Ophthalmology clinical trials: a specialist CRO’s perspective

Insights from EURETINA 2026, Vienna

Can a single treatment reduce years of eye injections? Can retinal implants provide lasting improvements in central vision? And how can clinical trials better measure changes that matter to patients? These questions featured in research presented at EURETINA 2026, held in Vienna from 1–4 October. The congress brought together the international retinal research community to share clinical evidence and explore new approaches to treatment.

Selection of images from Euretina 2026
Credit Euretina Congress Daily

PHARMExcel’s Business Development Manager, Paresh Parmar, attended to meet clients and sponsors. As a CRO specialising in ophthalmology clinical research, these conversations are an opportunity to understand sponsors’ study needs and discuss how PHARMExcel can support their programmes.

The developments below capture some of the promising results reported at the congress, alongside the questions that further research still needs to answer.

  • Wet AMD: can gene therapy reduce repeated injections?
    Gene therapy programmes for neovascular, or wet, age-related macular degeneration are investigating whether one treatment can provide sustained disease control. At EURETINA, longer follow-up helped researchers assess whether reductions in injection burden were maintained alongside visual and anatomical outcomes. Source: EURETINA late-breaking trials report. euretinacongressdaily.org
  • Sura-vec — five-year Phase I/IIa follow-up
    Two dose cohorts maintained or improved vision, with injection burden reduced by more than 64% compared with their pre-enrolment treatment history. No new safety signals related to the gene therapy or its administration were reported. euretinacongressdaily.org
  • SAR402663 — Phase I/II gene therapy study
    More than 90% of patients receiving the selected medium dose remained free from supplementary injections. The dose has progressed into a Phase III programme, with longer-term follow-up continuing. euretinacongressdaily.org
  • PRISM / 4D-150 — two-year Phase IIb findings
    Average visual and anatomical benefits were maintained alongside a sustained reduction in treatment burden following one injection. No new safety or intraocular inflammation signals emerged during follow-up. euretinacongressdaily.org
  • PRIMAvera — retinal implant for geographic atrophy, 36-month outcomes
    Among the 18 patients assessed at 36 months, 78% gained at least two lines of visual acuity, while natural vision remained stable. The findings suggest sustained benefit, although the small final follow-up group limits interpretation. Source: EURETINA congress highlights. org
  • PST-611 — first-in-human non-viral gene therapy study
    PulseSight reported favourable safety and tolerability at both doses in six patients, with early functional and anatomical signals in five. These are preliminary findings from a small study and require confirmation. Source: PulseSight. com
  • Eyecyte-RPE — investigational retinal cell therapy
    The investigator reported an approximately 11-letter mean vision gain maintained at around 18 months in Phase I, with similar early gains in Phase II. Phase II follow-up remains short; one patient developed endophthalmitis. Source: investigator interview, Ophthalmology Times. ophthalmologytimes.com

Inherited retinal disease: early signals of functional benefit

  • TTX-381 — CLN2 Batten disease-related retinopathy
    Phase I/II findings reported photoreceptor stabilisation or improvement in all five fully treated eyes during available follow-up. Preliminary functional observations also suggested stable vision after a single injection. euretinacongressdaily.org
  • NN-CNGA1 — CNGA1-related retinitis pigmentosa
    In a six-patient Phase Ib study, improved rod-related function was reported in the higher-dose group, with no efficacy signal at the lower dose. Intraocular inflammation was controlled with oral steroids. euretinacongressdaily.org

Source for the inherited retinal disease studies: EURETINA late-breaking trials report.

How do we measure meaningful improvements in retinal function?

Illustration of a retina showing signs of retinopathy, including yellow exudates, haemorrhages and pale cotton wool spots around the optic disc.An important research question extends beyond the treatment itself: which assessments can reliably detect changes in vision?

Baseline findings from the Phase II eDREAM trial supported patient-tailored microperimetry as a repeatable functional assessment in geographic atrophy. Microperimetry measures sensitivity at specific retinal locations; combining it with optical coherence tomography showed potential to identify areas of reduced or absent vision.

For researchers, the distinction matters: these findings concern the performance of an assessment method. They do not establish treatment efficacy for the investigational drug GAL-101. Source: Galimedix. rss.globenewswire.com

Robotic surgery: precision in treatment delivery

The Lotus study explored robotic assistance for subretinal injection in 20 eyes with submacular haemorrhage. It met its primary procedural endpoints, with successful cannula placement and no robot-related adverse events. Complete blood displacement was reported in 95% of eyes.

The study also identified practical limitations, including depth perception and the system’s range of movement. Its haemorrhage model does not directly establish performance in gene-therapy delivery, but it provides clinical evidence for further investigation of robotic assistance. Source: EURETINA robotics report. euretinacongressdaily.org

Where is ophthalmology research heading?

The programmes presented in Vienna are pursuing longer-lasting treatment, preservation of retinal function and restoration of vision. Alongside therapeutic development, researchers are investigating more precise delivery and more sensitive ways to assess functional outcomes.

Encouraging results also brought unresolved questions. The congress closing talk noted that some gene-therapy findings were not fully corroborated by other presentations. Larger studies and longer follow-up remain essential to determining which approaches deliver durable benefits and an acceptable safety profile.

For PHARMExcel, ophthalmology research is a specialist area of clinical trial delivery. Paresh’s attendance provided an opportunity to connect with clients and sponsors as these programmes continue to develop.

Planning an ophthalmology clinical trial? Tell us about your study needs and discuss your programme with PHARMExcel.

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