ProQR Announces Multiple Upcoming Scientific Conference Presentations Highlighting the Breadth of its Axiomer™ RNA Editing Platform

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 11, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced presentations at several upcoming scientific conferences, highlighting continued progress in the application of its Axiomer RNA editing platform.

“These presentations collectively expand the body of evidence supporting the potential of our Axiomer RNA editing platform to enable precise RNA editing across multiple targets,” said Gerard Platenburg, Co-founder and Chief Scientific Officer of ProQR. “Across liver and CNS applications, we continue to demonstrate the broad applicability of our technology, while also progressing our lead program, AX-0810, toward initial clinical data in healthy volunteers expected later this quarter.”

Presentations will include:

TIDES USA 2026 (Boston, MA, May 11–14)

Title: Advancing ADAR RNA Editing Technology for Broad Applicability in Precision Medicines

Oral presentation summary: Overview of Axiomer pipeline programs and upcoming milestones, highlighting AX-0422 targeting IDUA in Hurler syndrome, with preclinical data demonstrating potential for long-term glycosaminoglycans (GAGs) reduction in multiple organs and improved functional outcome compared to enzyme replacement therapy in vivo.
Presenter: Gerard Platenburg, Co-founder and CSO
May 13, 9:00–9:30 AM ET
Session: Oligonucleotide Discovery, Preclinical and Clinical

American Society of Gene and Cell Therapy (ASGCT) 29^th Annual Meeting (Boston, MA, May 11–15)

Title: Axiomer™ mediated RNA editing of premature termination codon results in functional correction in MECP2 for Rett syndrome

Oral presentation summary: AX-2402 in Rett syndrome demonstrates reversal of disease phenotype in severe in vivo model, supported by RNAseq analysis showing modulation of genes and neurological pathways relevant to Rett syndrome.
Presenter: Gerard Platenburg, Co-founder and CSO
May 14, 4:00–4:15 PM ET
Session: Programmable epigenetic and RNA editing for precision regulation

Title: Axiomer™ mediated RNA editing converts the pathogenic PNPLA3 I148M variant to a wild-type-like form to reduce hepatic steatosis in MASLD/MASH

Oral presentation summary: AX-2911 demonstrates superiority in fat liver reduction versus RNA knockdown approach in a humanized PNPLA3 I148M in vivo model reflecting disease genetics and progression.
Presenter: Gerard Platenburg, Co-founder and CSO
May 15, 3:30–3:45 PM ET
Session: Expanding cell and gene therapy through therapeutic RNA and epigenome engineering

RNA Society Annual Meeting (Montreal, Canada, May 26–31)

Title: An oligonucleotide design platform for ADAR-mediated RNA editing (ID# 625)

Poster presentation summary: Axiomer™ platform advancements lead to optimization of intracellular journey, EONs achieving efficient cellular uptake, and measurable endosomal escape in vitro, providing productive access to nuclear compartment required for editing.
Presenter: Nandan Varadarajan, Scientist

European Association for the Study of the Liver (EASL) Congress (Barcelona, Spain, May 27–30)

Title: Axiomer^TMmediated RNA editing converts the pathogenic PNPLA3 I148M variant to a wildtype-like form to reduce hepatic steatosis in MASLD/MASH (Abstract SAT-149)

Poster presentation summary: Axiomer RNA editing oligonucleotides effectively convert pathogenic PNPLA3 148M toward the PNPLA3 148V wild-type-like variant and markedly reduce macrovesicular lipid droplets and steatotic burden in a humanized PNPLA3 I148M in vivo model.
Presenter: Werner Helvensteijn, Senior scientist
May 30, 8:30 AM – 4:00 PM CET
Poster Session: MASLD: Experimental and pathophysiology

Presentation slides and posters will be made available in the Publications and Presentations section of the ProQR website at www.proqr.com.

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.

Forward Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our presentations at these conferences, statements regarding our business, technology, strategy, preclinical and clinical model data; our initial pipeline targets and the upcoming strategic priorities and milestones related thereto;the continued advancement of our lead development pipeline programs, including approved, ongoing and planned clinical trials; expectations regarding the ongoing Phase 1 clinical study of AX-0810 in NTCP for cholestatic diseases; expectations regarding the safety and therapeutic benefits of AX-0810, including the planned dosing levels and their efficacy; the anticipated timing of initial Phase 1 clinical data for our lead program in healthy volunteers, AX-0810, in Q2 2026; statements on AX-0422 targeting IDUA in Hurler syndrome, including on the efficacy thereof; statements on MECP2 for Rett syndrome and its efficacy; statements on PNPLA3; statements on the continued development and advancement of our Axiomer^™ platform; the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates; the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto; our patent estate, including our anticipated strength and our continued investment in it; and the potential of our technologies and product candidates; and our cash runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions, U.S. government shutdown and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, high inflation, rising interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor and media contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Investor contact:
Peter Kelleher
LifeSci Advisors
T: +1 617 430 7579
pkelleher@lifesciadvisors.com

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