Eye Diseases
Recent clinical, regulatory, research and industry developments relating to this disease.
Gut microbiota and eye diseases: A review.
A foundation model for generalizable disease detection from retinal images.
Ocular Vascular Diseases: From Retinal Immune Privilege to Inflammation.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 4 clinical trials expected to report results, the earliest in Q4 2027.
- 1 industry development reported.
- Q4 2027A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene
- Q4 2027Efficacy of Dapagliflozin in the Progression of Geographic Atrophy Secondary to Age-Related Macular Degeneration
- Q1 2028A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 (30 μg and 75 μg) Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001 in the PLATYPUS Study (Protocol # VP001-101) or WALLABY Study (Protocol # VP001-102) for a Minimum of 8 Weeks
- Q4 2028Vitrectomy, Subretinal Tissue Plasminogen Activator and Intravitreal Gas for Submacular Haemorrhage Secondary to Exudative Age-Related Macular Degeneration (TIGER): a Phase 3, Pan-European, Two-group, Observer-masked, Superiority, Randomised Controlled Surgical Trial.
Clinical MilestonesViewHide
- 2026-09-11Vitrectomy, Subretinal Tissue Plasminogen Activator and Intravitreal Gas for Submacular Haemorrhage Secondary to Exudative Age-Related Macular Degeneration (TIGER): a Phase 3, Pan-European, Two-group, Observer-masked, Superiority, Randomised Controlled Surgical Trial.Results expected Q4 2028
- 2026-08-26Efficacy of Dapagliflozin in the Progression of Geographic Atrophy Secondary to Age-Related Macular DegenerationResults expected Q4 2027
- 2026-06-25A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 GeneResults expected Q4 2027
- 2026-05-29A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 (30 μg and 75 μg) Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001 in the PLATYPUS Study (Protocol # VP001-101) or WALLABY Study (Protocol # VP001-102) for a Minimum of 8 WeeksResults expected Q1 2028
- 2026-06-30A Multicenter, Extension Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Two Doses of Linsitinib in Subjects With Active, Moderate to Severe Thyroid Eye Disease (TED)Primary completion
- 2026-01-31Evaluating the Impact of TRYPTYR on a Patient's Quality of Life and Ability to Perform WorkPrimary completion
Industry & MarketViewHide
- 2026-09-10Opus BEST1 and done? Gene therapy scores in rare eye diseasesIndustry · BioWorld Gene Therapy
- 2026-09-11ClinicalVitrectomy, Subretinal Tissue Plasminogen Activator and Intravitreal Gas for Submacular Haemorrhage Secondary to Exudative Age-Related Macular Degeneration (TIGER): a Phase 3, Pan-European, Two-group, Observer-masked, Superiority, Randomised Controlled Surgical Trial.Results expected Q4 2028
- 2026-09-10IndustryOpus BEST1 and done? Gene therapy scores in rare eye diseasesIndustry · BioWorld Gene Therapy
- 2026-08-26ClinicalEfficacy of Dapagliflozin in the Progression of Geographic Atrophy Secondary to Age-Related Macular DegenerationResults expected Q4 2027
- 2026-06-30ClinicalA Multicenter, Extension Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Two Doses of Linsitinib in Subjects With Active, Moderate to Severe Thyroid Eye Disease (TED)Primary completion
- 2026-06-25ClinicalA Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 GeneResults expected Q4 2027
- 2026-05-29ClinicalA Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 (30 μg and 75 μg) Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001 in the PLATYPUS Study (Protocol # VP001-101) or WALLABY Study (Protocol # VP001-102) for a Minimum of 8 WeeksResults expected Q1 2028
- 2026-01-31ClinicalEvaluating the Impact of TRYPTYR on a Patient's Quality of Life and Ability to Perform WorkPrimary completion
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Original application approval — Tuzulby is indicated as part of a comprehensive treatment programme for attention-deficit… (2025)
Generic approval — Nintedanib Viatris is indicated in adults for the treatment of idiopathic pulmonary fibro… (2025)
Original application approval — Jayempi is indicated in combination with other immunosuppressive agents for the prophylax… (2021)
Generic approval — Thiotepa Riemser is indicated, in combination with other chemotherapy medicinal products:… (2021)
Original application approval — High-dose of Phelinun used alone or in combination with other cytotoxic medicinal product… (2020)
Original application approval — Treosulfan in combination with fludarabine is indicated as part of conditioning treatment… (2019)
Generic approval — Carmustine is indicated n adults in the following malignant neoplasms as a single ag… (2018)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Eye Diseases6
- Mental Disorders2
- Artificial Intelligence1
- Biological Specimen Banks1
- Cardiovascular Diseases1
- Casein Kinase II1
- COVID-191
- COVID-19 Drug Treatment1
Leading journals6
- Nature2
- International journal of molecular sciences1
- JAMA1
- Journal of medical genetics1
- Medicine1
- Molecular therapy : the journal of the American Society of Gene Therapy1
Leading researchers8
- Bressler NM2
- Keane PA2
- Ting DSW2
- Wong TY2
- Abecasis GR1
- Abramoff M1
- Admard J1
- Alexander DC1
Affiliations (unnormalised)6
- Singapore Eye Research Institute2
- Center for Rare Disease1
- Centre for Medical Image Computing1
- Chinese University of Hong Kong1
- CNR Institute of Neuroscience1
- Departments of Ophthalmology & Medical Informatics and Clinical Epidemiology1
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Eye diseases are disorders that affect the eye and can lead to visual impairment or blindness. The literature emphasizes that many causes of vision loss are preventable or treatable, and that eye disease spans both anterior and posterior segment conditions.
The supplied grounding supports a broad, heterogeneous aetiology rather than a single cause. Reported contributors include genetic disease, infectious causes, immune/inflammatory processes, oxidative stress, and age-related or degenerative mechanisms.
The biological mechanisms vary by specific eye disorder. Grounding highlights reactive oxygen species and oxidative stress, immune privilege failure with inflammatory activation, apoptosis of ocular cells, and disease-related signaling pathways; it also notes that exome-based genetics and enzymology are relevant to the literature.
The grounding supports ageing as a major contextual risk factor for eye disease burden. It also indicates that inherited variants, immune/inflammatory dysregulation, and infectious exposures can increase risk for specific ocular diseases.
Management is described at a broad modality level and includes diagnosis, imaging, therapy, and drug therapy. The literature also supports gene therapy for selected inherited ocular diseases and the use of artificial intelligence-assisted image analysis for detection of conditions such as diabetic retinopathy, glaucoma, age-related macular degeneration, retinopathy of prematurity, and refractive error-related disease.
AI-generated summary grounded in MeSH and 6 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Diseases affecting the eye.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.