ProQR Announces Preclinical Proof of Concept Data for AX-0810 Axiomer RNA Editing Program Targeting NTCP for Cholestatic Diseases

• ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer RNA Editing Oligonucleotides
• Preclinical proof of concept for the Company’s AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT Annual Meeting
• Management webinar May 9, 2024 at 8:00 am EDT
  
  

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced new preclinical data for its proprietary Axiomer RNA editing technology platform, including the first preclinical proof of concept data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP. The data are being presented at a poster session today at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.

“Our Axiomer ADAR RNA editing data at ASGCT show the exciting potential of our technology and for the first time in the field we report proof of target engagement leading to meaningful changes in biomarkers in NHPs,” said Gerard Platenburg, Chief Scientific Officer and co-founder, ProQR. “In our AX-0810 program targeting NTCP for cholestatic diseases, we have demonstrated preclinical proof of concept showing that Axiomer RNA editing oligonucleotides can have a meaningful effect on levels of serum biomarker bile acid. We believe this is a significant derisking step as we approach the clinic as our NHP studies closely resemble the design of the first in human trial that will focus on target engagement and biomarkers. We look forward to sharing translational data for our AX-0810 program and to announcing our clinical program candidate later this year, as we progress to enter the clinic in late 2024/early 2025.”

Preclinical proof of concept for AX-0810

AX-0810 is an editing oligonucleotide (EON) development program targeting the SLC10A1 RNA using the Axiomer technology, providing a transient and controlled approach that aims to reduce bile acids concentration in the liver. By specifically affecting the main transporter for bile acids reuptake from the portal vein circulation to the liver, called NTCP (Na-taurocholate transporting polypeptide, SLC10A1 gene), the AX-0810 program represents a promising avenue to ameliorate the progression of cholestatic disorders.