ArticleNature medicine2026
Intravitreal photoswitch therapy in advanced retinitis pigmentosa: a phase 1 open-label trial.
Article in Nature medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05282953 (A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia), which is not on this map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
Who cites it
4 citing papers in PubMed.
- Restoration of Saccadic Eye Movements and Visually Guided Behavior in Ambient White Light with Photoswitchable Small Molecules.Journal of the American Chemical Society · 2026Article
- Photopharmacology: A new paradigm for vision restoration.Clinical and translational medicine · 2026Article
- Toward Chromoselective Transformations in Biological Systems: Perspectives and Challenges.Angewandte Chemie (International ed. in English) · 2026Review
- Image-guided activation of drugs with electromagnetic radiation.Nature chemical biology · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A small azobenzene photoswitch molecule (KIO-301), designed to confer light responsiveness to retinal ganglion cells, was evaluated for safety and feasibility in a first-in-human, phase 1, gene-agnostic, open-label, dose-escalation clinical trial in individuals with advanced retinitis pigmentosa (RP). KIO-301 was administered by intravitreal injection to 12 eyes of 6 participants. The primary outcome was ocular and systemic safety over 30 days. Secondary and exploratory assessments included functional vision testing, visual acuity, kinetic visual field, functional magnetic resonance imaging and participant-reported outcomes. The primary safety outcome was met, with no serious adverse events or dose-limiting toxicities observed at any point. No drug-related intraocular inflammation occurred, and all ocular adverse events were mild and procedure-related. Exploratory assessments identified variation in light perception and functional vision measures in some participants. Light-evoked blood-oxygen-level-dependent signal changes in visual cortical regions were observed following dosing and showed a temporal pattern compatible with pharmacodynamic activity. Participant-reported quality-of-life scores varied over time. In this small, nonrandomized phase 1 study in individuals with late-stage RP, intravitreal KIO-301 demonstrated an acceptable safety and tolerability profile, supporting the feasibility of photoswitch therapy in advanced RP, and motivating further evaluation in larger trials. ClinicalTrials.gov identifier: NCT05282953.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.