ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
This presentation reviews Axiomer editing oligonucleotides (EONs) structure activity, the potential of Axiomer platform as well as its applicability for the treatment of liver originated disorders.
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.
