term strategy embedded in its R&D system since the company’s inception. “The ability of the same underlying technology platform to continuously generate candidate products that meet global development standards indicates that the development and regulatory pathways for each pipeline asset are replicable.” In the future, iRegene will continue to advance the clinical development of its core pipelines in both China and the United States, while actively exploring opportunities for global multi-center clinical trials and international commercial collaborations.

In the field of cell therapy, global competition for cutting-edge innovative drugs is essentially a comprehensive contest involving technology, regulation, industrialization, and team execution. Many companies possess innovative technologies but struggle to advance their products into clinical stages in both China and the United States simultaneously.

From Parkinson’s disease to multiple system atrophy, and further to retinitis pigmentosa in ophthalmology, we have seen iRegene successfully achieve dual filings and dual approvals in both China and the United States for multiple pipeline assets, a direct testament to its globalized execution framework.

In the field of retinitis pigmentosa treatment, gene therapy has opened a window of opportunity, but it only benefits a small subset of patients carrying specific mutations. NouvSight001, representing off-the-shelf cell replacement therapy, is not an alternative to gene therapy; rather, the two are complementary, jointly expanding the therapeutic toolkit for inherited retinal diseases.

For the industry, the greater significance of NouvSight001 lies in validating this platform model: leveraging the same underlying “AI + chemical induction” technological capabilities, the company can continuously develop candidate products for multiple aging-related degenerative diseases, including neurological and ophthalmic conditions, enabling global simultaneous development and driving more cell therapy products with clinical translational value and scalable commercial potential toward global development.