As the global population ages and the incidence of vision-related conditions rises, ophthalmology is entering a transformative era. Beyond symptom management, the field is moving towards disease modification through cell and gene therapies like optogenetics while boosting artificial intelligence (AI) to speed up pipelines, and developing minimally invasive delivery systems for more patient-friendly therapies.

 

Unmet Needs Driving Innovation

Chronic eye conditions such as diabetic macular oedema (DME), age-related macular degeneration (AMD), and myopia – particularly prevalent in Asia –, are on the rise. Yet many therapeutic needs remain unmet. “The market dynamics reveal substantial treatment gaps,” Riad Sherif, CEO of Swiss ophthalmology biotech Oculis, points out. “While approximately 1.8 million Americans have been diagnosed with DME, less than half receive treatment.”

“Age-related macular degeneration represents perhaps the most critical unmet need, particularly the dry form of the disease,” warns Serge Picaud, executive director of the Paris-based Vision Institute. “Given the condition’s prevalence in industrialised nations and the demographic trends toward population aging, this represents a substantial public health challenge requiring innovative therapeutic approaches.”

Despite these treatment gaps, “protecting sight is a universal priority for patients, who are willing to pursue treatment options they might decline for other conditions,” Sherif asserts. “This creates a strong commercial opportunity and a significant mission to provide transformative therapies.”

 

Renewed Industry Attention and Investment

Meanwhile, blockbuster treatments have reignited investor and Big Pharma interest in ophthalmology. “The sector has experienced significant capital influx following the emergence of several blockbuster treatments that demonstrated substantial commercial viability,” Picaud affirms. “This success has fundamentally altered industry perception regarding the therapeutic potential and market opportunities within ophthalmology.”

At the 2025 ARVO Annual Meeting, a gathering that brings together eye and vision researchers from across the globe, participants emphasised the growth of funding and investment for ophthalmology in the fields of gene therapy, biologics, and digital health‑enabled development.

Big Pharma has a growing stake in the field. Merck recently snapped up EyeBio for USD 1.3 billion, gaining access to the biotech developer’s Restoret, a promising therapy for AMD and DME. Swiss pharma giant Roche has likewise doubled down on ophthalmology, naming it a core therapeutic area following the success of Genentech’s anti-VEGF drug Vabysmo. Roche now plans to expand its pipeline into glaucoma and other under-served indications.

Sanofi too has stepped up its ophthalmology drive, something that has recently paid off with an FDA fast-track for its gene therapy for geographic atrophy (GA). “One of the areas that we’re now building in earnest internally is ophthalmology,” said chief scientific officer and global head of research Mike Quigley at a recent meeting at Sanofi’s Boston R&D centre attended by PharmaBoardroom. “Retinal diseases in particular are driven by inflammatory processes core to our expertise and it makes a lot of sense for us to deliver therapies for unmet needs in ophthalmology.”

 

From Symptom Control to Disease Modification

Ophthalmology is fast becoming a proving ground for gene and cell therapies that go beyond managing symptoms to potentially halting – or even reversing – vision loss.

“Ophthalmology is now emerging as one of the most promising frontiers for gene and cell therapies,” says Picaud. “The central idea is to either correct the faulty genes causing vision loss — potentially offering a curative approach — or to design therapies that can be administered more regularly and with greater control.”

Recent ARVO presentations highlighted major progress in the domain. US-based Ocugen showcased several late-stage therapies for inherited retinal diseases, while Opus Genetics, Beacon Therapeutics, and AAVantgarde Bio revealed encouraging early-stage data.

Among the most exciting developments is optogenetics, which offers hope to patients with advanced retinal degeneration. Companies like RhyGaze and Nanoscope Therapeutics are racing to bring optogenetic therapies to market while the Vision Institute is also advancing an optogenetic candidate via its spin-off GenSight Biologics.

“We used gene therapy to introduce an opsin gene from green algae into these surviving retinal neurons,” Picaud explains. “Opsins are light-sensitive proteins — and in algae, they’re naturally linked to ion channels that directly trigger electrical activity in response to light. We took the genetic code for this algae opsin, packaged it into an AAV vector, and injected it into the retina.”

 

AI Accelerating Discovery and Personalisation

Another ophthalmology game changer is the use of artificial intelligence (AI) in drug discovery and clinical trials. AI is significantly transforming both areas by leveraging deep learning for diagnosis, predictive modelling, and accelerated candidate selection, which is significantly speeding up pipelines.

Sanofi has been embedding AI to supercharge every stage of R&D across its therapeutic areas, including opthalmology. “To narrow the [drug discovery] search space, we’re using AI-driven screening and protein language models to engineer them for ocular and other uses,” says Matt Truppo, Sanofi’s global head of research platforms and computational R&D.

“Artificial intelligence has been integral to our research methodology for years,” Picaud concurs, stressing the logical connection between AI and eye health. “The convergence of AI with ophthalmology occurs naturally, as both fields utilise neural network principles.”

The Vision Institute’s primary AI application focuses on disease progression modelling, enabling the prediction of patient outcomes over months or years. “This capability is revolutionary for treatment assessment, as it allows us to determine whether therapeutic interventions successfully alter disease trajectory and demonstrate clinical efficacy.”

 

Less Invasive Delivery Options

Eye health innovators are also looking to less invasive and more patient-friendly drug delivery systems to improve ophthalmological care. Retinal vascular disease patients for example are currently burdened with frequent intravitreal anti-VEGF therapy injections. New treatment approaches seek to either extend dosing intervals with longer-acting delivery modes, or to replace injections altogether.

Oculis is advancing a lead asset for DME, OCS-01, that looks to free patients of the need for frequent injections. Due to be launched in 2027-28, OCS-01 represents the only topical “eye drop” therapy globally capable of treating DME. “Our topical delivery system addresses multiple clinical challenges simultaneously,” claims Sherif. “It enables immediate treatment initiation upon diagnosis, potentially preventing irreversible vision loss. Second, it provides therapeutic options for the 40 percent of patients who demonstrate inadequate response to current injectable therapies.”

Oculis is also breaking ground in precision medicine for dry eye. By identifying a simple, saliva-based biomarker that predicts treatment response, the company is streamlining diagnosis and personalising care. “Traditional dry eye management often involves sequential therapeutic trials, which can lead to patient frustration and suboptimal outcomes. We have identified a simple biomarker – detectable through saliva testing similar to COVID diagnostics – that predicts therapeutic response,” says CEO Sherif.