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Disease

Retinitis Pigmentosa

Late-stage therapeutic developmentEmerging researchRising momentum
MeSHRetinitis Pigmentosa
3
Publications
20
Clinical trials
12
Associated genes
5
Related proteins
2023
Latest publication
Current focus
Aryl hydrocarbon biologyTherapeutic developmentGenetics & risk factors
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

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.
  • 4 industry developments reported.
Upcoming Milestones
Clinical Milestones7View
Results posted 1
Results expected 5
Recently completed 1
Industry & Market4View
Activity timeline11

Clinical trials

12 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
20
All trials
6
Active
10
Late-stage
6
Completed
Late-stage studies

A 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 Pigmentosa

Phase 2/3 · Active, not recruiting · Beacon Therapeutics

NAC 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

Phase 3 · Active, not recruiting · Johns Hopkins University

Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Phase 3 · Active, not recruiting · Janssen Research & Development, LLC

Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Phase 3 · Completed · Janssen Research & Development, LLC

Bioretina, Ankara University Technopolis

Phase 3 · Completed · Ankara Universitesi Teknokent

Cord Blood Platelet-rich Plasma (CB-PRP) in Retinitis Pigmentosa and Dry Age-related Macular Degeneration

Phase 3 · Completed · Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Recently completed

Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Phase 3 · Completed · Janssen Research & Development, LLC

Bioretina, Ankara University Technopolis

Phase 3 · Completed · Ankara Universitesi Teknokent

Cord Blood Platelet-rich Plasma (CB-PRP) in Retinitis Pigmentosa and Dry Age-related Macular Degeneration

Phase 3 · Completed · Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)

Phase 1/2 · Completed · AbbVie

Management of Retinitis Pigmentosa by Wharton's Jelly Derived Mesenchymal Stem Cells: Preliminary Clinical Results

Phase 3 · Completed · Ankara Universitesi Teknokent

Prospective Non-randomised Exploratory Study to Assess the Safety and Efficacy of Aflibercept (Eylea) in Cystoid Macular Oedema (CMO) Associated With Retinitis Pigmentosa (RP)

Phase 2 · Completed · Moorfields Eye Hospital NHS Foundation Trust

Research activity

3 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20202023
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

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

RHO0.85RPGR0.84USH2A0.84RPE650.83RP10.82EYS0.82PDE6B0.82TULP10.82PRPH20.82SNRNP2000.82RP20.82PRPF80.81

Disease biology

5 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Tyrosine-protein kinase Mervia MERTK0.80Inosine-5'-monophosphate dehydrogenase 1via IMPDH10.73Carbonic anhydrase 4via CA40.69Aryl hydrocarbon receptorvia AHR0.47CX3C chemokine receptor 1via CX3CR10.12

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
MeSHRetinitis Pigmentosa
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.