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An Open-Label, Dose Escalation and Double-Masked, Randomized,
Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in
Pediatric Subjects <8 Years of Age with Leber Congenital Amaurosis Type
10 (LCA10) due to the c.2991 +1655A>G (p.Cys998X) mutation

Primary: To evaluate safety and tolerability of sepofarsen in subjects with LCA10 &lt;8 years of age.Secondary: To evaluate the effect of sepofarsen on structural and functional ophthalmic outcome measures.