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").
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Frequency of Adverse Events related to EDIT-101
Timeframe: 1 year
Number of participants experiencing procedural related adverse events
Timeframe: 1 year
Incidence of dose limiting toxicities
Timeframe: 1 year