LEIDEN, The Netherlands & CAMBRIDGE, MA, USA I April 22, 2024 I ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.

“The in vitro and in vivo data that we will be presenting at ASGCT further build upon our experience with Axiomer and highlight the significant potential we see with the platform, including now preclinical proof of concept for cholestatic diseases targeting NTCP,” said Gerard Platenburg, Chief Scientific Officer and co-founder, ProQR. “We continue to be impressed by the robust editing efficiency of Axiomer editing oligonucleotides and look forward to leveraging these data as we advance AX-0810 toward the clinic.”

Wednesday, May 8, 2024 poster presentations:

P-705: “Robust and Durable RNA Editing In Vivo with Axiomer™ Editing Oligonucleotides in Non-Human Primates”

  • Building on its robust platform data, the Company will present additional non-human primate (NHP) data including with ACTB and NTCP and initial in vitro and in vivo preclinical proof of concept for its AX-0810 program targeting NTCP for cholestatic diseases, including hepatic editing in NHP with strong correlating impact on disease-relevant biomarkers.

P-726, partner poster presentation by Eli Lilly: “Complex Metabolism and Prolonged PK/PD of a GalNAc-Conjugated Editing Oligonucleotide (EON) in Mice”

Following presentation at ASGCT, the posters will be available via ProQR’s website in the Presentations and Publications section.

About Axiomer™

ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

Learn more about ProQR at www.proqr.com.