Retinal diseases are among the fastest growing causes of blindness, having a profound impact on patients’ independence and quality of life. As the global ophthalmology community gathers at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, attention is focused on how scientific advances are reshaping the future of eye care.
For Astellas, this momentum reinforces a broader belief: that the eye could serve as a powerful proving ground for regenerative medicine. Jotaro Suzuki, Primary Focus Lead, Blindness & Regeneration, Marci English, Head of BioPharma and Ophthalmology Development, and Erin Kimbrel, Head of Cell and Gene Therapy Research, reflect on how progress in cell therapy could change the future of degenerative diseases in ophthalmology and beyond.
Why is ophthalmology evolving so rapidly now?
Jotaro: When I look at how the field has evolved during my career, the pace of change over the last few years really stands out. A big driver of that acceleration is a convergence of scientific and technological advances – including a deeper understanding of retinal disease biology, progress in delivery approaches and emerging modalities like cell and gene therapy.
These advances – alongside the eye’s unique biology, which provides an optimal environment for innovation – are enabling researchers to address vision loss across more stages of disease and reshape expectations of what may be achievable for patients.
How do you see cell therapy shaping the future of ophthalmology, particularly in how vision loss is addressed?
Marci: For many retinal diseases, you see that vision loss is driven by the degeneration or loss of key retinal cells. Some therapies can slow progression and protect remaining cells, but there is a real opportunity to investigate approaches that potentially replace critical cells in the retina.
Cell therapy is one of those approaches. While an emerging modality, it builds on scientific foundations Astellas knows well – combining learnings from 30 years of innovation in immunosuppressants to advance modern organ transplantation with over a decade of focused investment and targeted acquisitions in ophthalmology and regenerative medicine.
Why has Astellas chosen the eye as a focus for regenerative medicine?
Erin: The eye is ideally suited to explore regenerative approaches. Its small size means a lower manufacturing burden for cell or viral vectors, while its immune-privileged characteristics help limit inflammatory responses. Progress can also be monitored through precise, non-invasive imaging, enabling clear assessment of anatomical and functional outcomes.
It’s why we’ve taken a deliberate approach to building experience in this setting. The data and learnings in ophthalmology are creating a foundation that may inform how regenerative modalities are explored in other organs.
What is required to move regenerative approaches from scientific promise to scalable patient impact?
Marci: Even with strong science, we know one of the biggest challenges is turning promise into therapies that can reliably reach patients. Translating regenerative medicine into meaningful impact requires end-to-end capabilities spanning discovery, development, manufacturing, and delivery.
Allogeneic cell platforms, for example, offer a path to more consistent manufacturing, broader access, and scalability beyond highly individualized cell therapies. At Astellas, we’re also leveraging technologies such as Universal Donor Cell and adeno-associated virus gene therapies as part of this approach.
Jotaro: That gap between discovery and real-world application is where many regenerative approaches struggle. Why? Because many of the challenges sit at the intersection of biology and execution – particularly around manufacturing consistently and at scale. No single organization has all the answers, which is why partnerships are so important.
One example is our collaboration with Yaskawa Electric through Cellafa Bioscience, which brings together 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 to support broader application over time.
What can advancements in ophthalmology and cell therapy teach regenerative medicine more broadly?
Erin: Through our ophthalmology efforts with retinal pigment epithelium grafts, we have demonstrated medium to long-term safety of a pluripotent stem cell (PSC)-based approach.1 In parallel, we and others have optimized PSC-directed differentiation and manufacturing for other cell types. Improvement in raw materials quality, analytics, bioreactors and automation are enabling scalable, off-the-shelf cell therapies across regenerative medicine.
More broadly, advances come from continuous learning, iteration, and persistence, underscoring long‑term commitment to progress.
Looking ahead, what could this mean for patients?
Marci: It’s an exciting time – we’re on the cusp of realizing what regenerative approaches may be capable of. By addressing retinal cell loss and degeneration, ophthalmology is opening up new ways of thinking about disease and recovery – with a focus on trying to restore or replace what disease has taken away.
The lessons learned in the eye extend beyond ophthalmology, helping to shape a broader vision for regenerative medicine – one grounded in applying scientific rigor today to expand what patients may hope for tomorrow.
References
1. Schwartz et al. Lancet. 2015. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. 385:509-16.