Single Ascending Dose Study in Participants With LCA10
Status:
Recruiting
Trial end date:
2024-03-22
Target enrollment:
Participant gender:
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").