- First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025
- Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA editing platform into clinical development
LEIDEN, The Netherlands & CAMBRIDGE, MA, USA I June 26, 2025 I ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of its lead pipeline program AX-0810 targeting NTCP, a liver cell protein that transports bile acids into cells. AX-0810 is an investigational ADAR-mediated RNA editing oligonucleotide (EON) designed to selectively modulate NTCP function by reducing toxic bile acid accumulation in the liver, potentially mitigating inflammation, fibrosis, and progression toward liver failure, which are common in cholestatic diseases.
“The submission of this CTA marks a significant milestone for ProQR, representing the first to advance our Axiomer RNA editing platform and pipeline into clinical development,” said Cristina Lopez Lopez, MD, PhD Chief Medical Officer of ProQR. “This step brings us into the clinic with AX-0810 as we initiate a Phase 1 study to evaluate safety and target engagement in healthy volunteers. Cholestatic liver diseases represent an area of high unmet medical need and by targeting NTCP, AX-0810 introduces a novel therapeutic approach underpinned by human genetics and compelling preclinical data.”
The proposed Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AX-0810 in healthy adult volunteers. The study will also explore biomarkers to assess early signals of target engagement. Pending regulatory clearance, the study is expected to commence at a single site in the Netherlands with initial data anticipated in Q4 2025.
About AX-0810
AX-0810 is an investigational GalNac-delivered RNA editing oligonucleotide designed to treat cholestatic liver disease by targeting NTCP. These diseases are driven by toxic bile acid accumulation due to impaired bile flow, leading to progressive liver damage. AX-0810 offers a novel therapeutic approach by selectively modulating NTCP function, which decreases bile acid reuptake into the liver. This mechanism is supported by human genetics data showing that naturally occurring NTCP variants safety reduce bile acid reuptake, reinforcing the potential of NTCP modulation by ADAR-mediated RNA editing to improve liver health and alter the course of cholestatic disease.
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 (external link).
SOURCE: ProQR
Post Views: 1,786
- First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025
- Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA editing platform into clinical development
LEIDEN, The Netherlands & CAMBRIDGE, MA, USA I June 26, 2025 I ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of its lead pipeline program AX-0810 targeting NTCP, a liver cell protein that transports bile acids into cells. AX-0810 is an investigational ADAR-mediated RNA editing oligonucleotide (EON) designed to selectively modulate NTCP function by reducing toxic bile acid accumulation in the liver, potentially mitigating inflammation, fibrosis, and progression toward liver failure, which are common in cholestatic diseases.
“The submission of this CTA marks a significant milestone for ProQR, representing the first to advance our Axiomer RNA editing platform and pipeline into clinical development,” said Cristina Lopez Lopez, MD, PhD Chief Medical Officer of ProQR. “This step brings us into the clinic with AX-0810 as we initiate a Phase 1 study to evaluate safety and target engagement in healthy volunteers. Cholestatic liver diseases represent an area of high unmet medical need and by targeting NTCP, AX-0810 introduces a novel therapeutic approach underpinned by human genetics and compelling preclinical data.”
The proposed Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AX-0810 in healthy adult volunteers. The study will also explore biomarkers to assess early signals of target engagement. Pending regulatory clearance, the study is expected to commence at a single site in the Netherlands with initial data anticipated in Q4 2025.
About AX-0810
AX-0810 is an investigational GalNac-delivered RNA editing oligonucleotide designed to treat cholestatic liver disease by targeting NTCP. These diseases are driven by toxic bile acid accumulation due to impaired bile flow, leading to progressive liver damage. AX-0810 offers a novel therapeutic approach by selectively modulating NTCP function, which decreases bile acid reuptake into the liver. This mechanism is supported by human genetics data showing that naturally occurring NTCP variants safety reduce bile acid reuptake, reinforcing the potential of NTCP modulation by ADAR-mediated RNA editing to improve liver health and alter the course of cholestatic disease.
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 (external link).
SOURCE: ProQR
Post Views: 1,786
