The ARVO 2025 Annual Meeting n Salt Lake City showcased a vibrant pipeline of ophthalmology innovation, from breakthrough retinal therapies to cutting-edge imaging and AI tools. Major presentations and posters revealed how scientists and companies are pushing the frontiers of eye disease treatment innovation. For sponsors and biotechs focused on ophthalmic programs, these meeting highlights offer strategic insights into emerging therapeutic trends and clinical development directions. The field is rapidly evolving, fueled by robust funding and big pharma investments, and ophthalmic drug development efforts are yielding novel treatments for sight-threatening diseases. In tandem, specialized contract research organizations (CROs) are expanding their ophthalmology expertise, ready to partner with innovators in bringing these advances to patients. This article explores pivotal insights from ARVO 2025 – advancements that are shaping the future of ophthalmology clinical development and accelerating access to innovative therapies.

ARVO 2024 Highlights At-a-Glance

Key themes from this year’s meeting included reducing anti-VEGF injection burden in retinal diseases, novel drug delivery platforms (like suprachoroidal injections), encouraging Phase 2 trial data in macular and corneal diseases, regenerative cell therapy reversing vision loss, and new treatments spanning dry eye, gene therapy for inherited retinal disorders, and even glaucoma surgery innovations. The most talked-about indications were age-related macular degeneration (AMD), both neovascular (wet AMD) and geographic atrophy (advanced dry AMD), along with diabetic retinal diseases, corneal and ocular surface disorders (e.g. dry eye disease), glaucoma, and pediatric ocular oncology (retinoblastoma). Emerging technologies in imaging and artificial intelligence (AI) were also in focus, promising to enhance disease detection and trial efficiency. Below, we delve into the top presentations, therapeutic trends, and company innovations that are shaping ophthalmology R&D in 2025.

Retinal Diseases: Reducing Treatment Burden in AMD and Beyond

Retinal vascular diseases like wet AMD and diabetic macular edema (DME) dominated ARVO 2025 discussions, with multiple new therapies aiming to ease the burden of frequent intravitreal injections. Anti-VEGF therapy has been the cornerstone for these conditions, but patients often require eye injections every 4–8 weeks. Innovative approaches presented at ARVO seek to extend dosing intervals or replace injections altogether:

• Subcutaneous anti-VEGF therapy: Ashvattha Therapeutics reported Phase 2 results for migaldendranib, an investigational subcutaneous treatment, showing it significantly reduced subretinal fluid and cut the need for intravitreal anti-VEGF injections in wet AMD and DME patients. This unique platform could lessen clinic visits and improve patient comfort by delivering anti-VEGF therapy via a simple under-the-skin injection rather than into the eye.

• Suprachoroidal delivery of long-acting drugs: Clearside Biomedical highlighted six ARVO abstracts on its suprachoroidal injection platform, including top-line Phase 2b trial data (ODYSSEY) of suprachoroidal CLS-AX (axitinib) in neovascular AMD. Suprachoroidal (injection into the space just beneath the retina) delivery achieved encouraging durability and safety, suggesting this route can prolong drug effects in macular diseases. One poster even described a novel deep learning algorithm to segment the suprachoroidal space on OCT images – an example of AI enhancing imaging for drug delivery optimization. By improving precision and training with synthetic eye models, these advances could accelerate adoption of suprachoroidal therapies.

• Real-world data and sustained-release implants: EyePoint Pharmaceuticals presented Phase 2 clinical trial readouts (VERONA and DAVIO 2) that demonstrated sustained efficacy in wet AMD and other indications with an acceptable safety profile, using trial designs that reflect real-world patient populations. Other companies showcased sustained-release implants and depot therapies. For example, Ocular Therapeutix discussed its hydrogel-based intravitreal implant (OTX-TKI) delivering a tyrosine kinase inhibitor; early trials (HELIOS) suggest it may maintain therapeutic levels for diabetic retinopathy without monthly injections. Collectively, these efforts underscore a trend toward reducing treatment burden – a critical factor for patient adherence and outcomes.

Meanwhile, geographic atrophy (GA) – the advanced dry form of AMD – saw exciting progress at ARVO 2025. GA has only recently gained its first approved therapies (complement inhibitors), and researchers are now racing toward even more potent solutions that can preserve or restore vision:

• Cell therapy reversing vision loss in GA: Eyestem Research’s presentation drew significant attention by reporting that its retinal pigment epithelium (RPE) cell therapy (Eyecyte-RPE) led to meaningful vision improvements in GA patients, with the first cohort of treated patients gaining an average of ~15 letters of visual acuity within 4–6 months. Even more remarkably, early retinal scans hinted at disease reversal, potentially regenerating tissue in an area traditionally considered irreversibly lost. This first-in-human Phase 1 result, achieved without serious adverse events, positions RPE cell transplantation as a promising regenerative strategy for dry AMD. Luxa Biotechnology also presented initial clinical results for an RPE stem cell therapy (RPESC-RPE-4W) in dry AMD, reinforcing the momentum behind cell-based regeneration of retinal tissue.

• Gene therapy targeting GA’s root causes: Expanding the fight against GA, gene therapy approaches made a debut. Notably, Kriya Therapeutics announced preclinical data and a new Phase 1/2 trial for KRIYA-825, an AAV gene therapy delivered via suprachoroidal injection that expresses a complement cascade inhibitor to halt GA progression. By continuously inhibiting complement C3 and C5 – pathways validated by FDA-approved GA drugs – a one-time gene therapy could provide long-term protection against retinal degeneration. The launch of KRIYA-825’s clinical trial in GA illustrates how ophthalmic drug development is extending beyond rare genetic disorders to target common age-related diseases with gene-based treatments.

Advanced Therapies: Gene and Cell Innovations on the Rise

ARVO 2025 underscored that gene and cell therapies are no longer futuristic concepts in ophthalmology – they are here and rapidly advancing through ophthalmology clinical trials. A wide array of presentations featured cutting-edge therapeutics for inherited retinal diseases, rare degenerative conditions, and even optic neuropathies:

• Gene therapies for inherited retinal disorders: Multiple biotechs shared updates on gene therapy trials. Ocugen, for example, detailed its modifier gene therapy platform with programs like OCU400 (targeting retinitis pigmentosa, now in a Phase 3 trial), OCU410 for Stargardt disease, and OCU410 for geographic atrophy – all in late-stage clinical testing. Opus Genetics revealed 12-month data from its Phase 1/2 trial of OPGx-LCA5 for Leber congenital amaurosis, showing sustained safety and hints of efficacy, and also presented preclinical results for gene therapies addressing MERTK and RDH12 mutations. These efforts aim to restore vision or halt degeneration in patients with inherited retinal dystrophies that were previously untreatable. In a similar vein, Beacon Therapeutics (via the DAWN trial) and AAVantgarde Bio shared promising early results for X-linked retinitis pigmentosa gene therapies, reflecting the broad industry commitment to genetic cures for blindness.

• Optogenetics for late-stage retinal disease: One of the most buzzed-about technologies is optogenetic therapy – using light-sensitive proteins to bypass damaged photoreceptors. Nanoscope Therapeutics reported 2.5-year (126-week) outcomes of its MCO-010 optogenetic therapy for advanced retinitis pigmentosa, delivered as a one-time intravitreal injection. Astonishingly, this approach – which enables remaining retinal cells to respond to light – is the first therapy shown to restore some visual function in patients with ultra-advanced RP. Attendees heard stories of previously blind individuals gaining navigational vision after treatment. Such results highlight how gene-based therapies (including optogenetics) are opening new horizons, effectively creating biological “bionic eyes” for those with end-stage disease.

• Cell therapies for cornea and optic nerve: Beyond the retina, ARVO featured progress in cell-based therapies for other ocular tissues. For instance, corneal regenerative medicine was discussed in the context of limbal stem cell transplantation and engineered corneal tissue – critical for patients with severe corneal damage. There is also growing interest in optic nerve protection and regeneration, where cell implants or exosome-based therapies might one day treat glaucoma or optic neuropathies. While many of these are in preclinical phases, the groundwork is being laid for clinical trials involving cell therapy in diverse indications.

Together, these gene and cell therapy advances are ushering in a new era of ophthalmology treatment. From the lab to the clinic, ophthalmology researchers are translating genomic discoveries and stem cell science into tangible therapies. Notably, ARVO 2025 even hosted dedicated sessions on gene therapy best practices, underlining that the community is actively addressing the practical aspects of trial design, patient enrollment, and regulatory pathways for these complex modalities. For sponsors, the message is clear: ophthalmology is at the cutting edge of biotech innovation, and having a development partner well-versed in ophthalmic gene/stem cell trials can be a key advantage.

With experienced teams and innovative technology, TFS HealthScience delivers efficient, high-quality results at every stage. Learn more about our Ophthalmology CRO Expertise.

Innovations in Imaging and AI for Ophthalmology

The intersection of imaging technology and artificial intelligence was another focal point at ARVO 2025. Advanced ocular imaging tools, from high-resolution optical coherence tomography (OCT) to ultra-widefield retinal cameras and adaptive optics systems, are generating unprecedented views of disease. Coupling these with AI-driven analysis is enabling earlier detection, objective endpoints for trials, and personalized treatment approaches:

• Enhancing diagnostics with AI: A highlight of the meeting was a provocative question posed by Prof. Pearse Keane, a pioneer of AI in ophthalmology: “Are you sick of AI yet?”Acknowledging the hype, Prof. Keane emphasized that AI in eye care is far from a fad – in fact, many powerful applications are still underappreciated. He outlined how by 2030, AI could transform clinical practice with differential diagnosis tools that integrate multimodal data, and “visual question-answering” systems where doctors input an eye image and receive instant clinical guidance. Importantly, Keane noted that AI’s impact extends beyond image analysis, addressing critical needs in ophthalmology where expert resources are limited. Indeed, several FDA-cleared AI systems are already in use – for example, autonomous algorithms now assist in detecting diabetic retinopathy from retinal photos. At ARVO, numerous abstracts explored AI for glaucoma progression, keratoconus screening, and even AI models predicting treatment responses. The consensus is that smart algorithms can augment clinicians, catching disease earlier and stratifying risk with greater precision.

• Imaging breakthroughs and digital endpoints: The Imaging in the Eye sessions at ARVO 2025 showcased how new imaging modalities are redefining what we can measure in clinical trials. Researchers presented cutting-edge results using adaptive optics retinal imaging, which visualizes individual cells in the living eye, to track photoreceptor changes in diseases like AMD. Others demonstrated ultra-widefield imaging that captures peripheral retinal pathology (crucial for diabetic retinopathy and uveitis trials) and functional OCT techniques that not only map anatomy but also blood flow and metabolism in ocular tissues. One notable poster, as mentioned, applied deep learning to OCT scans to delineate the suprachoroidal space for drug delivery research – combining imaging and AI to facilitate new therapies. Additionally, digital biomarkers are emerging: for instance, analyzing subtle eye movement patterns or visual field fluctuation with AI to quantify drug effects. The incorporation of these technologies into trial design could significantly enhance endpoint sensitivity and reduce trial sizes or durations. Sponsors are increasingly interested in such AI-driven ophthalmology innovations, as they promise to de-risk trials and support regulatory approvals with more robust data.

Expanding the Pipeline: Dry Eye Disease and Glaucoma Advances

While retinal diseases took center stage, ARVO 2025 also highlighted important developments in other high-impact ophthalmic indications, notably ocular surface diseases and glaucoma. These areas are essential for a comprehensive ophthalmology portfolio, as they affect millions and have significant unmet needs despite existing therapies.

Dry Eye Disease (DED): Chronic dry eye remains a widespread challenge, and innovators are seeking therapies that go beyond palliative relief to address underlying causes (inflammation, tear film instability, etc.). At the meeting, Iolyx Therapeutics presented updates on its lead candidate ILYX-002 for immune-driven dry eye, from a Phase 2 trial (ILYX-002-201). The data, shared in a dry eye session on May 8, indicated that targeting novel inflammatory pathways can yield significant improvements in patient symptoms and ocular surface health. This aligns with a broader trend of new mechanism-of-action drugs for dry eye (such as lymphocyte function-associated antigen inhibitors, kinases, and neurostimulatory agents) moving through clinical trials. Attendees also discussed real-world patient experience, with an emphasis on developing better endpoints (like measuring tear film biomarkers or using AI to analyze blink dynamics) to support DED drug approval. For sponsors in the ophthalmic space, the dry eye arena is heating up with competition, but also with opportunities for differentiation through improved formulations and precision medicine (e.g., identifying which subsets of DED patients respond to certain immunomodulators). Engaging a knowledgeable ophthalmology clinical research partner can help navigate these nuances in trial design and regulatory expectations for dry eye programs.

Glaucoma: The perennial goal in glaucoma research is not only lowering intraocular pressure (IOP) – which current drugs and surgeries achieve – but protecting the optic nerve from damage (neuroprotection) and tailoring treatment to individual risk profiles. ARVO 2025 provided glimpses into both fronts. In an award lecture, Dr. Alex Hewitt highlighted that the future is now for polygenic risk assessment in glaucoma, showing how genetic profiling might soon identify high-risk patients and guide earlier, more aggressive intervention. This approach could transform glaucoma screening by indicating which patients will benefit most from intensive monitoring or novel therapies, embodying the push toward personalized medicine. On the therapeutic side, a notable poster by TME Pharma and Singapore Eye Research Institute introduced an anti-scarring compound mNOX-E36 to improve outcomes of glaucoma filtration surgery. In preclinical studies, mNOX-E36 was as effective as mitomycin-C (the current standard anti-scarring agent) at reducing post-surgery fibrosis and inflammation, potentially leading to better long-term surgical success. Such an agent could be a game-changer in glaucoma surgery by making procedures safer and more reliable. Additionally, companies like ONL Therapeutics are advancing neuroprotective drugs aiming to prevent retinal ganglion cell death in glaucoma. Taken together, these developments underscore that glaucoma management is evolving, embracing genetics, novel pharmaceuticals, and improved surgical adjuncts, to tackle the disease from multiple angles.

Spotlight on Retinoblastoma: Awareness and Advances

In honor of World Retinoblastoma Awareness Week(May 11–17, 2025), ARVO 2025 placed a special emphasis on retinoblastoma research and progress. Retinoblastoma is a rare but critical pediatric eye cancer that affects young children, and global awareness efforts aim to promote early detection (often by noticing a child’s abnormal “white pupil” reflection) and access to care. At the ARVO meeting, ocular oncologists and researchers shared both scientific breakthroughs and public health insights that could improve outcomes for retinoblastoma patients worldwide.

Early detection and global disparities: A keynote from Dr. Swathi Kaliki, a leading clinician-scientist in ocular oncology, highlighted the urgent need for noninvasive screening strategies in retinoblastoma. With clinical experience managing thousands of cases, Dr. Kaliki’s research revealed striking differences in care and outcomes across 21 treatment centers on five continents. Her team has been identifying gaps (“lacunae”) in order to optimize retinoblastoma care, especially in low-resource settings. One promising avenue is using biomarkers and AI for earlier diagnosis. For example, Kaliki’s lab has studied small extracellular vesicles (sEVs) from retinoblastoma tumors, discovering molecular signatures that distinguish tumors with high-risk features. This could lead to a blood or urine test for retinoblastoma activity, complementing traditional exams. She emphasized integrating global research networks and artificial intelligence to “revolutionize global eye cancer outcomes” – hinting at tools like smartphone apps that detect leukocoria (the telltale white pupillary glow) in child photographs, which have already shown success in pilot studies. The push for awareness, teaching parents and primary doctors to recognize early signs, combined with novel screening tech, is expected to improve early catch of retinoblastoma, when it’s most curable.

Advances in treatment: On the clinical side, recent studies are refining how retinoblastoma is treated to maximize cure rates while minimizing harm to the child. A landmark multicenter trial published in late 2024 demonstrated that less chemotherapy may be just as effective for certain high-risk retinoblastoma patients. Traditionally, children with high-risk features after tumor removal (enucleation) receive 6 cycles of adjuvant chemotherapy (carboplatin, etoposide, vincristine). The trial, however, showed that 3 cycles of this regimen achieved equivalent 5-year disease-free survival (around 90%) as 6 cycles, with significantly fewer side effects. These findings suggest a new standard of care could emerge: using only three chemotherapy cycles post-surgery, thereby reducing toxicity and improving quality of life for young patients without compromising outcomes. Such evidence exemplifies how retinoblastoma clinical trials are directly influencing practice, making therapy safer for children. Additionally, ARVO sessions discussed targeted therapies under investigation – for instance, intra-arterial chemotherapy that delivers drugs straight to the eye, and exploratory use of targeted agents (like MEK inhibitors or immune checkpoint inhibitors) for rare metastatic cases. While these are mostly in early-phase research, they represent hope for more precise and less invasive retinoblastoma treatments in the future.

Overall, the retinoblastoma community is rallying around both science and awareness. Global collaborations and awareness campaigns each May are driving home the message that early detection saves lives. The progress from 2024–2025, as evident in ARVO presentations, is twofold: improve how soon we find the cancer (through awareness and novel screening) and improve how we treat the cancer (through optimized chemo, localized therapies, and biology-driven interventions). This dual approach is gradually closing the outcome gap between developed and developing regions, moving toward a world where no child dies or loses an eye due to retinoblastoma.

See how TFS HealthScience participated in World Retinoblastoma Awareness Week. 

Investment and Collaboration Trends in Ophthalmology

The flurry of innovation at ARVO 2025 also reflects a broader trend: significant investment activity in ophthalmic drug development. In recent years, ophthalmology has attracted major venture capital funding and partnership deals, signaling confidence in this specialty. Industry leaders at the meeting noted that dozens of ophthalmology startups and incubators are advancing new therapies, often showcased at venues like the Eyecelerator forum alongside ARVO. Notably, large pharmaceutical companies are actively investing in ophthalmology pipelines through acquisitions and alliances:

• As a prime example, in 2024 Merck announced it would acquire ophthalmology biotech EyeBio for up to $3 billion (including $1.3B upfront). EyeBio’s lead candidate, a first-in-class Wnt pathway agonist antibody for DME and wet AMD, is poised to enter pivotal trials. This high-value deal underscores big pharma’s appetite for innovative retinal treatments and validates the potential of mid-stage ophthalmology assets.

• Numerous smaller deals and funding rounds have fueled the field: e.g., companies like Character Biosciences (gene therapy for macular disease) and ONL Therapeutics (neuroprotective therapy for retinal detachment and glaucoma) each recently secured significant venture financing. Meanwhile, BIOPHYTIS and others raised seed rounds to progress novel eye disease therapies. According to industry analyses, the ophthalmology sector saw hundreds of millions in equity funding in the past year, even amid broader biotech headwinds. This reflects the compelling demographics (aging populations and prevalent diseases like AMD, glaucoma) and recent successes (approvals of therapies like gene therapy and complement inhibitors) that make ophthalmology an attractive investment area.

• Collaboration is also key: many biotechs are seeking experienced partners to conduct efficient trials and navigate regulatory hurdles in ophthalmology. We see sponsors teaming up with academic centers, foundations (for rare diseases), and specialized CROs to leverage domain expertise. For instance, several ARVO-highlighted programs (from inherited retinal disease gene therapies to stem cell trials) are backed by foundations like Foundation Fighting Blindness or government grants, in partnership with industry. This cross-sector collaboration accelerates progress while sharing risk and knowledge.

For sponsors and biotech executives, these trends emphasize the importance of aligning with the right partners. Whether it’s securing funding, co-development deals, or enlisting top-tier service providers, a collaborative strategy can greatly enhance the likelihood of success in ophthalmology R&D. The ophthalmology CRO landscape in particular has matured, with niche providers, like TFS HealthScience, building dedicated ophthalmic teams that understand the unique challenges – from complex ophthalmic endpoints and imaging to recruiting patients for specialized trials. Engaging a CRO that has a track record in ophthalmology clinical trials can bring invaluable operational excellence and strategic insight, ensuring that cutting-edge science is translated into a robust clinical development plan.

Conclusion: Partnering for Ophthalmology Clinical Research Success

The ARVO 2025 meeting made one thing abundantly clear: ophthalmology is a dynamic and rapidly advancing field, with innovations spanning from the front of the eye to the retina and beyond. Breakthroughs in therapies, be it a gene therapy that may preserve an aging person’s sight from macular degeneration, or a cell therapy that could give a child a second chance at vision, are closer than ever to reality. For companies driving these innovations, staying ahead means not only understanding the science but also executing efficient, adaptive clinical development programs. This is where choosing the right ophthalmology clinical research partner becomes crucial. An experienced partner brings deep knowledge of regulatory pathways, patient recruitment networks (including key ophthalmic centers of excellence), and technical expertise in specialized assessments like visual acuity, visual fields, OCT imaging, and more.

About TFS HealthScience

TFS HealthScience prides itself on being such a partner – a global ophthalmology CRO with comprehensive capabilities and a passion for ophthalmic innovation. We have supported trials across the spectrum of eye diseases, from rare pediatric conditions to large Phase III programs in AMD and glaucoma. Our teams are continually informed by the latest scientific insights and are equipped to implement novel trial designs, digital health tools, and patient-centric approaches in ophthalmology studies. In an environment where eye disease treatment innovation is accelerating, TFS stands ready to guide sponsors through complexity, mitigate risks, and ultimately bring life-changing ophthalmic therapies to patients worldwide. By leveraging the trends and knowledge outlined above – and partnering with experts who understand them – sponsors and biotechs can confidently navigate the road ahead in ophthalmology drug development. The future of vision research is bright, and together with the right collaborators, we can turn that scientific vision into reality for those who need it most.