When discussing cell and gene therapy, or CGT, ophthalmologists have in fact long stood at the forefront of the industry.

From LUXTURNA (voretigene neparvovec-rzyl), the world’s first approved gene therapy drug, to the world’s first iPSC-derived cell transplantation, and then to the first gene-editing therapy to enter clinical application, multiple global “firsts” have taken place in ophthalmology. Behind these milestones lie not only clinical needs, but also the long-term exploration and persistence of ophthalmologists.

On June 11, 2026, Global Ophthalmology Conference 2026 was held at the Zhongguancun Exhibition Center Conference Center in Beijing, China. At the conference, Professor Zi-Bing Jin, Vice President of Beijing Tongren Hospital, Capital Medical University, delivered a keynote presentation titled “New Opportunities in Ophthalmic Cell and Gene Therapy.” Starting from the global development trajectory of CGT, Professor Jin systematically reviewed advances in gene therapy, stem cell therapy, photoreceptor transplantation, and other directions. He also shared his team’s independent explorations in inherited retinal diseases and age-related macular degeneration, or AMD.

Professor Jin’s central message was clear: thanks to its unique advantages — relative anatomical independence, safety and controllability, and easily assessable therapeutic effects — ophthalmology has become one of the most important “first launchpads” for cell and gene therapy. In the future, it will remain one of the most intensive clinical scenarios for breakthrough CGT innovation.

Below, EyeFuture reviews several key points from Professor Jin Zi-Bing’s presentation.

Five Global “Firsts”: Why Has Ophthalmology Broken Through First?

Looking back at the global history of CGT development, Professor Jin highlighted a phenomenon that deserves the attention of the ophthalmic community: multiple milestone global “firsts” have occurred in ophthalmology.

• The first truly successful gene therapy: Clinical exploration was completed in London in 2005. Twelve years later, in 2017, the first gene therapy drug, Luxturna, was approved for marketing. Its target indication was RPE65-related inherited retinal disease.

• The first optogenetic therapy: Clinical exploration was first launched in ophthalmology.

• The first FDA-cleared clinical trial using human embryonic stem cell-derived retinal pigment epithelial cells: Preliminary Phase I results were published in 2012, marking the first reported transplantation of hESC-derived cells into human patients.

• The first iPSC-derived cell therapy: In September 2014, Japan completed the world’s first transplantation of an autologous iPSC-derived RPE cell sheet for wet AMD.

• The first clinical application of gene editing: This was also first initiated in ophthalmology.

Why have so many “firsts” been concentrated in ophthalmology? Professor Jin’s explanation is likely also the answer many clinicians already have in mind: the eye is a relatively independent, safe, and controllable organ. Therapeutic outcomes can be assessed directly and intuitively. Whether a patient “can see or cannot see” is one of the simplest and most direct standards of evaluation. This unique advantage makes ophthalmology an ideal clinical validation scenario for new therapeutic technologies.

“In the global CGT field, ophthalmology actually represents a very large space.”

A Dual-Track Policy Framework: New Space for Ophthalmic CGT Under Order No. 818 and the 828 Pathway

When discussing China’s domestic industrial environment, Professor Jin specifically mentioned two policies that are crucial to the development of ophthalmic CGT.

Order No. 818: This policy focuses on “new biomedical technologies.” Led by the National Health Commission, it follows a technology-oriented pathway. It was released in 2025, its implementation plan was launched in 2026, and it officially came into effect on May 1, 2026. It covers five major tracks, among which gene therapy and cell and derivative therapy are directly related to ophthalmic CGT.

The 828 pathway: This continues the traditional drug pathway, led by the National Medical Products Administration, and advances projects according to the conventional drug development process.

The parallel operation of these two pathways provides more flexible clinical translation channels for ophthalmic gene therapy and cell therapy. Professor Jin noted that related research projects from his team have already advanced investigator-initiated trial explorations through the 818 pathway.

For clinicians, this means that future exploratory studies initiated by investigators will have a clearer pathway under a compliant framework.

Gene Therapy: From BCD to Stargardt Disease, China’s Independent Exploration Is Accelerating

In the section on gene therapy, Professor Jin focused on two types of China-based research that are currently being advanced.

1. BCD: Bietti Crystalline Dystrophy and the CYP4V2 Gene

The teams led by Professor Yang Liping from Peking University Third Hospital and Professor Li Wei from the Institute of Zoology, Chinese Academy of Sciences, have each independently advanced CYP4V2 gene therapy from scratch. These projects have now entered Phase III clinical development and are expected to become the first domestic breakthrough in approved ophthalmic gene therapy.

2. Stargardt Disease: Juvenile Macular Degeneration and the ABCA4 Gene

Approximately 90% of Stargardt disease cases are caused by ABCA4 gene variants, making it one of the important monogenic inherited diseases leading to blindness in children. Many patients begin to experience visual decline at around the age of 10, with vision dropping to 0.1–0.2, significantly affecting the lives of children and adolescents.

Because the ABCA4 gene is nearly 7 kb in length, exceeding the packaging capacity of a single AAV vector, international approaches mainly use lentiviral vectors or dual-AAV vector strategies. Multiple clinical trials have already been initiated worldwide, and several clinical trials have also been completed in China.

Professor Jin’s team was the first in China to establish unique genetically engineered monkey models of Stargardt disease in the world. Using this model, the team found that the primary lesion of the disease is not limited to the foveal photoreceptors, as previously believed. The underlying retinal pigment epithelium also shows primary lesions. This discovery provides a key basis for new gene therapy strategies.

He also introduced his team’s long-term work in genetic diagnosis for inherited retinal degeneration. The diagnostic detection rate has increased from 13.5% in 2008 — already the highest level worldwide at that time — to approximately 70% today. This has laid a solid foundation for the precise development of subsequent gene therapies.

For many retinal disease specialists, inherited retinal diseases were once considered conditions with no available treatment. Today, however, genetic diagnosis combined with gene therapy is enabling the clinical pathway of “identifying the cause and intervening precisely” to become a reality.

Cell Therapy: iPSC-RPE Transplantation, from Japan’s First Case to China’s Practice

Cell therapy was another key focus of Professor Jin’s presentation. He pointed out that AMD ranks third among major irreversible blinding diseases in ophthalmology in terms of prevalence and burden. Wet AMD already has multiple treatment options, including anti-VEGF therapies. However, dry AMD, particularly geographic atrophy, still lacks effective treatment.

Although C3 and C5 complement inhibitors have been approved in the United States, they can only slow disease progression and cannot truly reverse visual function damage.

iPSC-RPE transplantation is offering a new therapeutic approach for dry AMD.

l In September 2014, Japan completed the world’s first iPSC-RPE transplantation. The recipient was a wet AMD patient in her seventies. Professor Jin was working with the team from 2007 to 2010.

l Follow-ups at one year, three years, and five years after surgery all showed that the transplanted cells survived intact. No immunosuppressants were used. The thickness of the neurosensory retina corresponding to the transplanted area was significantly better than that of the non-transplanted area, and the patient was completely free from anti-VEGF treatment.

l The latest follow-up in April 2025 showed that the patient’s condition remained at an early postoperative level. The patient reported that “the world in front of the eye had become significantly brighter.”

After returning to China, Professor Jin’s team completed IIT registration in 2025 and has carried out several exploratory cases of iPSC-RPE transplantation for dry AMD.

l At one to three months after surgery, ETDRS visual acuity testing showed monthly improvement.

l The dark areas on patients’ Amsler grids shrank significantly, and the range of visual distortion decreased.

l In the second end-stage patient, the central scotoma had almost disappeared at the three-month time point.

Professor Jin particularly noted that this is China’s first clinical exploration project using iPSC-derived RPE transplantation to treat dry AMD, and it is still being actively advanced.

The Next Step: Photoreceptor Transplantation and Combined “Gene + Cell” Therapy

If RPE transplantation addresses the “foundation,” then photoreceptor transplantation is directly related to the true restoration of central vision. It is also one of the most challenging directions in cell therapy.

Professor Jin introduced a landmark study published by his former mentor at the end of December 2023. In that study, patient-derived autologous iPSCs were differentiated into retinal organoids. A “cap” region measuring approximately 0.3 × 0.3 × 1 mm was cut and transplanted into the subretinal space of two end-stage retinitis pigmentosa patients. At 104 weeks, or roughly two years of follow-up, the grafts remained intact and survived. In one patient who received transplantation beneath the fovea, full-field stimulus testing showed significant functional recovery.

However, Professor Jin also objectively pointed out that OCT images showed that although the transplanted cells survived, they had not yet achieved complete integration with the host retina. This remains the biggest challenge in the field. His team is now optimizing the approach through new transplantation methods. In animal experiments, they have observed transplanted cells migrating toward the outer retinal layer and achieving a certain degree of functional recovery.

In 2026, the U.S. FDA granted orphan drug designation to retinal cells derived from an allogeneic human induced pluripotent stem cell line for the treatment of retinitis pigmentosa, and related clinical trials are expected to move forward.

Speaking about the future landscape, Professor Jin offered a clear judgment:

There are more than 200 disease-causing genes related to retinal diseases, of which approximately 10 are the most important. If therapies can be developed for these 10 genes, nearly half of patients with inherited retinal diseases could be covered.

For patients in the middle and late stages, where photoreceptors have already undergone extensive apoptosis, gene therapy will no longer be effective. Cell transplantation will become an irreplaceable treatment option.

Gene therapy and cell therapy together will form a complete therapeutic closed loop for blinding ophthalmic diseases in the future.

Conclusion

From the first truly successful gene therapy in 2005, to the first iPSC-RPE transplantation in 2014, and then to China’s independent clinical explorations now advancing in 2026, ophthalmic CGT has moved from being a “global first-mover testing ground” to entering a critical period of China-led realization.

EyeFuture will continue to follow the clinical progress and industrial implementation of this field.