PURPOSE: An intravitreally injected antisense oligonucleotide, sepofarsen, was designed to modulate splicing within retinas of patients with severe vision loss due to deep intronic c.2991 + 1655A > G variant in the
OBSERVATIONS: Visual function was evaluated with best corrected standard and low-luminance visual acuities, microperimetry, dark-adapted chromatic perimetry, and full-field sensitivity testing. Retinal structure was evaluated with OCT imaging. At the fovea, all visual function measures and IS/OS intensity of the OCT showed transient improvements peaking at 3-6 months, remaining better than baseline at ∼2 years, and returning to baseline by 3-4 years after each single injection.
CONCLUSIONS AND IMPORTANCE: These results suggest that sepofarsen reinjection intervals may need to be longer than 2 years.
Cideciyan, Artur V; Jacobson, Samuel G; Ho, Allen; Swider, Malgorzata; Sumaroka, Alexander; Roman, Alejandro J; Wu, Vivian; Russell, Robert C; Viarbitskaya, Iryna; Garafalo, Alexandra V; Schwartz, Michael R; and Girach, Aniz, "Durable Vision Improvement After a Single Intravitreal Treatment With Antisense Oligonucleotide in CEP290-Lca: Replication in Two Eyes" (2023). Wills Eye Hospital Papers. Paper 192.
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