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Retinitis pigmentosa (RP) is an inherited retinal dystrophy leading to progressive loss of the photoreceptors and retinal pigment epithelium and resulting in blindness usually after several decades.
Features include always present findings: Rod-cone dystrophy. 4 total HPO annotations.
Age of onset: adolescence, childhood, adulthood, middle age, later in life, infancy.
The three phenotypes of autosomal recessive RPE65-related retinal degeneration, from most severe to mildest, are Leber congenital amaurosis (LCA), early-onset severe retinal dystrophy (EOSRD), and juvenile retinitis pigmentosa (RP). Systemic manifestations have not been reported in autosomal recessive RPE65-related retinal degeneration. RPE65-related LCA is a severe inherited retinal degeneration with onset of visual manifestations frequently within in the first year of life . Visual function is generally poor (but in some instances central vision is variably preserved) and is often accompanied by nystagmus and sluggish or near-absent pupillary responses.
No consensus clinical diagnostic criteria for autosomal recessive RPE65-related retinal degeneration have been published.
Autosomal recessive RPE65-related retinal degeneration should be suspected in individuals with the following clinical, electroretinographic, and imaging findings and family history.
Clinical findings
Source: GeneReviews — "Autosomal Recessive RPE65-Related Retinal Degeneration"
No approved treatments are currently available for retinitis pigmentosa. An additional 46 compounds hold orphan drug designation.
While no drugs are FDA-approved specifically for retinitis pigmentosa, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for retinitis pigmentosa. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
To monitor existing manifestations, the individual's response to supportive care, and the emergence of new manifestations, the evaluations summarized in are recommended. Note: No consensus surveillance guidelines exist; the intervals shown are based on expert opinion and clinical practice. Table 5. Autosomal Recessive RPE65-Related Retinal Degeneration: Recommended Surveillance
109 clinical trials registered, 38 recruiting. Interventions under study include other interventions, drug therapy, biologic therapy, and medical devices. Pipeline includes 8 PHASE3, 10 PHASE2, 37 PHASE1. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT06787482](https://clinicaltrials.gov/study/NCT06787482) |
Data assembled from 8 of 12 sources · Last updated Oct 3, 2026, 6:00 AM UTC
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Source: GeneReviews — "Autosomal Recessive RPE65-Related Retinal Degeneration"
See Nonsyndromic Leber Congenital Amaurosis/ Early-Onset Severe Retinal Dystrophy Overview.
Source: GeneReviews — "Autosomal Recessive RPE65-Related Retinal Degeneration"
Biomarker and diagnostic research for retinitis pigmentosa has been reported in the published literature.
Designated
Exclusivity End |
|---|
Designation Status |
|---|
allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells | allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells | BlueRock Therapeutics LP | 2025 | — | Designated |
recombinant adeno-associated virus vector expressing ChronosFP | recombinant adeno-associated virus vector expressing ChronosFP | Bionic Sight, Inc. | 2025 | — | Designated |
N6-(3-aminopropyl)-L-lysine trihydrochloride | N6-(3-aminopropyl)-L-lysine trihydrochloride | Ren Bioscience LLC | 2025 | — | Designated |
adeno-associated virus vector with a codon-optimized chicken Opsin 5 gene | adeno-associated virus vector with a codon-optimized chicken Opsin 5 gene | GenAns Biotechnology Co., Ltd | 2025 | — | Designated |
recombinant human adeno-associated virus serotype 2 containing PsCatCh2.0 gene | recombinant human adeno-associated virus serotype 2 containing PsCatCh2.0 gene | Zhongmou Therapeutics, Inc. | 2024 | — | Designated |
adeno associated virus carrying opn3 | adeno associated virus carrying opn3 | NeoVec Biotherapeutics Inc. | 2024 | — | Designated |
adeno-associated virus (AAV) gene therapy product that comprised a modified AAV2 capsid and a light sensitive transgene | adeno-associated virus (AAV) gene therapy product that comprised a modified AAV2 capsid and a light sensitive transgene | Skyline Therapeutics (US) Inc. | 2024 | — | Designated |
Human induced pluripotent stem cells (iPSC)-derived Photoreceptor Progenitor Cells | Human induced pluripotent stem cells (iPSC)-derived Photoreceptor Progenitor Cells | iRegene Therapeutics Co., Ltd | 2024 | — | Designated |
Human Retinal Pigment Epithelial Cell Injection | Human Retinal Pigment Epithelial Cell Injection | Eyecure Therapeutics Inc. | 2023 | — | Designated |
Adeno-associated viral vector serotype 2.NN encoding the human cyclic nucleotide-gated channel subunit A1 gene | Adeno-associated viral vector serotype 2.NN encoding the human cyclic nucleotide-gated channel subunit A1 gene | ViGeneron GmbH | 2023 | — | Designated |
Human Nuclear Hormone Receptor Subfamily 2 Group E Member 3 (hNR2E3) | Human Nuclear Hormone Receptor Subfamily 2 Group E Member 3 (hNR2E3) | Ocugen, Inc. | 2022 | — | Designated |
Methotrexate | Methotrexate | Aldeyra Therapeutics, Inc. | 2021 | — | Designated |
Melatonin | Melatonin | WORPHMED Srl | 2021 | — | Designated |
Small molecule inhibitor of kinase mediators of the Wnt pathway | Small molecule inhibitor of kinase mediators of the Wnt pathway | Endogena Therapeutics, Inc, | 2021 | — | Designated |
(+)-5-chloro-1-ethyl-3-(2-hydroxy-3-methoxybenzyl)-2-oxoindolin-3-yl dimethylcarbamate | (+)-5-chloro-1-ethyl-3-(2-hydroxy-3-methoxybenzyl)-2-oxoindolin-3-yl dimethylcarbamate | MitoChem Therapeutics, Inc. | 2021 | — | Designated |
Chemically induced photoreceptor-like cells | Chemically induced photoreceptor-like cells | CiRC Biosciences, Inc. | 2021 | — | Designated |
DNA plasmid encoding the human transferrin gene | DNA plasmid encoding the human transferrin gene | PulseSight Therapeutics | 2020 | — | Designated |
sulindac | sulindac | Prolindox, Inc. | 2020 | — | Designated |
Adeno-associated viral vector serotype 8 containing cDNA of the human PDE6A protein | Adeno-associated viral vector serotype 8 containing cDNA of the human PDE6A protein | Universitätsklinikum Tübingen (UKT) | 2020 | — | Designated |
2¿-O-(2-methoxyethyl) modified antisense oligonucleotide targeting exon 13 in the USH2A gene | 2¿-O-(2-methoxyethyl) modified antisense oligonucleotide targeting exon 13 in the USH2A gene | Laboratoires Théa | 2017 | — | Designated |
Adeno Associated Virus carried Multi Characteristic Opsin | Adeno Associated Virus carried Multi Characteristic Opsin | Nanoscope Therapeutics Inc. | 2017 | — | Designated |
recombinant adeno-associated virus vector expressing the retinitis pigmentosa GTPase regulator | recombinant adeno-associated virus vector expressing the retinitis pigmentosa GTPase regulator | Beacon Therapeutics | 2017 | — | Designated |
antisense oligonucleotide targeting exon 13 of the USH2A gene | antisense oligonucleotide targeting exon 13 of the USH2A gene | ProQR Therapeutics IV B.V. | 2017 | — | Designated |
antisense oligonucleotide targeting the c.7595-2144A>G mutation in intron 40 of the USH2A gene | antisense oligonucleotide targeting the c.7595-2144A>G mutation in intron 40 of the USH2A gene | ProQR Therapeutics IV B.V. | 2017 | — | Designated |
adeno-associated viral vector serotype 2.7m8 containing the chrimsonR-tdTomato gene | adeno-associated viral vector serotype 2.7m8 containing the chrimsonR-tdTomato gene | GenSight Biologics | 2017 | — | Designated |
adenovirus-associated viral vector serotype 5 containing the human pde6B gene | adenovirus-associated viral vector serotype 5 containing the human pde6B gene | eyeDNA Therapeutics | 2016 | — | Designated |
4-[(2E)-1-Oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide | 4-[(2E)-1-Oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide | Shire HGT, Inc. | 2016 | — | Withdrawn |
human recombinant mesencephalic, astrocyte derived neurotrophic factor | human recombinant mesencephalic, astrocyte derived neurotrophic factor | Amarantus BioScience Holdings, Inc. | 2014 | — | Designated |
non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein | non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein | Allergan, Inc. | 2014 | — | Withdrawn |
all-cis-docosa-4,7,10,13,16,19-hexaenoic acid | all-cis-docosa-4,7,10,13,16,19-hexaenoic acid | Celavista Mitobiogenesis, S.L. | 2014 | — | Designated |
recombinant lens epithelium derived growth factor 1-326 | recombinant lens epithelium derived growth factor 1-326 | Ocugen, Inc. | 2014 | — | Designated |
N-acetyl cysteine amide | N-acetyl cysteine amide | Nacuity Pharmaceuticals, Inc. | 2013 | — | Designated |
expanded human allogeneic neural retinal progenitor cells extracted from neural retina | expanded human allogeneic neural retinal progenitor cells extracted from neural retina | ReNeuron Ltd | 2013 | — | Designated |
recombinant human nerve growth factor | recombinant human nerve growth factor | Dompe S.p.A. | 2013 | — | Designated |
adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing DNA encoding a rhodopsin gene | adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing DNA encoding a rhodopsin gene | Spark Therapeutics Ireland Ltd. | 2012 | — | Designated |
human retinal progenitor cells | human retinal progenitor cells | jCyte, Inc. | 2012 | — | Designated |
zuretinol acetate | zuretinol acetate | Retinagenix LLC | 2010 | — | Withdrawn |
unoprostone isopropyl | unoprostone isopropyl | R-Tech Ueno, Ltd. | 2010 | — | Withdrawn |
Epitalon | Epitalon | BioDiem Ltd | 2010 | — | Withdrawn |
lentiviral vector containing the human MY07A gene | lentiviral vector containing the human MY07A gene | Sanofi US Services Inc. | 2010 | — | Designated |
recombinant human proinsulin (Including rhPI-Methionine) | recombinant human proinsulin (Including rhPI-Methionine) | ProRetina Therapeutics, S.L. | 2008 | — | Designated |
recombinant human rod-derived cone viability factor | recombinant human rod-derived cone viability factor | Fovea Pharmaceuticals | 2008 | — | Withdrawn |
Human umbilical tissue-derived cells | Human umbilical tissue-derived cells | Janssen Research & Development, LLC | 2006 | — | Withdrawn |
Urea for intravitreal injection | Urea for intravitreal injection | Vitreo Retinal Techologies, Inc | 2005 | — | Designated |
revakinagene taroretcel | revakinagene taroretcel | Neurotech USA, Inc. | 2004 | — | Designated |
Gangliosides as sodium salts | Gangliosides as sodium salts | Fidia Pharmaceutical Corp. | 1988 | — | Withdrawn |
No clinical practice guidelines for autosomal recessive RPE65-related retinal degeneration have been published. In the absence of published guidelines, the following recommendations are based on the authors' personal experience managing individuals with this disorder.
To establish the extent of disease and needs of individuals diagnosed with autosomal recessive RPE65-related retinal degeneration, the evaluations included (if not performed as part of the evaluation that led to the diagnosis) are recommended, and their purpose summarized. Note that some evaluations may be indicated only in individuals considering subretinal gene supplementation therapy .
Table 3.
Autosomal Recessive RPE65-Related Retinal Degeneration: Recommended Evaluations Following Initial Diagnosis
Evaluation | Purpose
| BCVA | To determine visual acuity provide baseline for comparison of future assessments
| To prescribe corrective lenses
| To document anterior segment findings such as cataract
| To document fundus findings
| To map out entire visual field provide baseline
| To determine retinal sensitivity in any given location in visual field to provide baseline
OCT | To assess anatomic structure of retina, which may identify persons more likely to benefit from RPE65 gene replacement therapy
| To document fundus findings prov...
Source: GeneReviews — "Autosomal Recessive RPE65-Related Retinal Degeneration"
Children should be discouraged whenever possible from repeatedly poking and pressing on their eyes, which may cause damage to the cornea and/or retina.
Source: GeneReviews — "Autosomal Recessive RPE65-Related Retinal Degeneration"
Clinical investigations of variations of the FDA-approved gene replacement therapy treatment for autosomal recessive RPE65-related retinal degeneration (i.e., subretinal injection of an AAV2 vector expressing full-length RPE65-encoded protein, retinoid isomerohydrolase) have been completed. A Phase I/IIb clinical trial (NCT02781480) of an AAV2/5 vector with codon-optimized RPE65 has been completed, but no peer-reviewed outcomes have yet been published. Search ClinicalTrials.gov in the US and EU Clinical Trials Register in Europe for access to information on clinical studies for a wide range of diseases and conditions.
Source: GeneReviews — "Autosomal Recessive RPE65-Related Retinal Degeneration"
109 trials found
Evaluation
Purpose |
|---|
Frequency |
|---|
BCVA | To assess visual acuity | Yearly Refractive error |
Kinetic visual perimetry | To assess changes in entire visual field | Yearly if possible |
Static visual perimetry | To assess changes in retinal sensitivity in any given location in visual field | Yearly OCT |
Fundus photography | To document fundus changes, which may identify disease progression | Yearly if possible FAF |
Full-field ERG | To assess residual activity of rods (dark-adapted [scotopic] ERG) cones (light-adapted [photopic] ERG) | Every 3-5 yrs FST test |
Developmental/educational assessment | To assess developmental/educational needs | Yearly or as needed Neurobehavioral/psychiatric assessment |
Source: GeneReviews — "Autosomal Recessive RPE65-Related Retinal Degeneration"
Phenotype severity distribution: 1 always present feature.
Estimated prevalence: 1-5 in 10,000 (Uncommon).
Evaluating a New Peptide Therapy for Retinal Diseases: AMD, Diabetic Retinopathy, and Dystrophies |
PHASE1 |
Ace Cells Lab Limited |
RECRUITING |
[NCT06936787](https://clinicaltrials.gov/study/NCT06936787) | An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa | PHASE1 | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | RECRUITING |
[NCT05874310](https://clinicaltrials.gov/study/NCT05874310) | Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa | EARLY_PHASE1 | Frontera Therapeutics | RECRUITING |
[NCT05355415](https://clinicaltrials.gov/study/NCT05355415) | Adaptive Optics Imaging of Outer Retinal Diseases | — | Food and Drug Administration (FDA) | RECRUITING |
[NCT06092346](https://clinicaltrials.gov/study/NCT06092346) | A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders | — | National Human Genome Research Institute (NHGRI) | RECRUITING |
500 publications have been identified in PubMed for retinitis pigmentosa. Research spans Basic Science / Preclinical (31%), Case Report / Case Series (18%), and Epidemiology / Natural History (14%).
Research Type | Count | % of Total |
|---|---|---|
Laboratory research | 157 | 31% |
Patient case studies | 90 | 18% |
Disease patterns and progression | 70 | 14% |
Research summaries | 68 | 14% |
New treatment approaches | 54 | 11% |
Testing and diagnosis research | 31 | 6% |
Clinical study results | 21 | 4% |
Other research | 9 | 2% |
Hiraoka M (2026). [PMID: 42212888](https://pubmed.ncbi.nlm.nih.gov/42212888/). *Transl Vis Sci Technol*. [Diagnostic / Biomarker]
Gürsoy N (2026). [PMID: 41827546](https://pubmed.ncbi.nlm.nih.gov/41827546/). *Healthcare (Basel)*. [Epidemiology / Natural History]
Rachwał A (2026). [PMID: 41781416](https://pubmed.ncbi.nlm.nih.gov/41781416/). *Sci Rep*. [Other]
Yang Y (2026). [PMID: 41897304](https://pubmed.ncbi.nlm.nih.gov/41897304/). *Biomolecules*. [Review / Meta-Analysis]
Zhang R (2026). [PMID: 42545893](https://pubmed.ncbi.nlm.nih.gov/42545893/). *CNS Neurosci Ther*. [Basic Science / Preclinical]
Hameed M (2026). [PMID: 42363358](https://pubmed.ncbi.nlm.nih.gov/42363358/). *J Pak Med Assoc*. [Epidemiology / Natural History]
Zimmann F (2026). [PMID: 41731009](https://pubmed.ncbi.nlm.nih.gov/41731009/). *Sci Rep*. [Basic Science / Preclinical]
Seyedtaghia MR (2026). [PMID: 40252141](https://pubmed.ncbi.nlm.nih.gov/40252141/). *Biochem Genet*. [Basic Science / Preclinical]
Manian KV (2026). [PMID: 42525778](https://pubmed.ncbi.nlm.nih.gov/42525778/). *Sci Adv*. [Basic Science / Preclinical]
Goda T (2026). [PMID: 41956113](https://pubmed.ncbi.nlm.nih.gov/41956113/). *J Pediatr Endocrinol Metab*. [Case Report / Case Series]
AI-curated news mentioning retinitis pigmentosa
Updated Sep 22, 2026
A novel variant in the PRPF31 gene has been identified as associated with autosomal dominant retinitis pigmentosa in Japanese families. This discovery adds to the understanding of genetic factors contributing to this form of retinal degeneration.
A multicenter case series identifies biallelic RDH11 variants as a cause of syndromic retinitis pigmentosa, early-onset cataracts, and neurodevelopmental delay. This discovery enhances understanding of the genetic basis for these conditions.
A new phenotype of sector retinitis pigmentosa associated with cone dystrophy has been defined, enhancing understanding of RPGR-related retinal diseases. This discovery could inform future research and therapeutic strategies.
Abu Dhabi launches the UAE's first clinical trial for a novel gene therapy targeting MerTK-related retinitis pigmentosa, a rare inherited eye disease causing vision loss. Developed by Opus Genetics, this initiative aims to accelerate the development of treatments for inherited retinal diseases affecting about 5% of the population.
Access to gene therapies remains limited due to high costs and geographic barriers, leaving many patients untreated. Dustin Vidrine, who has retinitis pigmentosa, is seeking clinical trials for therapies that could address his specific genetic mutation.