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Gene Therapy for LCA10: The Brilliance Phase 1/2 Clinical Trial Of AGN-151587 (EDIT-101) Update

The Brilliance Phase 1/2 Clinical Trial Of AGN-151587 (EDIT-101) For The Treatment Of LCA10 Description This is an open-label, single ascending dose study of AGN-151587 (EDIT-101) in adult and pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Approximately 18 participants will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of AGN-151587 in this study. AGN-151587 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26. Single-dose of AGN-151587 administered by subretinal injection surgery Participants will receive a single dose of AGN-151587 administered via subretinal injection in the study eye. Up to 5 cohorts across 3 doses will be enrolled in this study. Primary Outcome Measure Frequency of Adverse Events related to AGN-151587  Number of participants experiencing procedural related adverse events  Incidence of dose limiting toxicities Seconda...