Ophthalmology is the field of medicine that involves the diagnosis and treatment of eye diseases. From common refractive errors to conditions like glaucoma, age-related macular degeneration (AMD), and diabetic retinopathy, at least 2.2 million patients worldwide are estimated to experience vision impairment or blindness as a result of ocular diseases. With global trends predicting a growing proportion of older adults in the population, this number is expected to increase even further in the future. Therefore, there is an immediate need for new and effective treatments in ophthalmology, as well as greater awareness about preventative measures to maintain eye health. This increasing demand for advanced ocular therapies can be seen in the ophthalmology clinical trials market size, which was valued at US$1.5 billion in 2022 and is expected to grow at a compound annual growth rate (CAGR) of 6.6% between 2023 and 2030.

Full-service ophthalmology contract research organizations (CROs) like TFS HealthScience have played a key role in supporting this field, particularly by addressing unmet needs for specialized imaging techniques and long-term follow-up studies to assess efficacy. Sponsors have leveraged CRO expertise in trial design, patient recruitment, and regulatory compliance to accelerate the pace of advancements in ophthalmology clinical research. With so many exciting new treatment approaches emerging, this article will highlight key updates in the clinical landscape of ophthalmology, covering various innovative therapies and cutting-edge technologies.

Missed our feature in the Spring 2023 edition of the esteemed European Biotechnology magazine? Read more here about how TFS CRO is driving the future of ophthalmology!

 

1. Gene Therapy Breakthroughs in Inherited Retinal Diseases

Gene therapy has been revolutionizing several areas of medicine and ophthalmology is no exception. This approach involves treating diseases by delivering genetic material (DNA or RNA) into the retina of the patient to modify abnormal gene expression and potentially delay vision loss. Advancements in gene therapy research have especially been a key step forward for the treatment of inherited retinal diseases, which are characterized by a heterogeneous group of genetic mutations in the eye. Back in 2017, the United States (U.S.) Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved voretigene neparvovec-rzyl (Luxturna), the first gene therapy treatment to reach the market.

The approval of Luxturna paved the way for more gene therapies, and as of 2024, several new treatments have made it to advanced stages of clinical trials. For example, ATSN-101, a subretinal adeno-associated virus 5 (AAV5) gene therapy for Leber congenital amaurosis 1 (LCA1). Caused by mutations in the GUCY2D gene, LCA1 is a rare inherited retinal disease that typically causes blindness in early childhood. In an open-label Phase 1/Phase 2 clinical trial, high-dose treatment with ATSN-101 was associated with clinically significant improvements in retinal sensitivity.

This year’s annual meeting of the American Academy of Ophthalmology in Chicago also revealed the latest Phase 2/Phase 3 trial results on the gene therapy, MCO-10, for treating retinitis pigmentosa. Of the 27 treated patients, 40% to 50% gained significant vision, such as going from light perception to 20/400. MCO-10 has already received Fast Track designation from the FDA and a Biologic License Application (BLA) is expected to be submitted to the FDA in early 2025.

In addition to genetic therapies showing promise for treating other retinal diseases like AMD and diabetic retinopathy, there is an increasing body of evidence investigating CRISPR-Cas9 gene editing technology in gene therapy development for ocular diseases.

Read more about the Challenges and Considerations for Gene Therapy Trials for Inherited Retinal Diseases (IRDs) in our article here.

 

2. Artificial Intelligence in Ophthalmology Diagnostics

In the last few years alone, artificial intelligence (AI) technology has advanced at a staggering speed, and today, it is opening new avenues for diagnosing and managing eye diseases. As of 2024, it has already become possible for ophthalmologists to rely on AI-powered diagnostic tools. Computerized analysis of retinal images offers greater accuracy than human observers by detecting subtle signs of early disease that could be missed otherwise.

As early as in 2016, Google developed their Automated Retinal Disease Assessment (ARDA), which uses AI to help healthcare workers detect diabetic retinopathy. It was designed to support high-quality diabetic retinopathy screening programs in countries without enough eye specialists, such as India and Thailand. ARDA conducts over 3,000 new screenings each week and it is currently undergoing clinical trials in the US and Thailand.

The benefit of leveraging AI technology increases the pace of screenings for ocular diseases significantly frees up time and resources for clinicians in ophthalmology clinics. In the coming years, machine learning (ML) models will likely support early diagnosis, predict disease progression, and significantly improve patient outcomes. Implementing AI screening tools more widely can enable faster disease detection and earlier treatment, reducing the chances of blindness or permanent damage to the retina.

 

3. Advancements in Minimally Invasive Glaucoma Surgery (MIGS)

Minimally invasive glaucoma surgery (MIGS) refers to a group of various procedures that are used to reduce intraocular pressure (IOP) in the eyes of patients with glaucoma. Examples of MIGS procedures include trabecular micro-bypass stents, suprachoroidal shunts, and subconjunctival filtration devices. In recent years, these types of procedures were developed using microscopic-sized equipment and tiny incisions to offer an alternative to standard glaucoma surgeries like trabeculectomy and ExPRESS shunts. Operations classified as MIGS reduce the incidence of complications in patients while lowering eye pressure and preventing progression of glaucoma, but some effectiveness is admittedly traded off relative to standard glaucoma procedures for this increased safety.

Clinical studies have demonstrated that many MIGS procedures can successfully lower IOP and reduce the need for glaucoma medications in these patients. For example, this 2023 review analyzed evidence from randomized clinical trials evaluating Trabectome^®, iStent inject^®, Kahook Dual Blade^®, XEN^® Gel Stent, and Hydrus^®. The study found MIGS is generally safe and efficacious, but high-quality large prospective trials are required for longer term analysis.

 

4. Innovations in Corneal Treatments

Corneal dystrophies are another class of ophthalmology diseases that have seen considerable treatment progress. Initially, the clinical landscape of therapies for these conditions included anterior lamellar keratoplasty, Descemet stripping automated endothelial keratoplasty (DSAEK), and Descemet membrane endothelial keratoplasty (DMEK). These procedures enabled a disease-focused approach and allowed patients to receive more tailored strategies for corneal implants. However, other technologies have also emerged in recent years.

For example, to address the scarcity of donated human corneas, bioengineering corneas have now become a clinical reality, offering hope to patients with severe corneal damage or those waiting for donor tissues. These artificial corneas are made up of synthetic materials (e.g., porcine collagen, recombinant human collagen), negating the need for human donors, and can integrate with the patient’s own cells. This approach is also known as “bioprinting” and some early clinical studies have assessed bioengineered corneal implants, demonstrating promise for corneal dystrophy treatment in the coming years.

Other key technologies include corneal cross-linking, a newer FDA-approved treatment for keratoconus, which has become a faster and more comfortable procedure for patients. Novel drug delivery systems are also being investigated, including sustained-release implants and nanoparticle-based therapies, to treat corneal infections and inflammatory retinal symptoms with greater precision.

 

5. Telemedicine and Remote Monitoring in Eye Care

Finally, the clinical landscape of ophthalmology care has seen significantly greater adoption of telemedicine, especially since the COVID-19 pandemic. Remote monitoring technologies are becoming standard practices for physicians to better deliver eye care to a wider range of patients. For example, there are smartphone apps and portable health devices that enable home-based basic vision tests and even retinal imaging for remote assessment by ophthalmologists.

Among the apps are myVisiontrack (Vital Art and Science/Genentech), Alleye (Oculocare Medical), OdySight (Novartis/Tilak Healthcare). There is also the ForeseeHome, an FDA-approved AI-enabled device for patient self-testing for AMD by using macular perimetry based on hyperacuity. Coupled with video conference platforms, these tools have helped ophthalmologists significantly widen their reach to patients with virtual consultations and chronic condition monitoring without the need for in-person visits. This has been especially crucial for addressing inequities in the delivery of vision care and ophthalmic health services to patients from underserved populations or remote geographic areas.

 

Conclusion

In conclusion, the field of ophthalmology is seeing several exciting updates to the clinical therapeutic landscape, from gene therapy and AI breakthroughs to advanced technologies in ocular surgeries and remote patient monitoring capabilities. The massive collaborative effort between sponsors, researchers, patients, and ophthalmology CROs has made these crucial developments possible and we can look forward to more key changes in the future for retinal disease therapy.

To learn more about the latest treatment advancements in ocular diseases in 2024, visit our article here!

 

TFS HealthScience CRO: Your Industry-Leading Partner in Ophthalmology

Experience the difference with TFS HealthScience Ophthalmology CRO for your next ophthalmology clinical trial! With a global mindset and local understanding, we provide a comprehensive suite of services, including study protocol design, medical monitoring, and project delivery. Our experienced teams and innovative technology ensure efficient delivery, no matter the complexity. Trust TFS HealthScience to take your study to the next level with our dedicated Ophthalmology team. Visit TFS Ophthalmology CRO’s website to learn more about the data-driven solutions we can offer for your next clinical trial or connect with a TFS representative here!

Discover the top ten CROs to consider for your next ophthalmology clinical trial here!

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