Description of the Action
Enabling Translation of Retinal Disease Diagnosis and Therapies: A Roadmap for Future (Retina4Future)
Retinal diseases are significant contributors to visual impairment, imposing a substantial global economic and well-being burden. Once retinal degeneration occurs, there are currently no therapeutic solutions that can completely cure retinal diseases. It is imperative to prioritize efforts aimed at accelerating European research translation in the field of retinal disease therapies.
This includes improving early diagnosis, enhancing retinal imaging tools and functional diagnostics, and identifying new structural, functional, and molecular biomarkers for disease and its progression.
Additionally, it is crucial to develop innovative therapeutic approaches for the early stages of these diseases to prevent their progression into severe forms that could compromise vision. These developments have the potential to control the progression of retinal diseases before they adversely affect vision.
The primary objective of Retina4Future is to expedite the translation of European vision research into novel biomarkers and advanced therapies for retinal diseases. This will be achieved by establishing a research and innovation network and promoting interdisciplinary research. To accomplish this, the network will encompass a diverse group of contributors, including basic and clinical researchers, clinicians, small and medium-sized enterprises (SMEs), patient organizations, ethics experts and policymakers.
The ultimate goal is to improve the quality of life and healthcare for patients and their families while also fostering the career development of young researchers and innovators. This COST will benefit clinical, social, and economic sectors in Europe.
Scientific Challenges
Main Challenges in Retinal Disease Research and Translation
Despite significant scientific progress, several key challenges continue to limit the effective translation of retinal research into clinical practice. Earlier diagnosis, better biomarkers and stronger integration between research, clinics and innovation remain critical needs.
Late Diagnosis and Limited Early Biomarkers
Many retinal diseases progress silently, with clinical symptoms appearing only after irreversible damage has occurred. Current diagnostic tools and clinical endpoints often fail to detect early-stage disease, limiting opportunities for timely intervention. There is a critical need for robust structural, functional and molecular biomarkers capable of identifying early pathological changes and monitoring disease progression.
Gaps Between Preclinical Models and Human Disease
Although animal models and emerging human-based systems (such as retinal organoids and microphysiological systems) have advanced retinal research, no single model fully replicates human retinal disease. Limited standardization, incomplete disease representation and insufficient cross-validation reduce translational efficiency.
Challenges in Therapeutic Delivery to the Retina
The eye presents unique anatomical and physiological barriers to treatment. While intraocular injections are effective, they are invasive and associated with risks. Alternative delivery routes often suffer from low bioavailability. Innovative, safe and efficient drug and gene delivery systems remain a major unmet need.
Bottlenecks in Drug Discovery and Advanced Therapies
Developing new therapies is constrained by incomplete understanding of disease mechanisms, limited access to suitable screening platforms and challenges in scaling advanced therapies such as gene and regenerative medicine. Although drug repurposing offers opportunities, systematic approaches and shared resources are still lacking.
Limitations in Clinical Trial Design
Traditional clinical endpoints, such as visual acuity, often change only in late disease stages, making them unsuitable for early intervention trials. The absence of validated surrogate endpoints and harmonized methodologies complicates trial design, particularly for rare retinal diseases with small patient cohorts.
Fragmentation of Data and Expertise
Retinal research expertise, datasets and technologies are distributed across Europe but remain fragmented. Limited data sharing, lack of standardized protocols and insufficient integration of AI, big data and clinical registries hinder large-scale analysis and robust conclusions.
Insufficient Integration of Patient Perspectives
Patient-reported outcomes, quality-of-life measures and patient priorities are not consistently integrated into research and clinical development. Stronger patient-oriented research frameworks are needed to ensure relevance, acceptance and long-term impact.
Regulatory, Economic and Societal Barriers
Translating innovation to clinical and market adoption requires alignment with regulatory frameworks, health technology assessment and reimbursement pathways. Economic sustainability, public trust in data use and equitable access to innovation remain cross-cutting challenges.
Specific Objectives
Research and Capacity-Building Objectives
Research Coordination Objectives (RCO)
Develop strategies for early diagnosis by integrating AI-enhanced retinal imaging with centralised clinical sample data to identify novel biomarkers.
Assess and harmonise preclinical models, samples and protocols to improve reproducibility and translational relevance.
Advance the discovery of new therapeutic targets through collaboration on molecular mechanisms of retinal diseases.
Improve drug, gene and advanced therapy delivery systems, addressing key bottlenecks from discovery to clinical validation.
Harmonise clinical study and trial methodologies, defining biomarkers and surrogate endpoints to strengthen translational research.
Promote market-driven translational research by facilitating SME access to fundamental research, patients and clinical centres.
Embed patient-driven research and advocacy, ensuring Patient and Public Involvement and Engagement (PPIE) across all activities.
Increase awareness among patients and the public on early diagnosis, emerging therapies and participation in clinical studies.
Capacity-building Objectives (CBO)
Build a transdisciplinary European research and innovation network to translate biomarkers, diagnostics and therapies for retinal diseases.
Attract and empower Young Researchers and Innovators (YRIs), women and Inclusiveness Target Countries (ITCs) through leadership and training opportunities.
Create a central online platform connecting researchers, clinicians, patients, innovators and SMEs to support collaboration and sustainability.
Develop shared inventories and resources, including biomarkers, clinical samples, preclinical models, delivery systems and clinical expertise.
Identify priority areas for new clinical studies and trials by addressing scientific, technical and logistical gaps.
Strengthen YRI–SME collaboration to build transferable skills and industry-relevant experience.
Facilitate knowledge and technology transfer between academia and industry to accelerate innovation and commercial potential.
Actively involve patient organisations in defining research priorities and disseminating outcomes.