Retinal diseases are the fastest growing cause of blindness and have a significant impact on patients’ independence and quality of life. As the global ophthalmology community gathers for the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, all eyes are on how scientific advances are reshaping the future of eye care.
For Astellas, this momentum reinforces the broader belief that the eye can serve as a powerful testing ground for regenerative medicine. Jotaro SuzukiPrimary Focus Lead, Blindness and Regeneration, Marcy EnglishHead of Biopharmaceutical and Ophthalmology Development, and Erin KimbrelAs director of cell and gene therapy research, he considers how advances in cell therapy could change the future of degenerative diseases in ophthalmology and other fields.
Why is ophthalmology progressing so rapidly?
Jotaro: Looking at how the field has evolved over the course of my career, the speed of change over the past few years is truly remarkable. A major driver of this acceleration is the convergence of advances in science and technology, including a deeper understanding of the biology of retinal diseases, advances in therapeutic approaches, and new treatments such as cell and gene therapy.
These advances, along with the eye’s unique biology providing the perfect environment for innovation, are enabling researchers to address vision loss across more stages of the disease and reshape expectations about what is achievable for patients.
How do you think cell therapy will shape the future of ophthalmology, particularly the way vision loss is addressed?
Marcy: Many retinal diseases show that vision loss is caused by degeneration or loss of key retinal cells. While some treatments can slow progression and protect remaining cells, there is a real opportunity to study approaches that have the potential to replace critical cells in the retina.
Cell therapy is one of those approaches. Although this is an emerging therapy, it is based on a scientific foundation that Astellas is familiar with, combining insights from three decades of innovation in immunosuppressants to advance modern organ transplantation with more than a decade of focused investments and targeted acquisitions in ophthalmology and regenerative medicine.
Why did Astellas Pharma choose the eye as a target for regenerative medicine?
Erin: The eye is ideally suited to explore regenerative approaches. Its small size means a low manufacturing burden for cells or viral vectors, and its immunoprivileged properties help limit inflammatory responses. Progress can also be monitored through accurate non-invasive imaging, allowing for a clear assessment of anatomical and functional outcomes.
That’s why we took a deliberate approach to gaining experience in this environment. Data and learning in ophthalmology is building a foundation that may inform how regenerative therapies are being studied in other organs.
What will it take to move regenerative approaches from scientific promise to scalable patient impact?
Marcy: We know that even with strong science, one of the biggest challenges is translating promise into treatments that can reliably reach patients. Translating regenerative medicine into meaningful impact requires end-to-end capabilities across discovery, development, manufacturing, and delivery.
For example, allogeneic cell platforms offer a path to more consistent manufacturing, broader access, and scalability beyond highly personalized cell therapies. Astellas also leverages technologies such as universal donor cells and adeno-associated virus gene therapy as part of this approach.
Jotaro: The gap between discovery and real-world application is where many regenerative approaches struggle. why? Because many of the challenges exist at the intersection of biology and execution, especially when it comes to consistency and large-scale manufacturing. No single organization has all the answers, which is why partnerships are so important.
One example is our partnership with Yaskawa Electric through Cellafa Bioscience, which combines regenerative medicine expertise with advanced robotics and AI. The focus is on learning how to digitize and automate cell manufacturing and developing standardized, transferable processes that support a broader range of applications over time.
Could advances in ophthalmology and cell therapy inform regenerative medicine more broadly?
Erin: Through our work in ophthalmology with retinal pigment epithelial grafts, we have demonstrated the medium- to long-term safety of a pluripotent stem cell (PSC)-based approach. 1 In parallel, we and other researchers have optimized PSC-mediated differentiation and production of other cell types. Improvements in raw material quality, analysis, bioreactors, and automation have enabled off-the-shelf cell therapies that are scalable across regenerative medicine.
More broadly, progress comes from continuous learning, repetition, and persistence, emphasizing a long-term commitment to progress.
Looking to the future, what does this mean for patients?
Marcy: These are exciting times. We are trying to understand what potential regenerative approaches have. By addressing the loss and degeneration of retinal cells, ophthalmology is opening up new ways of thinking about disease and recovery, focusing on restoring or replacing what disease has taken away.
Lessons learned from the eye extend beyond ophthalmology and help shape a broader vision for regenerative medicine. That vision is based on applying today’s scientific rigor to expand what patients can expect tomorrow.
References
1. Schwartz Others. lancet. 2015. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt macular dystrophy: Follow-up of two open-label phase 1/2 trials. 385:509-16.
