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NCT03872479

Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants With Leber Congenital Amaurosis Type 10 (LCA10), With Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene ("LCA10-IVS26")

Study Summary

The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26").

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Interventions

EDIT-101DRUG
Participants will receive a single dose of EDIT-101 administered via subretinal injection in the study eye. Up to 5 cohorts across 3 doses will be enrolled in this study.

Study Locations

FacilityCityStateCountry
Bascom Palmer Eye InstituteMiamiFloridaUnited States
Massachusetts Eye and Ear InfirmaryBostonMassachusettsUnited States
W.K. Kellogg Eye Center - University of MichiganAnn ArborMichiganUnited States
Casey Eye Institute - OSHUPortlandOregonUnited States
University of PennsylvaniaPhiladelphiaPennsylvaniaUnited States

Official Trial Information

View on ClinicalTrials.gov

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