ARVO 2021 presentation by Stephen R. Russell from Iowa University on the Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in CEP290 mediated Leber Congenital Amaurosis type 10.
ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
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.
ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
