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Disease
Retinal Degeneration
Late-stage therapeutic developmentEmerging researchSteady momentum
4
Publications
17
Clinical trials
12
Associated genes
2023
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions.
Research2023-06-01Investigative ophthalmology & visual science
Microglia in Retinal Degeneration.
Research2019-08-20Frontiers in immunology
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia.
Research2018-10-08Nature medicine
The human ATP-binding cassette (ABC) transporter superfamily.
Research2001-07-01Journal of lipid research
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.
- 4 clinical trials expected to report results, the earliest in Q4 2027.
- 1 industry development reported.
Upcoming Milestones
- 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
- Q1 2028Oral Metformin for Treatment of ABCA4 Retinopathy
- Q4 2029A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
Major developments
Important regulatory approvalHigh impact
Vislyfa is indicated in adults for: The treatment of neovascular (wet) age-related macular degeneration (AMD) The treatment of visual impairment due to diabetic macular oedema (DME) The treatment of proliferative diabetic retinopathy (PDR) The treatment of visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) The treatment of visual impairment due to choroidal neovascularisation (CNV)2026-07-15
Upcoming trial readoutImportant
Results expected Q4 20292026-07-06
Upcoming trial readoutImportant
Results expected Q1 20282026-06-29
Clinical MilestonesViewHide
Results expected
- 2026-07-06A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor DiseaseResults expected Q4 2029
- 2026-06-29Oral Metformin for Treatment of ABCA4 RetinopathyResults expected Q1 2028
- 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
- 2025-12-18Efficacy of Dapagliflozin in the Progression of Geographic Atrophy Secondary to Age-Related Macular DegenerationResults expected Q4 2027
Recently completed
- 2025-12-31The Efficacy and Safety of Minocycline for Chronic Autoimmune UveitisPrimary completion
- 2025-10-01Randomized, Double-blind, Parallel, Active Controlled Study to Compare Efficacy & Safety Between Ranibizumab 10mg/ml Injection of Incepta and Lucentis in Patients With Diabetic Macular Edema by ITV InjectionPrimary completion
Industry & MarketViewHide
- 2026-05-22Centripetal Retinal Degeneration Follows an Exponential Decay and May Be Reversed Following Gene TherapyIndustry · American Journal of Ophthalmology
Regulatory UpdatesViewHide
- 2026-07-15Approval — RanibizumabVislyfa is indicated in adults for: The treatment of neovascular (wet) age-related macular degeneration (AMD) The treatment of visual impairment due to diabetic macular oedema (DME) The treatment of proliferative diabetic retinopathy (PDR) The treatment of visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) The treatment of visual impairment due to choroidal neovascularisation (CNV)
Activity timeline8
- 2026-07-15RegulatoryApproval — RanibizumabVislyfa is indicated in adults for: The treatment of neovascular (wet) age-related macular degeneration (AMD) The treatment of visual impairment due to diabetic macular oedema (DME) The treatment of proliferative diabetic retinopathy (PDR) The treatment of visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) The treatment of visual impairment due to choroidal neovascularisation (CNV)
- 2026-07-06ClinicalA Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor DiseaseResults expected Q4 2029
- 2026-06-29ClinicalOral Metformin for Treatment of ABCA4 RetinopathyResults expected Q1 2028
- 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
- 2026-05-22IndustryCentripetal Retinal Degeneration Follows an Exponential Decay and May Be Reversed Following Gene TherapyIndustry · American Journal of Ophthalmology
- 2025-12-31ClinicalThe Efficacy and Safety of Minocycline for Chronic Autoimmune UveitisPrimary completion
- 2025-12-18ClinicalEfficacy of Dapagliflozin in the Progression of Geographic Atrophy Secondary to Age-Related Macular DegenerationResults expected Q4 2027
- 2025-10-01ClinicalRandomized, Double-blind, Parallel, Active Controlled Study to Compare Efficacy & Safety Between Ranibizumab 10mg/ml Injection of Incepta and Lucentis in Patients With Diabetic Macular Edema by ITV InjectionPrimary completion
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
Ranibizumabapproved
Approval — Vislyfa is indicated in adults for: The treatment of neovascular (wet) age-related macul… (2026)
Afliberceptapproved
Approval — Eyluxvi is indicated for adults for the treatment of- neovascular (wet) age-related macul… (2025)
Clinical trials
The current development programme across all trial phases.
Clinical programme
17
6
4
6
Late-stage studies
Recruiting
Recently completed
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
First approvals
2026emaApproval — Ranibizumab· Vislyfa is indicated in adults for: The treatment of neovascular (wet) age-related macular degeneration (AMD) The treatment of visual impairment due to diabetic macular oedema (DME) The treatment of proliferative diabetic retinopathy (PDR) The treatment of visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) The treatment of visual impairment due to choroidal neovascularisation (CNV) source ↗
2025emaApproval — Aflibercept· Eyluxvi is indicated for adults for the treatment of- neovascular (wet) age-related macular degeneration (AMD) (see section 5.1),- visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) (see section 5.1),- visual impairment due to diabetic macular oedema (DME) (see section 5.1),- visual impairment due to myopic choroidal neovascularisation (myopic CNV) (see section 5.1). source ↗
2025emaApproval — Aflibercept· Afiveg is indicated for adults for the treatment of neovascular (wet) age-related macular degeneration (AMD) (see section 1), visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) (see section 5.1), visual impairment due to diabetic macular oedema (DME) (see section1), visual impairment due to myopic choroidal neovascularisation (myopic CNV) (see section 5.1). source ↗
2025emaApproval — Aflibercept· Mynzepli is indicated for adults for the treatment of- neovascular (wet) age-related macular degeneration (AMD),- visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO),- visual impairment due to diabetic macular oedema (DME),- visual impairment due to myopic choroidal neovascularisation (myopic CNV) source ↗
2025emaApproval — Aflibercept· Eiyzey is indicated for adults for the treatment of- neovascular (wet) age-related macular degeneration (AMD) (see section 5.1),- visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) (see section 5.1),- visual impairment due to diabetic macular oedema (DME) (see section 5.1),- visual impairment due to myopic choroidal neovascularisation (myopic CNV) (see section 5.1)s. source ↗
2025emaApproval — Aflibercept· Vgenfli is indicated for adults for the treatment of- neovascular (wet) age-related macular degeneration (AMD),- visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO),- visual impairment due to diabetic macular oedema (DME),- visual impairment due to myopic choroidal neovascularisation (myopic CNV). source ↗
2025emaApproval — Aflibercept· Eydenzelt is indicated for adults for the treatment of: neovascular (wet) age-related macular degeneration (AMD) (see section 5.1), visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) (see section 5.1), visual impairment due to diabetic macular oedema (DME) (see section 5.1), visual impairment due to myopic choroidal neovascularisation (myopic CNV) (see section 5.1). source ↗
2025emaApproval — Aflibercept· Ahzantive is indicated for adults for the treatment of • neovascular (wet) age-related macular degeneration (AMD) (see section 5.1), • visual impairment due to macular oedema secondary to retinal vein occlusion (branch RVO or central RVO) (see section 5.1), • visual impairment due to diabetic macular oedema (DME) (see section 5.1), • visual impairment due to myopic choroidal neovascularisation (myopic CNV) (see section 5.1). 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
20012023
Most influential
Major themes2
- Genetic Therapy1
- Retinal Degeneration1
Leading journals4
- Frontiers in immunology1
- Investigative ophthalmology & visual science1
- Journal of lipid research1
- Nature medicine1
Leading researchers8
- Akhtar-Schaefer I1
- Barnard AR1
- Black GC1
- Chimini G1
- Dean M1
- Dotiwala F1
- Downes SM1
- Drag S1
Affiliations (unnormalised)6
- Center for Molecular Medicine Cologne1
- Chronic Diseases Research Centre1
- Clinical Neurosciences Group1
- Laboratory for Experimental Immunology of the Eye1
- Laboratory of Genomic Diversity1
- Manchester Centre for Genomic Medicine1
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A retrogressive pathological change in the retina, focal or generalized, caused by genetic defects, inflammation, trauma, vascular disease, or aging. Degeneration affecting predominantly the macula lutea of the retina is MACULAR DEGENERATION. (Newell, Ophthalmology: Principles and Concepts, 7th ed, p304)
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.