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ProQR Unveils Preclinical Proof of Concept Findings for AX-0810 Axiomer™ RNA Editing Initiative Targeting NTCP in Combatting Cholestatic Conditions

Thursday, May 09, 2024

ProQR Therapeutics NV (Nasdaq: PRQR) revealed significant preclinical findings at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Baltimore, Maryland. Their proprietary Axiomer™ RNA editing technology platform showcased promising results, notably the first preclinical proof of concept data for their AX-0810 pipeline program, aimed at treating cholestatic diseases by targeting NTCP.

Gerard Platenburg, ProQR's Chief Scientific Officer, emphasized the significance of the Axiomer ADAR RNA editing data, noting the technology's potential. He highlighted the achievement of proof of target engagement resulting in significant biomarker changes in non-human primates (NHPs). Specifically, the AX-0810 program demonstrated preclinical proof of concept by effectively reducing serum bile acid levels, a pivotal step in addressing cholestatic disorders.

AX-0810 targets the SLC10A1 RNA using Axiomer technology, offering a controlled approach to decreasing bile acid concentration in the liver. By modulating NTCP, the main transporter for bile acids, ProQR aims to mitigate the progression of cholestatic disorders.

Key findings from the preclinical data include:

  • Axiomer EONs effectively modulated NTCP protein function, leading to a significant increase in serum Total Bile Acid biomarkers.
  • Early-generation Axiomer EONs achieved up to 29% editing of NTCP in NHP livers, resulting in an 8-fold decrease in serum bile acids within 72 hours post-treatment.
  • Continued optimization enabled up to 60% editing in vitro, demonstrating translatability across models.

ProQR intends to advance its AX-0810 program to clinical trials in late 2024 or early 2025. Additionally, they anticipate reporting translational data for AX-0810 and preclinical proof of concept data for AX-1412, a program targeting B4GALT1 for cardiovascular disease, in the latter half of 2024.

Investors and analysts can learn more about these developments through an investor webcast scheduled for May 9, 2024, where ProQR's management team will discuss the presented data and engage in a Q&A session. The ASGCT poster presentations, including details of ProQR's findings, are available on the company's website.

ProQR's Axiomer platform represents a cutting-edge approach to RNA editing, holding promise for treating a wide range of diseases. By leveraging molecular machinery within human cells, Axiomer EONs offer precise, targeted modifications to RNA, potentially correcting disease-causing mutations and modulating protein expression. These advancements herald a new era in therapeutic innovation, offering hope for patients with previously untreatable conditions.



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