Search

… on key steps required to advance AX-0810 and AX-1412 to clinical trials with translational data updates ahead of … the winding down of ophthalmology programs, including the clinical trials. General and administrative costs for the …

… out of twelve patients have been enrolled in a Phase 1/2 clinical trial of QR-110 in adults and children with LCA 10. Interim …

… to be safe and well-tolerated across all doses in this trial with no serious adverse events related to treatment. A … top-line results from a Phase 1b safety and tolerability clinical trial (Study PQ-010-001; NCT02532764 ) of QR-010, a …

… Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting … updates on this program, including preclinical data, clinical trial design, and biomarkers that will be assessed, along …

… and efficacy of sepofarsen in a Ph1b/2 INSIGHT extension trial in CEP290-associated Leber congenital amaurosis (LCA10) … missing the primary endpoint in the Illuminate Phase 2/3 clinical trial, sepofarsen showed an encouraging efficacy …

… timing, progress and results of our preclinical studies, clinical trials and other development activities, including … our business operations, as well as the timing of our clinical development. Forward-looking statements are based on … the cost, timing and results of preclinical studies and clinical trials and other development activities by us and …

… studies closely resemble the design of the first in human trial that will focus on target engagement and biomarkers. We … data for our AX-0810 program and to announcing our clinical program candidate later this year, as we progress to … reported in H2 2024, with further detail on design for the clinical trial. AX-1412 preclinical proof of concept data and …

… Additional analyses ongoing from Phase 2/3 Illuminate trial of sepofarsen in LCA10 with updates on any potential … anticipated in Q2 2022 Phase 2/3 Sirius and Celeste clinical trials of ultevursen (QR-421a) in Usher syndrome and …

… and Financial Results Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with … Q2 2021 Further pipeline progress anticipated with first clinical data in 2021 from QR-1123 program for autosomal … plan to advance the QR-504a program into a first-in-human clinical trial in late-stage disease patients in 2021. …

… November 2017 that will fund operations through potential clinical data readouts in three different programs. Positive results from the Phase 1b safety and tolerability clinical trial of QR-010 in cystic fibrosis (CF) patients with the …