Phuturdex › Diseases › Hereditary eye disease
Drugs in development · updated 30 October 2026
Hereditary eye disease: drugs in development
As of 30 October 2026 there are 5 active clinical trials (recruiting or ongoing, phases I–III) on 4 drugs for hereditary eye disease. 4 are experimental, that is not yet approved in the United States.
Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| Sepofarsen | II–III | 2 | NCT03913143 |
| QR-1123 | I–II | 1 | NCT04123626 |
| VP-001 | I–II | 1 | NCT06852963 |
| ETHY-001 | I | 1 | NCT07885605 |
Sorted by most advanced phase and number of active trials.
All 4 drugs, with the details
In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.
Open in Phuturdex →Specific forms
- Graves' ophthalmopathy 19 drugs
- Retinitis pigmentosa 25 drugs
- Stargardt disease 14 drugs
- Retinal degeneration 15 drugs
- Eye disease 19 drugs
- Retinal disease 18 drugs
- Retinoblastoma 14 drugs
- Macular degeneration 40 drugs
- Leber congenital amaurosis 10 drugs
- Retinal Dystrophies 8 drugs
- Usher Syndromes 5 drugs
- Fuchs' endothelial dystrophy 4 drugs
Broader categories
- Genetic disease 33 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Nervous System Diseases 39 drugs
- Metabolic disease 30 drugs
- Nutritional and metabolic diseases 5 drugs
- Congenital Abnormalities 14 drugs
Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D015785 “Eye Diseases, Hereditary”.