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Disease
Retinal Dystrophies
Active therapeutic pipelineEmerging research
1
Publications
8
Clinical trials
2021
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Inherited retinal diseases: Therapeutics, clinical trials and end points-A review.
Research2021-03-20Clinical & experimental ophthalmology
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Low
Key developments
- 2 clinical trials expected to report results, the earliest in Q1 2028.
Upcoming Milestones
- 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
- Q2 2029The Effects of Disulfiram on Visual Acuity in Patients With Retinal Degeneration
Major developments
Upcoming trial readoutImportant
Results expected Q1 20282026-05-29
Upcoming trial readoutWorth watching
Results expected Q2 20292025-09-29
Clinical MilestonesViewHide
Results expected
- 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
- 2025-09-29The Effects of Disulfiram on Visual Acuity in Patients With Retinal DegenerationResults expected Q2 2029
Activity timeline3
- 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
- 2025-09-29ClinicalThe Effects of Disulfiram on Visual Acuity in Patients With Retinal DegenerationResults expected Q2 2029
- 2025-09-24ClinicalA Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants With Confirmed PRPF31 Mutation-Associated Retinal DystrophyCompleted
Clinical trials
The current development programme across all trial phases.
Clinical programme
8
3
0
4
Recently completed
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes3
- Leber Congenital Amaurosis1
- Retinal Dystrophies1
- Retinitis Pigmentosa1
Leading journals1
- Clinical & experimental ophthalmology1
Leading researchers3
- Fujinami K1
- Georgiou M1
- Michaelides M1
Affiliations (unnormalised)4
- Keio University School of Medicine1
- Laboratory of Visual Physiology1
- Moorfields Eye Hospital NHS Foundation Trust1
- UCL Institute of Ophthalmology1
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A group of disorders involving predominantly the posterior portion of the ocular fundus, due to degeneration in the sensory layer of the RETINA; RETINAL PIGMENT EPITHELIUM; BRUCH MEMBRANE; CHOROID; or a combination of these tissues.
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.