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Key Takeaways from ARVO and ASGCT 2026

Recent presentations at ARVO and ASGCT highlighted several important developments in the treatment landscape for geographic atrophy (GA) and inherited retinal diseases, reinforcing growing industry interest in durable gene therapy and approaches that combine neuroprotection with complement pathway modulation.

1. The Field Is Moving Beyond Complement Monotherapy

Multiple real-world studies presented at ARVO highlighted the challenges and limitations associated with complement inhibitor monotherapies. Real world patient compliance is relatively poor due to the low visual benefit and risk of new-onset wet AMD. There is a growing consensus on the importance of a durable treatment and the ability to address multiple drivers of GA and retinal degeneration.

2. Growing Validation of Neuroprotection and PEDF Biology

There was convergence of evidence supporting pigment epithelium-derived factor (PEDF) as a neuroprotective strategy for retinal disease. New basic science presentations further elucidated PEDF receptor biology and downstream signaling pathways associated with photoreceptor and retinal pigment epithelium (RPE) protection. Complementary translational studies demonstrated photoreceptor rescue and preservation across multiple disease models and delivery approaches. Importantly, clinical data presented from an investigator-sponsored study of a PEDF gene therapy demonstrated encouraging long-term visual outcomes, supporting the clinical therapeutic potential of PEDF-mediated neuroprotection in retinal degeneration.

3. Independent Validation of Dual-Pathway Therapeutic Design

Other scientific presentations independently converged on therapeutic architectures combining multiple mechanisms of action to target complex disease pathologies including addition neurotrophic or neuroprotective factors to treat GA. These programs provide additional validation for the broader scientific rationale that addressing both inflammatory and degenerative pathways may be necessary to achieve meaningful clinical benefit in retinal disease.

4. Benefits of Gene Therapy Durability

Gene therapy offers a durable and steady approach compared to frequent eye injections that cause drug levels to rise and fall over time. These fluctuations, along with the challenge of keeping up with repeated treatments, can reduce real-world effectiveness. In eyes with an inherited retinal dystrophy, it was shown that a single subretinal dose of an approved AAV gene therapy improved visual function over 5 years. Another Phase 2 study found that patients receiving an anti-VEGF subretinal gene therapy had a more stable retinal thickness that tended to achieve better vision outcomes while requiring fewer supplemental injections.

As a result, the value of gene therapy may extend beyond reducing treatment burden. By delivering a steady, long-lasting therapeutic effect, it may also help improve visual outcomes compared with treatments that require frequent repeat dosing.

5. Ikarovec’s IKAR-001 gene therapy for Geographic Atrophy

IKAR-001 brings together all four key themes. It is a one-time subretinal gene therapy for geographic atrophy that goes beyond complement inhibition alone. The therapy combines PEDF-driven neuroprotection with complement regulation in a single construct, and is designed to preserve visual function, which current approved therapies do not. This dual approach reflects the direction the field is increasingly taking. By using sustained gene expression, IKAR-001 is designed to provide long-lasting, steady treatment without the fixed, frequent injections that current approved therapies require. In many ways, IKAR-001 was built around the same principles now being highlighted by ARVO and ASGCT.