Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Strengthened leadership with appointments of Chief Financial Officer and Chief
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
… and product candidates, 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 … the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines …
In line with corporate strategy update in April, ProQR to focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas; update on initial pipeline targets expected in late 2022/early 2023
Additi
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
Senior leader with extensive global rare disease commercialization experience
Top-line data from Phase 2/3 pivotal Illuminate trial of sepofarsen for CEP290-mediated LCA10 on track for H1 2022