ProQR Expands Horizons with AX-0810 for Cholestatic Diseases
ProQR's Breakthrough with AX-0810
ProQR Therapeutics N.V. (Nasdaq: PRQR), a leader in transformative RNA therapies, has reached a pivotal milestone with the authorization of its Clinical Trial Application (CTA) for AX-0810. This momentous achievement follows a thorough review process by the European Medicines Agency (EMA). With this approval, ProQR is set to initiate its Phase 1 study of AX-0810, focusing on healthy volunteers. AX-0810 is particularly exciting as it targets NTCP, aiming to address cholestatic diseases such as primary sclerosing cholangitis and biliary atresia.
Phase 1 Study Details
The North Dutch-based study will assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of AX-0810, with plans to gather critical biomarker data to establish evidence of target engagement. This is a crucial step in understanding how AX-0810 could potentially transform the landscape of treatment for cholestatic conditions that significantly impair liver functionality.
Virtual Investor and Analyst Event
To commemorate this significant milestone, ProQR will host a virtual Investor and Analyst Event titled “Entering the Clinic with AX-0810: Establishing Safety, PK, and the Biomarker Roadmap for Proof of Target Engagement.” This online gathering is scheduled for November 3, 2025, at 10 am ET, featuring key presentations from ProQR Management and Professor Henkjan J. Verkade, MD, PhD, a recognized expert in pediatric gastroenterology.
Event Registration and Format
For those interested in participating, the event will provide an interactive platform featuring live presentations and a Q&A session where analysts can engage with ProQR's leadership. This opportunity promises invaluable insights into the ongoing development and future of AX-0810. The registration information can be accessed on ProQR's official website.
Insights on AX-0810
AX-0810 represents a groundbreaking approach as a first-in-class investigational RNA editing oligonucleotide (EON). It utilizes the body’s endogenous ADAR enzymes to regulate NTCP function selectively. This innovative mechanism is designed to mitigate toxic bile acid buildup in the liver, potentially leading to improved clinical outcomes in patients suffering from cholestatic diseases. By strategically targeting mechanisms driving disease progression, AX-0810 has the potential to modify the disease itself, making it a focus of keen interest in the medical community.
Meet Professor Henkjan J. Verkade
Professor Verkade will provide valuable insights during the upcoming event. With a distinguished career in pediatric gastro/hepatology and over 300 peer-reviewed publications to his credit, his research focuses on fundamental aspects of liver diseases and their treatments. His expertise will be vital as ProQR navigates the early stages of clinical development for AX-0810, further establishing the therapeutic roadmap for engaging biomarkers.
Axiomer™ Technology and ProQR's Future
At its core, ProQR is advancing a next-generation RNA technology platform known as Axiomer™. This pioneering approach to RNA base editing provides potential treatments for various diseases that currently lack effective therapies. The unique ability of Axiomer™ to enact precise edits at the RNA level positions ProQR to make significant strides in addressing both rare and prevalent diseases. This foundation supports ProQR's expanding pipeline, firmly focused on improving lives through impactful medical innovations.
About ProQR
ProQR Therapeutics is on a mission to change lives through the development of innovative RNA treatments. As a frontrunner in RNA therapy, ProQR harnesses the body's natural resources, enabling novel therapeutic approaches aimed at correcting genetic mutations or altering protein expressions associated with various diseases. With a strong emphasis on patient-centric solutions, ProQR is dedicated to pushing the boundaries of science to meet unmet medical needs.
Frequently Asked Questions
What is AX-0810 and its purpose?
AX-0810 is an investigational RNA editing oligonucleotide targeting NTCP, developed for treating cholestatic diseases.
When will the Phase 1 study of AX-0810 commence?
The Phase 1 study of AX-0810 is authorized to begin dosing immediately following the CTA approval.
How can I register for the virtual event?
Registration details for the virtual event can be found on ProQR's official website.
Who is hosting the virtual event?
The event will be hosted by ProQR's management team along with medical expert Professor Henkjan J. Verkade.
What distinguishes Axiomer™ technology?
Axiomer™ technology allows for precise RNA editing, potentially leading to new treatments for various genetic disorders.
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