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.
AAO 2020 poster by Allen Ho et al. Recognized with best poster award. Data show that FST seems a more sensitive method in patients with severely impaired vision.
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
