Ahead of ARVO 2025 next week where Emmes is poised to launch the first dedicated ophthalmology clinical trial technology platform, we spoke with Saqib Parkar, Managing Director at Emmes’ OptymEdge, about how CROs can reshape gene therapy trials in ophthalmology. . With only one FDA-approved ocular gene therapy on the market, the stakes are high and timelines are critical. Ophthalmologists working with Saqib emphasise the growing importance of CROs as strategic partners—valued for their transparency, therapeutic expertise, and collaboration.
As cell and gene therapies redefine treatment possibilities, clinicians play a key role in shaping trials that are not only scientifically sound but also tailored to the unique needs of rare disease patients. In this article we explore the pivotal role they play in helping deliver bespoke clinical trials for gene therapies in eye diseases and the profound benefits these trials bring to both practitioners and their patients.
The Evolving Landscape of Clinical Trials
The field of ophthalmology has witnessed extraordinary progress, thanks to breakthroughs in cell and gene therapies. These innovations offer a lifeline to patients with debilitating eye diseases. Dr. Paul A. Sieving, Director, Center for Ocular Regenerative Therapy at UC Davis School of Medicine, underscores the critical need for these trials, particularly in addressing rare inherited ocular disorders such as the Inherited Retinal Dystrophies (IRDs). He states, ‘these conditions, although individually rare, collectively impact a substantial number of individuals within communities”. The advent of gene therapies has rekindled hope for these patients, providing treatment options where none previously existed.
While it’s important to note that results from ocular gene therapy trials have exhibited variability, and only one therapy has received FDA approval thus far, Dr. Ian M MacDonald, a distinguished ophthalmologist from the Department of Ophthalmology & Visual Sciences at the University of Alberta, reminds us that these trials are inherently experimental. “They serve as invaluable learning opportunities for the ophthalmic community, allowing for the refinement of protocols, redirection of research efforts, and, most significantly, unwavering support for patients as they embark on a journey towards potential breakthroughs,” added MacDonald.
The Significance of Gene Therapy Trials
Dr Sieving has been involved with a gene therapy trial for X-linked retinoschisis (XLRS). XLRS represents just one of many genetic conditions causing progressive vision loss, ranking among the top 10 most frequent conditions within the realm of Inherited Retinal Dystrophies (IRDs). When I spoke with him recently he agreed that the impact of such eye diseases on communities is substantial and stressed on the urgency of starting and hopefully completing successful trials. He highlighted why working more closely with CROs, and the relationship between sponsor trial manager and clinician has never been more important. A breakdown here will inevitably lead to delays and, in the worst of cases, can be a highly complicit factor in a trial’s failure – so we owe to patients to ensure this is avoided whenever possible.
But first let’s look at a couple of successes, and Dr. Sieving is hopeful that the approval of Luxturna will herald in encouragement from clinicians to search out more trials and help these patients through what can be a dauting trial journey. While Luxturna – a Leber congenital amaurosis (LCA) treatment – was approved back in 2017, it remains the only one available and is a historic milestone as the first in vivo gene therapy. Infants and children treated with Luxturna experience vision restoration, offering tangible evidence of gene therapy’s potential to address genetic eye diseases.
However, progress in the field since 2017 has been slower than anyone would have liked, and there is a clear need for more Human Gene Therapy ocular trials. What is encouraging as a CRO is the clinicians we work with strongly believe that these trials should be more collaborative. If we can share more expertise – maybe the practical learnings across competing trials that now we are positioned to appropriately leverage this potential. The challenge of course is that gene therapy trials for the eyes come with their unique set of difficulties and requirements, including skilled drug delivery, patient recruitment challenges, lengthy patient follow-up, and intricate trial data analysis. And, while it might be understandable people say ‘you would say that’, I would encourage clinicians to bring in one of the handful of experienced CROs in the space because trials design here are fundamental not only to the patients, but in how to engage with the regulators as we start to see successes.
