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.
… of protocols for every cell line.” How did you test sepofarsen in the organoids? “We have started testing sepofarsen, our investigational RNA therapy for Leber …
… 2023 Additional pivotal trial recommended by EMA for sepofarsen prior to a regulatory submission Company will seek … – including Illuminate , Insight , and Brighten for sepofarsen, along with Sirius and Helia for ultevursen – will …
… Positive preliminary data from InSight extension study of sepofarsen for LCA10 reported – consistent with benefit seen … data from our InSight extension study supporting sepofarsen’s potential to treat patients with LCA10 and …
… Financial Results Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data … for adRP, data from the Phase 1/2 extension study of sepofarsen, and to start dosing patients with QR-504a for …
… and Financial Results Illuminate Phase 2/3 trial of sepofarsen expected to complete enrollment in Q1 2021; … retinal diseases.” Business Operations and Program Updates Sepofarsen, lead clinical candidate for Leber congenital …
… strategy, which includes starting the Phase 2/3 trial for sepofarsen in LCA10 patients, initiating the first-in-human … (PRIME) program by the European Medicines Agency (EMA) for sepofarsen. The PRIME program is particularly focused on …
… retinal diseases (IRDs). The positive interim data for sepofarsen in LCA10 has resulted in our ability to move … blindness.” Corporate Highlights and Business Update Sepofarsen (formerly QR-110) for LCA10 In January 2019, …
