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.
AAO 2020 poster by Stephen Russel et al. Recognized with best poster award. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
