“Top-notch science that leads to a treatment” Story 2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year. March 31, 2017
Phase 2/3 Illuminate trial results of sepofarsen in CEP290-mediated LCA10 News update Top-line results showed the Illuminate study did not meet its primary endpoint or secondary endpoints. We will continue to analyze the data. February 11, 2022