BENGALURU, INDIA — In a landmark development for regenerative medicine, Eyestem Research Pvt Ltd announced on January 28, 2026, that it has received approval from the Central Drugs Standard Control Organisation (CDSCO) to initiate Phase 2 clinical trials for Eyecyte-RPE™.
This investigational cell therapy is designed to treat blindness caused by Geographic Atrophy (GA), the advanced stage of dry age-related macular degeneration (dry AMD), a condition that currently has no available cure to reverse vision loss.
Restoring the “Foundation” of Sight
At the heart of Eyestem’s innovation is a scalable, allogeneic approach using human induced pluripotent stem cells (hiPSCs). In the human eye, the Retinal Pigment Epithelium (RPE) serves as a critical support layer for photoreceptors the cells responsible for capturing light. In dry AMD, this “foundation” layer degenerates, leading to the collapse of retinal structures and irreversible blindness.
Eyecyte-RPE™ works by replacing these damaged or lost RPE cells with healthy, lab-grown equivalents. Unlike previous attempts at cell therapy that were difficult to scale, Eyestem’s platform is designed to produce high-quality cells in a cost-effective, “off-the-shelf” format, potentially making the treatment accessible to millions.
Read More: Eyestem’s Eyecyte-RPE™ Trial Shows Vision Rescue for Geographic Atrophy Patients
Phase 1 Success: Vision Gains that “Reshape the Landscape”
The move to Phase 2 follows an exceptionally successful Phase 1 study. While traditional safety trials often aim merely for the absence of side effects, Eyestem reported significant functional improvements in patients:
- Clinical Efficacy: Subjects with advanced GA recorded an average improvement of 14.8 letters on vision charts at six months.
- Sustained Gains: At the one-year mark, patients maintained an average gain of 14.6 letters.
- Surgical Validation: The trial involved six different surgeons, confirming that the delivery method is standardized and not dependent on a single specialist’s “magic touch.”
There is no available therapy that reverses blindness caused by geographic atrophy (GA), affecting an estimated 8 million patients worldwide. Eyestem is among a few companies globally pursuing a scalable cell therapy approach toward a potential cure. CDSCO approval enables us to advance Eyecyte-RPE™ into Phase 2 clinical evaluation, allowing broader, randomized validation of our best-in-class Phase 1 outcomes. With AMD projected to affect 288 million people by 2040, this marks a major milestone in our mission to transform retinal care and patient lives worldwide.
Dr. Jogin Desai
Read More: Eyestem Raises Funds to Advance Eyecyte-RPE™ Through Phase 2 Clinical Trial and Towards US IND
| Statistic | Impact |
| Current GA Patients | ~8 Million worldwide |
| Total AMD Projection (2040) | 288 Million |
| Current Standard of Care | Slows progression; cannot reverse blindness |
| Eyestem’s Goal | Disease reversal and vision restoration |
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Expansion and Regulatory Milestones
The upcoming randomized, controlled Phase 2 trials in India will focus on safety and efficacy in a larger cohort of subjects with moderate GA. Simultaneously, Eyestem is preparing to file an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) to begin clinical studies in the United States.
Backed by a recent oversubscribed funding round, the company is well-positioned to navigate the complex global regulatory landscape. If successful, Eyecyte-RPE™ could become a first-in-class treatment, shifting the paradigm of retinal care from “managing decline” to “restoring life.”
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