Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseasesInitiating Phase 1 study of AX-0810 in healthy volunteers, with initial safety, tolerability, and PK data from Cohort 1 expected by year-end, and target engagemen
AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024$4.5 million in milestones
Additional analyses ongoing from Phase 2/3 Illuminate trial of sepofarsen in LCA10 with updates on any potential next steps with this program and overall strategy anticipated in Q2 2022
Phase 2/3 Sirius and Celeste clinical trials of ultevur
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1
Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areas
Stre
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
Top-line data from pivotal Phase 2/3 Illuminate trial of sepofarsen for CEP290-mediated LCA10 anticipated late Q1/early Q2 2022
Five-target collaboration with Lilly highlights significant potential of ProQR’s Axiomer® RNA base-editing pla
AX-0810 targeting NTCP for cholestatic diseases to be first ProQR Axiomer™ RNA editing program to enter the clinic Company to host virtual Investor and Analyst Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting