Retinal Dystrophies
Recent clinical, regulatory, research and industry developments relating to this disease.
Inherited retinal diseases: Therapeutics, clinical trials and end points-A review.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 2 clinical trials expected to report results, the earliest in Q1 2028.
- 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
Clinical MilestonesViewHide
- 2026-09-16The Effects of Disulfiram on Visual Acuity in Patients With Retinal DegenerationResults expected Q2 2029
- 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
- 2026-09-16ClinicalThe Effects of Disulfiram on Visual Acuity in Patients With Retinal DegenerationResults expected Q2 2029
- 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
- 2025-09-24ClinicalA 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.
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
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.
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.
- 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.