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Disease
Eye Diseases
Late-stage therapeutic developmentEmerging researchRising momentum
13
Publications
19
Clinical trials
8
Related conditions
2024
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Research2024-11-01The oncologist
Research2024-10-28Molecular therapy : the journal of the American Society of Gene Therapy
Gut microbiota and eye diseases: A review.
Research2024-09-01Medicine
Research2024-01-19Journal of medical genetics
A foundation model for generalizable disease detection from retinal images.
Research2023-09-13Nature
Ocular Vascular Diseases: From Retinal Immune Privilege to Inflammation.
Research2023-07-28International journal of molecular sciences
Research2023-01-01Psychiatria Danubina
Exome sequencing and analysis of 454,787 UK Biobank participants.
Research2021-10-18Nature
Protein kinase CK2: a potential therapeutic target for diverse human diseases.
Research2021-05-17Signal transduction and targeted therapy
Research2020-12-01The Lancet. Global health
Research2017-12-01JAMA
Research2016-01-10Oxidative medicine and cellular longevity
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 1 regulatory approval from EMA on record.
- 2 clinical trials expected to report results, the earliest in Q4 2027.
Upcoming Milestones
- 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 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.
Major developments
Upcoming trial readoutHigh impact
Results expected Q4 20272026-06-25
Upcoming trial readoutHigh impact
Results expected Q4 20282026-05-15
Important regulatory approvalImportant
Nintedanib Viatris is indicated in adults for the treatment of idiopathic pulmonary fibrosis (IPF). Nintedanib Viatris is also indicated in adults for the treatment of other chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype. Nintedanib Viatris is indicated in children and adolescents from 6 to 17 years old for the treatment of clinically significant, progressive fibrosing interstitial lung diseases (ILDs). Nintedanib Viatris is indicated in adults, adolescents and children aged 6 years and older for the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD).2025-08-22
Clinical MilestonesViewHide
Results expected
- 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
- 2026-05-15Vitrectomy, 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
Recently completed
- 2026-01-31Evaluating the Impact of TRYPTYR on a Patient's Quality of Life and Ability to Perform WorkPrimary completion
- 2026-01-01Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for the Treatment of Non-arteritic Ischemic Optic NeuropathyPrimary completion
- 2025-10-01Effects of Topical Insulin on Corneal Epithelium Healing After Corneal Crosslinking in Patients With Keratoconus: A Randomized Clinical TrialPrimary completion
Regulatory UpdatesViewHide
- 2025-08-22Approval — NintedanibNintedanib Viatris is indicated in adults for the treatment of idiopathic pulmonary fibrosis (IPF). Nintedanib Viatris is also indicated in adults for the treatment of other chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype. Nintedanib Viatris is indicated in children and adolescents from 6 to 17 years old for the treatment of clinically significant, progressive fibrosing interstitial lung diseases (ILDs). Nintedanib Viatris is indicated in adults, adolescents and children aged 6 years and older for the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD).
Activity timeline6
- 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
- 2026-05-15ClinicalVitrectomy, 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
- 2026-01-31ClinicalEvaluating the Impact of TRYPTYR on a Patient's Quality of Life and Ability to Perform WorkPrimary completion
- 2026-01-01ClinicalSafety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for the Treatment of Non-arteritic Ischemic Optic NeuropathyPrimary completion
- 2025-10-01ClinicalEffects of Topical Insulin on Corneal Epithelium Healing After Corneal Crosslinking in Patients With Keratoconus: A Randomized Clinical TrialPrimary completion
- 2025-08-22RegulatoryApproval — NintedanibNintedanib Viatris is indicated in adults for the treatment of idiopathic pulmonary fibrosis (IPF). Nintedanib Viatris is also indicated in adults for the treatment of other chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype. Nintedanib Viatris is indicated in children and adolescents from 6 to 17 years old for the treatment of clinically significant, progressive fibrosing interstitial lung diseases (ILDs). Nintedanib Viatris is indicated in adults, adolescents and children aged 6 years and older for the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD).
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approved & established therapies
Methylphenidate hydrochlorideapproved
Approval — Tuzulby is indicated as part of a comprehensive treatment programme for attention-deficit… (2025)
Nintedanibapproved
Approval — Nintedanib Viatris is indicated in adults for the treatment of idiopathic pulmonary fibro… (2025)
Azathioprineapproved
Approval — Jayempi is indicated in combination with other immunosuppressive agents for the prophylax… (2021)
Thiotepaapproved
Approval — Thiotepa Riemser is indicated, in combination with other chemotherapy medicinal products:… (2021)
Melphalan hydrochlorideapproved
Approval — High-dose of Phelinun used alone or in combination with other cytotoxic medicinal product… (2020)
Treosulfanapproved
Approval — Treosulfan in combination with fludarabine is indicated as part of conditioning treatment… (2019)
Carmustineapproved
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.
Clinical programme
19
6
11
6
Late-stage studies
Recruiting
Recently completed
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
First approvals
2025emaApproval — Nintedanib· Nintedanib Viatris is indicated in adults for the treatment of idiopathic pulmonary fibrosis (IPF). Nintedanib Viatris is also indicated in adults for the treatment of other chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype. Nintedanib Viatris is indicated in children and adolescents from 6 to 17 years old for the treatment of clinically significant, progressive fibrosing interstitial lung diseases (ILDs). Nintedanib Viatris is indicated in adults, adolescents and children aged 6 years and older for the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD). source ↗
2025emaApproval — Methylphenidate hydrochloride· Tuzulby is indicated as part of a comprehensive treatment programme for attention-deficit / hyperactivity disorder (ADHD) in children and adolescents 6-17 years old when remedial measures alone prove insufficient. Treatment must be under the supervision of a specialist in childhood behavioural disorders. Diagnosis should be made according to Diagnostic and Statistical Manual of Mental Disorders Fourth Edition (DSM-IV) criteria or the guidelines in International Classification of Diseases, Tenth Revision (ICD-10) and should be based on a complete history and evaluation of the patient. Diagnosis cannot be made solely on the presence of one or more symptoms. source ↗
2024emaApproval — Nintedanib· Nintedanib Accord is indicated in adults for the treatment of idiopathic pulmonary fibrosis (IPF). Nintedanib Accord is also indicated in adults for the treatment of other chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype (see section 5.1). Nintedanib Accord is indicated in adults for the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD). Nintedanib Accord is indicated in adults for the treatment of idiopathic pulmonary fibrosis (IPF). Nintedanib Accord is also indicated in adults for the treatment of other chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype. Nintedanib Accord is indicated in children and adolescents from 6 to 17 years old for the treatment of clinically significant, progressive fibrosing interstitial lung diseases (ILDs). Nintedanib Accord is indicated in adults, adolescents and children aged 6 years and older for the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD). source ↗
2021emaApproval — Azathioprine· Jayempi is indicated in combination with other immunosuppressive agents for the prophylaxis of transplant rejection in patients receiving allogenic kidney, liver, heart, lung or pancreas transplants. Azathioprine is indicated in immunosuppressive regimens as an adjunct to immunosuppressive agents that form the mainstay of treatment (basis immunosuppression). Jayempi is used as an immunosuppressant antimetabolite either alone or, more commonly, in combination with other agents (usually corticosteroids) and/ or procedures which influence the immune response. Jayempi is indicated in patients who are intolerant to glucocorticosteroids or if the therapeutic response is inadequate despite treatment with high doses of glucocorticosteroids, in the following diseases: severe active rheumatoid arthritis (chronic polyarthritis) that cannot be kept under control by less toxic agents (disease-modifying anti-rheumatic -medicinal products – DMARDs) auto-immune hepatitis systemic lupus erythematosus dermatomyositis polyarteritis nodosa pemphigus vulgaris and bullous pemphigoid Behçet’s disease refractory auto-immune haemolytic anaemia, caused by warm IgG antibodies chronic refractory idiopathic thrombocytopenic purpura Jayempi is used for the treatment of moderately severe to severe forms of chronic inflammatory bowel disease (IBD) (Crohn’s disease or ulcerative colitis) in patients in whom glucocorticosteroid therapy is necessary, but where glucocorticosteroids are not tolerated, or in whom the disease is untreatable with other common means of first choice. It is also indicated in adult patients in relapsing multiple sclerosis, if an immunomodulatory therapy is indicated but beta interferon therapy is not possible, or a stable course has been achieved with previous treatment with azathioprine. 3 Jayempi is indicated for the treatment of generalised myasthenia gravis. Depending on the severity of the disease, Jayempi should be given in combination with glucocorticosteroids because of slow onset of action at the beginning of treatment and the glucocorticosteroid dose should be gradually reduced after several months of treatment. source ↗
2021emaApproval — Thiotepa· Thiotepa Riemser is indicated, in combination with other chemotherapy medicinal products: with or without total body irradiation (TBI), as conditioning treatment prior to allogeneic or autologous haematopoietic progenitor cell transplantation (HPCT) in haematological diseases in adult and paediatric patients; when high dose chemotherapy with HPCT support is appropriate for the treatment of solid tumours in adult and paediatric patients. Thiotepa Riemser is indicated, in combination with other chemotherapy medicinal products: with or without total body irradiation (TBI), as conditioning treatment prior to allogeneic or autologous haematopoietic progenitor cell transplantation (HPCT) in haematological diseases in adult and paediatric patients; when high dose chemotherapy with HPCT support is appropriate for the treatment of solid tumours in adult and paediatric patients source ↗
2020emaApproval — Melphalan hydrochloride· High-dose of Phelinun used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: multiple myeloma, malignant lymphoma (Hodgkin, non-Hodgkin lymphoma), acute lymphoblastic and myeloblastic leukemia, childhood neuroblastoma, ovarian cancer, mammary adenocarcinoma. Phelinun in combination with other cytotoxic medicinal products is indicated as reduced intensity conditioning (RIC) treatment prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in malignant haematological diseases in adults. Phelinun in combination with other cytotoxic medicinal products is indicated as conditioning regimen prior to allogeneic haematopoietic stem cell transplantation in haematological diseases in the paediatric population as: Myeloablative conditioning (MAC) treatment in case of malignant haematological diseases RIC treatment in case of non-malignant haematological diseases. source ↗
2019emaApproval — Treosulfan· Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients and in paediatric patients older than one month with malignant and non-malignant diseases. source ↗
2018emaApproval — Carmustine· Carmustine is indicated n adults in the following malignant neoplasms as a single agent or in combination with other antineoplastic agents and/or other therapeutic measures (radiotherapy, surgery): Brain tumours (glioblastoma, brain-stem gliomas, medulloblastoma, astrocytoma and ependymoma), brain metastases Secondary therapy in non-Hodgkin’s lymphoma and Hodgkin’s disease as conditioning treatment prior to autologous haematopoietic progenitor cell transplantation (HPCT) in malignant haematological diseases (Hodgkin’s disease / Non-hodgkin’s lymphoma). source ↗
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.
Publications over time
20162024
Most influential
Recent publications
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
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.
Overview
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.
Causes
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.
Pathophysiology
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.
Risk factors
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.
Current standard of care
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.
Defined in MeSH
Diseases affecting the eye.
Identifiers
References & data sources
- 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.