ProQR will present new preclinical data for Axiomer, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the ASGCT Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.
The presentation reviews EONs structure activity, the potential of the Axiomer platform as well as its applicability for the treatment of liver originated disorders. Presented at TIDES USA on May 9, 2023 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.
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.
… Ho 1 , Artur V Cideciyan 2 , Samuel G Jacobson 2 , Stephen R Russell 3 , Arlene V Drack 3 , Bart P Leroy 4, 5, 6 , Michael R Schwartz 7 , Wilhelmina den Hollander 7 , Hsin Yi Huang van … Hospital of Philadelphia, Philadelphia, Pennsylvania; 7 ProQR Therapeutics, Leiden, the Netherlands. Ho A, Cideciyan …
… visual acuity, and full-field stimulus testing. Stephen R. Russell 1 , Arlene V. Drack 1 , Artur V. Cideciyan 2 , … Zaeytijd 3 , Aniz Girach 6 , Wil den Hollander 6 , Michael R. Schwartz 6 , David M. Rodman 6 1 Department of … Thomas Jefferson University, Philadelphia, PA, USA; 6 ProQR Therapeutics, Leiden, the Netherlands. Russell SR, …
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.
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.
