ProQR Announces Upcoming Scientific Presentations at ASGCT and TIDES Conferences

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- 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 that it will present at two upcoming scientific conferences: the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, taking place May 13-17, 2025, in New Orleans, Louisiana and the TIDES USA 2025: Oligonucleotide and Peptide Therapeutics Conference, being held May 19-22, 2025, in San Diego, California.

"We are excited to share these presentations with the scientific community, demonstrating the breadth and versatility of our Axiomer RNA editing technology," said Gerard Platenburg, Chief Scientific Officer of ProQR. "The data we will present at ASGCT and TIDES further support our vision of building a differentiated and diversified pipeline and advancing RNA editing therapies that could offer transformative benefits for patients."

These presentations will highlight the Company's proprietary Axiomer RNA editing platform and pipeline programs. Details of ProQR's presentations are as follows:

American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting

May 13-17, 2025 | New Orleans, Louisiana, USA

Oral Presentation

• Title: ADAR-Mediated RNA Editing of SLC10A1(NTCP) as a Therapeutic Approach to Reduce Liver Bile Acid Re-Uptake in Cholestatic Diseases (abstract 333)
• Presenter: Gerard Platenburg, Chief Scientific Officer
• Date/Time: May 16, 2025, 4:15 PM CT
• Session: Oligonucleotide Therapeutics II

Poster Presentations

• Title: ADAR-Mediated RNA Editing-Based Correction of PNPLA3 I148M Functionality to Address Hepatic Steatosis (abstract 1654)
  • Presenter: Gerard Platenburg, Chief Scientific Officer
  • Date/Time: May 15, 2025, 5:30-7:00 PM CT
  • Poster Number: AMA1324
• Title: ADAR-Mediated RNA Editing of Premature Termination Codon Results in Functional Correction in MECP2 for Rett Syndrome (abstract 1645)
  • Presenter: Gerard Platenburg, Chief Scientific Officer
  • Date/Time: May 15, 2025, 5:30-7:00 PM CT
  • Poster Number: AMA1427

TIDES USA: Oligonucleotide and Peptide Therapeutics Conference

May 19-22, 2025 | San Diego, California, USA

Podium Presentation

• Title: Advancing Axiomer ADAR RNA Editing Platform - Editing oligonucleotides optimization for therapeutic use
• Presenter: Peter A. Beal, PhD, Chief ADAR Scientist
• Date/Time: May 22, 2025, 2:00 PM PST
• Session/Track: Genome Editing Technology and Applications

Following the conferences, presentation materials 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 for ProQR

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, our business, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer™ RNA editing technology platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to expand preclinical in vivo and in vitro data, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, and the potential of our technologies and product candidates, as well as the timing of our clinical development. 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 anticipated in 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; 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 or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; 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; 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 instability and conflicts. 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. 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 "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 participation in this conference, our business, technology, strategy, our Axiomer platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides, and the potential of our technologies and product candidates. 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 anticipated in 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 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

[email protected]

or

Investor contact:

Peter Kelleher

LifeSci Advisors

T: +1 617 430 7579

[email protected]