Home » Clinical Trials » Inherited retinal dystrophy » NCT06467344
NCT06467344

ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy

Study Summary

This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.

Want to learn more about this trial?

Request More Info

Interventions

ACDN-01DRUG
ACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection.

Study Locations

FacilityCityStateCountry
University of San FranciscoSan FranciscoCaliforniaUnited States
Vitreo Retinal AssociatesGainesvilleFloridaUnited States
Bascom Palmer Eye InstituteMiamiFloridaUnited States
Wilmer Eye Institute at John HopkinsBaltimoreMarylandUnited States
Massachusetts Eye and EarBostonMassachusettsUnited States
University of Michigan Kellogg Eye CenterAnn ArborMichiganUnited States
Cincinnati Eye InstituteCincinnatiOhioUnited States
Casey Eye Institute OHSUPortlandOregonUnited States
Retina Foundation of the SouthwestDallasTexasUnited States
Retina Consultants of TexasHoustonTexasUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026