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.
Axiomer platform presentation highlighting the potential of Editing Oligonucleotides (EONs) to address liver originated disorders. Presented at the RNA Editing Gordon Research Conference on March 23, 2023 by Gerard Platenburg - ProQR Therapeutics.
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.
Axiomer® platform applicability and potential in therapeutically relevant models for targeted RNA editing presented at TIDES EU 2018 by Antti Aalto - ProQR Therapeutics
Progress on Development of RNA Base Editing Technologies for Precision Medicines, including Axiomer® platform presented at TIDES EU by Gerard Platenburg - ProQR Therapeutics
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
Modification of a guide RNA can mimic the effect of hyperactive mutants and advance the approach of recruiting endogenous ADARs for site-directed RNA editing reported in Journal of the American Chemical Society (JACS) by Doherty EE et al.
Development of RNA Base Editing Technologies for Precision Medicines with Axiomer® and Trident® presented at TIDES USA 2022 by Gerard Platenburg - ProQR Therapeutics.
ARVO 2021 presentation by Stephen R. Russell from Iowa University on the Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in CEP290 mediated Leber Congenital Amaurosis type 10.
ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.