Shots:

• The P-II/III Illuminate study involves assessing target registration dose of sepofarsen (160 μg/80 μg loading dose/maintenance doses), a low dose of sepofarsen for masking (80 μg/40 μg) vs sham in 36 patients in a ratio (1:1:1) aged ≥ 8yrs. with genetically confirmed LCA10 due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene, at 14 study sites in 9 countries
• The study did not meet its 1EPs of BCVA @12mos. or 2EPs of FST and mobility. No benefits were observed in either treatment arm or in sham
• Sepofarsen is an investigational RNA therapy and was well tolerated. Consistent findings were observed in P-I/ll study, cataracts, CME, and retinal thinning were observed

 Ref: ProQr | Image: Market Beat