ProQR Therapeutics to Host Virtual Analyst and Investor Event on December 11, 2024

PRQR 12.05.2024

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Recent News

  • 12.11.2024 - ProQR Therapeutics Announces $8.1 Million in New Funding from Rett Syndrome Research Trust to Expand RNA Editing Collaboration
  • 12.10.2024 - ProQR Appoints Peter A. Beal, PhD, as Chief ADAR Scientist
  • 12.05.2024 - ProQR Therapeutics to Host Virtual Analyst and Investor Event on December 11, 2024

Recent Filings

  • 12.12.2024 - 6-K/A Report of foreign issuer [Rules 13a-16 and 15d-16]
  • 12.11.2024 - EX-99.1 EX-99.1

LEIDEN,Netherlands&CAMBRIDGE, Mass.,Dec. 05, 2024(GLOBE NEWSWIRE) --ProQR Therapeutics N.V.(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced that the Company will host a virtual Analyst and Investor Event onWednesday, December 11, 2024from10:00 amuntil approximately12:30 pm EST, including an Analyst Q&A session with members of the ProQR Management Team.

During the event, ProQR will highlight its proprietary AxiomerTM ADAR-mediated RNA editing platform, along with updates on its pipeline of development candidates including data updates and next steps on its programs for NTCP and B4GALT1, AX-0810 and AX-1412. Presentations will feature members of the Management Team includingDaniel A. de Boer, Founder and CEO, andGerard Platenburg, Chief Scientific Officer;René Beukema, Chief Corporate Development Officer will also join the analyst Q&A session with the Management Team.

Additionally, the following speakers will present at the event:

  • Peter Beal, PhD, will share his perspectives on driving innovation in the ADAR field.Dr. Bealis a Professor in theDepartment of Chemistryat theUniversity of CaliforniaatDavisand Director of theNIH-funded UC Davis Chemical Biology Graduate Program and member of theScientific Advisory Boardat ProQR.
  • Prof.Gideon Hirschfield, MA(Oxon) MB BChir (Cantab) FRCP PhD, a key opinion leader, will share his views on the unmet need in the field and the opportunity for AX-0810. He is Professor of Gastroenterology and Hepatology,Toronto Centre for Liver Disease.

More detailed biographies are available below.

Event Registration Details

To register for the Investor and Analyst webcast (December 11, 2024), please clickhere. A live webcast of the event will be available under “Events” in the “Investors & Media” section of ProQR’s website athttps://www.proqr.com/events.

To join via phone, participants may preregister to receive dedicated dial-in details to access the call via the following website:

https://register.vevent.com/register/BI219c7b4f854c4c718b45b428b613d113

It is suggested that participants dial into the conference call 15 minutes prior to the scheduled start time to avoid any delays in attendance. An archived version of the webcast will be available for replay via ProQR’s website for approximately 30 days following the event.

Speaker Biographies

Peter A. Beal, PhD

Peter A. Bealis a Professor in theDepartment of Chemistryat theUniversity of CaliforniaatDavisand current Director of theNIH-funded UC Davis Chemical Biology Graduate Program. Research from the Beal laboratory has advanced understanding of the structures and mechanism of action for ADAR enzymes responsible for adenosine to inosine RNA editing in humans. Recently, this knowledge has been applied to the design of guiding oligonucleotides capable of directing ADARs to make edits that correct disease-causing mutations in the transcriptome. Targets include mutations in mRNAs associated with Rett syndrome, Alzheimer’s disease and Parkinson’s disease.Dr. Bealteaches organic chemistry at the undergraduate level and several classes in nucleic acids chemistry and chemical biology at the graduate level.

Prof.Gideon Hirschfield, MA(Oxon) MB BChir (Cantab) FRCP PhD

Prof.Gideon M. Hirschfieldis a distinguished clinician-scientist specializing in autoimmune liver diseases. He holds the Lily andTerry Horner ChairinAutoimmune Liver Disease Researchat theToronto Centre for Liver Disease,Toronto General Hospital, and serves as a Professor of Medicine in theDivision of Gastroenterologyat theUniversity of Toronto.

Prof. Hirschfieldearned his medical degree from theUniversity of Cambridgein 1996 and completed his undergraduate studies at theUniversity of Oxfordin 1994. He was awarded a PhD from theUniversity of Londonin 2006. His postgraduate training included positions atHammersmith HospitalandRoyal Brompton HospitalinLondon, as well as a fellowship inToronto, Canada.

An internationally recognized expert,Prof. Hirschfieldhas published over 200 peer-reviewed articles, including lead authorship in high-impact journals such as theNew England Journal of Medicine,The Lancet, and Nature Genetics.

His research focuses on advancing therapies for inflammatory liver diseases to prevent the need for transplantation

About AxiomerTM

ProQR is pioneering a next-generation RNA base editing technology called AxiomerTM, which could potentially yield a new class of medicines for diverse types of diseases. AxiomerTM“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). AxiomerTMEONs 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 Therapeuticsis dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called AxiomerTM, 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 atwww.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, express and implied statements regarding this event, the potential of our technologies and product candidates, our business, technology, strategy, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our AxiomerTMplatform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates, our development programs, including the timing, progress and results of our preclinical studies, clinical trials and other development activities, including the release of data related thereto, our business operations, 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 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 theSecurities 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 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 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 Eli Lilly and Company; 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.

ProQR Therapeutics N.V.

Investor contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorPeter KelleherLifeSci AdvisorsT: +1 617 430 7579pkelleher@lifesciadvisors.com

Media contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com

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Source: ProQR Therapeutics N.V.

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