On behalf of all ProQRians, happy Rare Disease Day 2022. Both within ProQR and outside, we join the community in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, and supporters.
On March 29, 2023 ProQR will host a virtual R&D event to showcase its proprietary Axiomer RNA-editing technology platform. ProQR will also announce initial pipeline targets for internal development.
… initiatives that support both the growth of our people and the goals of the business. This is a hands-on, … in which people from diverse backgrounds are encouraged to develop themselves, personally and professionally. We believe that happy and energized people, working well together in an environment in which they …
… are a true believer of the value of a workforce in which people from diverse backgrounds are encouraged to develop themselves, personally and professionally. We believe that happy and energized people, working well together in an environment in which they …
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 Company expects up to four clinical data readouts in 2025 and 2026 across multi
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022
Positive data reported from the Phase 1/2 clinical trial of QR-421a for the treatment of Usher syndrome and non-syndromic retini
Preclinical proof of concept data for the AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT 27th Annual Meeting – Management to host webinar today at 8:00 am EDT to highlight dataMartin Maier, PhD nominated to Board with Annual General
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
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