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
… biomarker study for QR-504a for Fuchs endothelial corneal dystrophy to be reported in 2022 Cash runway into mid-to-late … individuals from the Phase 1/2 Stellar trial in the Helia open-label extension study, which will include multiple dose … data from this trial in 2022. ProQR has been further optimizing the Axiomer ® Editing Oligonucleotide (EON) …
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
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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
… access to the PRIority MEdicines (PRIME) program by the European Medicines Agency (EMA) for sepofarsen. The PRIME … that are for indications that currently have no treatment options. To be eligible and accepted for PRIME, a medicine … PRIME, the EMA offers additional support to medicine developers including early interaction and dialogue. The …
Continued advancement of platform and initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1Further strengthened leading intellectual property (IP) position with issuance of new patent in the United S