Applicability of a structure-based modelling approach as a tool for in silico screening of oligonucleotides for targeted RNA editing presented at Oligonucleotide Therapeutics Society (OTS) 2018 by Julien Boudet
This presentation highlights the therapeutic possibilities of Axiomer platform that are not limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to engineer proteins or modify their function as well as creating de novo mutations.
This virtual event on March 29, 2023 featured presentations by ProQR Management on the company's proprietary Axiomer® RNA editing technology platform, detailed the initial pipeline targets, and provided guidance on the advancement of programs toward the clinic. ProQR also reported its 2022 year-end financials and the extension of its cash runway guidance.
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
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