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.
There are currently around 160 people working at ProQR, but that wasn’t always the case. When Hee Lam Chan joined in 2013, she was only employee number six. As a result, she has seen the company grow and develop.
… 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 in Phase 1/2 and building confidence in Phase 2/3 Illuminate trial;
Strategic convertible debt financing extends runway into 2023 if fully draw
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022; Brighten pediatric trial of sepofarsen to begin in 2021; additional data from Phase 1/2 InSight extension study to be reported in
Illuminate Phase 2/3 trial of sepofarsen expected to complete enrollment in Q1 2021; additional data from Phase 1/2 InSight extension study to be reported in H2 2021
Enrollment completed for QR-421a dosing cohorts and data from next Phase 1/2 interim
… 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 …
Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion and escalation planned;Updated data from the Phase 1/2 InSight extension st
… 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, …
