LEIDEN, Netherlands & CAMBRIDGE, Mass., March 07, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced several presentations highlighting the Company’s proprietary Axiomer® RNA editing technology platform at upcoming scientific and industry conferences, including the 8th annual Oligonucleotide and Precision Therapeutics (OPT) Congress, March 13-14, 2023 in Boston, MA, U.S.; RNA Leaders Europe Congress, March 15-16, 2023, in Basel, Switzerland; and the bi-annual RNA Editing Gordon Research Conference (GRC), March 19-24, 2023 in Ventura, CA, U.S.
Presentation and panel session at the Oligonucleotide and Precision Therapeutics (OPT) Congress
Oral presentation title: Therapeutic potential of an RNA editing platform using Editing Oligonucleotides (EONs)
Presenter: Gerard Platenburg, CSO and co-founder, ProQR Therapeutics
Session: Oligonucleotide Discovery and Delivery - Optimizing Design, Delivery and Performance
Date: Tuesday, March 14 – 9:35 am EDT
Abstract: Axiomer, an RNA editing technology, uses Editing Oligonucleotides (EONs) to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA (ADAR). This presentation will highlight the therapeutic possibilities of this platform that are not limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to engineer proteins or modify their function as well as creating de novo mutations.
Panel session title: Integrating Innovative Technologies into Oligo Therapeutics Discovery and Delivery
Presenter: Gerard Platenburg, CSO and co-founder, ProQR Therapeutics
Session: Oligonucleotide Discovery and Delivery - Optimizing Design, Delivery and Performance
Date: Tuesday, March 14 – 10:05 am EDT
Presentation at RNA Leaders Europe Congress
Oral presentation title: Unlocking the potential of Axiomer® platform to develop transformative RNA editing therapies
Presenter: Daniel A. de Boer, Founder and CEO, ProQR Therapeutics
Date: Thursday, March 16 – 14:35 CET
Abstract: The presentation will describe: Axiomer, the next-generation RNA technology using the cell’s own editing machinery; explore therapeutic opportunities of Axiomer, beyond disease-causing mutations; and the advancement of Axiomer through in-house pipeline programs and partnering strategy.
Presentation at the RNA Editing Gordon Research Conference (GRC)
Oral presentation title: Development of RNA editing oligonucleotides technology to address liver originated disorders
Presenter: Gerard Platenburg, CSO and co-founder, ProQR Therapeutics
Date: Thursday, March 23
Abstract: Axiomer, an RNA editing platform, uses chemically modified and complementary single stranded editing oligonucleotides (EONs) to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA (ADAR). Through now further developed design principles there has been an established improvement of specificity and efficacity of EONs, and we have tested the RNA editing efficacy and short-term safety of EONs acting on various genes and models. The increasing knowledge on the platform have unlocked therapeutic applications beyond disease-causing mutations and the presentation will review the potential of Axiomer platform in multiple organs as well as its applicability for the treatment of liver originated disorders including data from preclinical models.
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 "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 these events, as well as the potential of our Axiomer RNA editing technology platform 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 annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies 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; 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 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; and general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties. 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.
For ProQR Therapeutics N.V.
Investor contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Hans Vitzthum
LifeSci Advisors
T: +1 617 430 7578
hans@lifesciadvisors.com
Media contact:
Robert Stanislaro
FTI Consulting
T: +1 212 850 5657
robert.stanislaro@fticonsulting.com
