Restore Vision’s latest progress
Where do we stand now in our journey of advancing treatments for rare eye diseases in Europe? The past period have marked significant progress – from preclinical work, ensuring safe and effective formulations, to moving toward clinical trials. Beyond the lab, we have also moved forward in navigating ethical and regulatory pathways, as well as in communication and patient involvement efforts. Explore our latest developments in each field.
Human Studies and Patient Involvement
A central strand of RESTORE VISION is the collection of patient samples to confirm therapeutic targets of the drugs we are investigating. Recruitment has begun at Ospedale San Rafaelle in Italy, where 74 participants are already enrolled, and approvals are now in place for expansion in France (INSERM), and more recently in Germany (University of Cologne). Samples such as tears, conjunctival cells and blood are being analysed to identify key genetic and protein markers linked to rare eye diseases.
Progress in Preclinical Research
Significant advances were reported in laboratory and animal models. Spironolactone showed particularly strong regenerative and anti-inflammatory effects, positioning it as a leading candidate for translation into clinical testing within the project. Olisens demonstrated encouraging results in long-term studies, supporting its move toward formal trials.
Other compounds are being evaluated in parallel. Extracellular vesicles derived from mesenchymal stromal cells showed early promise and will be tested in several pre-clinical models of corneal disease, while Duloxetine studies require further optimisation and will be re-tested at different time points. AB10 is being investigated as a novel therapeutic drug stimulating nerve activity and corneal nerve regeneration. It has shown promise in in vitro models with preclinical testing ongoing. New accelerated disease models have been developed, including advanced genetic mouse lines, to better reflect patient conditions and enable more predictive drug testing.
These preclinical results not only highlight which treatments may have the greatest potential to explore within the project but also provide valuable insight into the biological mechanisms underlying disease and therapy.
Ensuring Safe and Effective Formulations
Another key focus has been the development of safe and stable drug formulations. Eye-drop preparations of Spironolactone and Losartan have been optimised, while Aprepitant has required the design of new microsuspension techniques to overcome solubility and safety issues. A novel eye drop formulation is being developed for Prima-1MET, which will be investigated for its potential therapeutic benefits. Modern analytical methods are being introduced to track how drugs and their metabolites behave in the eye, which will guide safe dosing and prepare for future regulatory discussions.
From Pre-clinical research to Clinical Trials
Looking ahead, preparations are advancing for first-in-human pilot clinical trials. The first formal submissions for two Olisens trials have been achieved, with a trial submission for Mesenchymal stromal cells to follow soon. Trial submissions for additional candidates such as Spironolactone will follow as soon as regulatory and formulation requirements are complete. These trials will represent an important milestone for RESTORE VISION, bringing innovative therapies into the clinic for the first time.
Ethics and Regulatory Pathways
Regulatory and ethics experts continue to support the consortium in the review and development of required clinical documentation. Material transfer agreements and regulatory dossiers are in development, and Orphan Drug Designation applications have been prepared for compounds such as Spironolactone and Olisens, which could help accelerate their path to the clinic.
Communication, Engagement and Impact
The project continues to expand its visibility. Over the past year, partners have presented at 11 international conferences, published four scientific articles, launched different promotional materials and shared updates through newsletters and social media. Patient representatives remain closely involved in communication activities to ensure that outputs are meaningful and relevant.
Looking Ahead
The Alicante meeting showed a consortium making steady progress across scientific, clinical, and outreach dimensions. While challenges remain in ethics approvals, formulation, and regulatory readiness, the project is moving closer to delivering its first clinical trials.
Next up, the consortium will meet in-person at the Annual Consortium Meeting in Milan in May 2026, hosted by Ospedale San Raffaele, where the consortium will review trial progress and prepare for the final stages of the project!