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Disease
Retinitis Pigmentosa
Late-stage therapeutic developmentEmerging researchRising momentum
3
Publications
20
Clinical trials
2023
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Research2023-08-15Nature communications
Inherited retinal diseases: Therapeutics, clinical trials and end points-A review.
Research2021-03-20Clinical & experimental ophthalmology
Research2020-02-24Nature medicine
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
- 5 clinical trials expected to report results, the earliest in Q3 2027.
Upcoming Milestones
- Q3 2027A Phase II, Randomised, Controlled, Double Masked, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
- Q3 2028A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
- Q2 2029NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa
- Q3 2029Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
- Q4 2029A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa
Major developments
Upcoming trial readoutHigh impact
Results expected Q3 20292026-07-06
Upcoming trial readoutHigh impact
Results expected Q2 20292026-06-15
Clinical MilestonesViewHide
Results posted
Results expected
- 2026-07-09A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A GeneResults expected Q3 2028
- 2026-07-06Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GeneResults expected Q3 2029
- 2026-06-25A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis PigmentosaResults expected Q4 2029
- 2026-06-18A Phase II, Randomised, Controlled, Double Masked, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis PigmentosaResults expected Q3 2027
- 2026-06-15NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis PigmentosaResults expected Q2 2029
Activity timeline7
- 2026-07-09ClinicalA Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A GeneResults expected Q3 2028
- 2026-07-06ClinicalPhase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GeneResults expected Q3 2029
- 2026-06-30ClinicalA Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis PigmentosaPrimary completion
- 2026-06-25ClinicalA Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis PigmentosaResults expected Q4 2029
- 2026-06-18ClinicalA Phase II, Randomised, Controlled, Double Masked, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis PigmentosaResults expected Q3 2027
- 2026-06-15ClinicalNAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis PigmentosaResults expected Q2 2029
- 2026-01-12ClinicalPhase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)Results posted
Clinical trials
The current development programme across all trial phases.
Clinical programme
20
6
10
6
Late-stage studies
Recently completed
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Publications over time
20202023
Most influential
Recent publications
Major themes4
- Retinitis Pigmentosa2
- Genetic Therapy1
- Leber Congenital Amaurosis1
- Retinal Dystrophies1
Leading journals3
- Clinical & experimental ophthalmology1
- Nature communications1
- Nature medicine1
Leading researchers8
- Aylward JW1
- Barnard AR1
- Bi C1
- Black GCM1
- Cehajic-Kapetanovic J1
- Chen X1
- Davies A1
- Davis JL1
Affiliations (unnormalised)6
- Bascom Palmer Eye Institute1
- Casey Eye Institute1
- Clinical Neurosciences Research Group1
- College of Life Science1
- Keio University School of Medicine1
- Laboratory of Visual Physiology1
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
Hereditary, progressive degeneration of the retina due to death of ROD PHOTORECEPTORS initially and subsequent death of CONE PHOTORECEPTORS. It is characterized by deposition of pigment in the retina.
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.