… Board with Leaders in Inherited Retinal Disease and RNA Therapy LEIDEN, Netherlands & CAMBRIDGE, Mass., July 21, … to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today … of leading experts in inherited retinal disease and RNA therapies,” said Daniel A. de Boer, Chief Executive …
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
… to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today hosted a … Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber … to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare …
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
… to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today … “The visual field improvements observed indicate that RNA therapy could potentially be used to treat early stage … to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare …
Top-line data from Phase 2/3 pivotal Illuminate trial of sepofarsen for CEP290-mediated LCA10 on track for H1 2022
QR-421a pivotal Sirius and Celeste Phase 2/3 trials in Usher syndrome and retinitis pigmentosa expected to start by yea
… to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced the … 2/3 Sirius and Celeste clinical trials of investigational RNA therapy QR-421a for people with USH2A mediated retinitis … to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare …
… to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today reported its … to be diligent with our capital, as we work to advance RNA therapies for individuals with high unmet need in genetic … About Sepofarsen Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital …