ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA
ProQR Therapeutics today announced positive results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.
Top-line results expected H1 2022
Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan.
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra
