ProQR Therapeutics N.V., a biopharmaceutical company, engages in the discovery and development of RNA-based therapeutics for the treatment of genetic disorders. It is primarily developing sepofarsen that is in phase II/III illuminate trial for treating leber's congenital amaurosis 10 disease; QR-421a, which is in phase I/II stellar trial for usher syndrome type 2 and non-syndromic retinitis pigmentosa; QR-1123 that is in phase I/II aurora trial for the treatment of autosomal dominant retinitis pigmentosa; and QR-504a, which is in first clinical trial for the treatment of fuchs endothelial corneal dystrophy. It has a license agreement with Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. ProQR Therapeutics N.V. was incorporated in 2012 and is headquartered in Leiden, the Netherlands.
IPO Year: 2014
Exchange: NASDAQ
Website: proqr.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 4/29/2025 | $5.00 | Outperform | Evercore ISI |
| 4/29/2025 | $8.00 | Overweight | Cantor Fitzgerald |
| 3/10/2025 | $4.00 | Neutral → Buy | Citigroup |
| 1/10/2025 | $15.00 | Outperform | Oppenheimer |
| 10/29/2024 | $6.00 → $14.00 | Outperform → Strong Buy | Raymond James |
| 11/8/2023 | $2.00 | Neutral → Buy | Chardan Capital Markets |
| 3/30/2023 | $1.50 → $5.00 | Mkt Perform → Mkt Outperform | JMP Securities |
| 12/22/2022 | $0.80 → $5.00 | Neutral → Overweight | Cantor Fitzgerald |
| 2/14/2022 | $18.00 → $8.00 | Buy | Chardan Capital |
| 2/14/2022 | $37.00 → $1.70 | Buy → Neutral | Citigroup |
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Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseasesAdvancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett SyndromeHosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 Phase 1 trial design and 2025 data expectations, plus updates across differentiated liver and CNS pipeline€ 119.8 million cash and cash equivalents as of end Q2 – providing runway into mid-2027, not including additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 07, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through tran
LEIDEN, Netherlands & CAMBRIDGE, Mass., July 28, 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 participate in the RNA Editing Summit taking place July 29-31, 2025, in Boston, MA. "We recently achieved a key milestone with the submission of the CTA for AX-0810, targeting NTCP for cholestatic diseases, and we're building on this momentum with continued progress in CNS applications of our Axiomer technology," said Gerard Platenburg, Chief Scientific Officer of ProQR. "Our presentation at the RNA Edit
First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025Marks the first submitted Clinical Trial Application (CTA) advancing ProQR's Axiomer™ ADAR-mediated RNA editing platform into clinical development LEIDEN, Netherlands & CAMBRIDGE, Mass., June 26, 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 the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a
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 breadt
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Strengthened leadership with appointments of Chief Financial Officer and Chief Medical Officer€ 132.4 million cash and cash equivalents as of end Q1 providing runway into mid-2027, plus additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today r
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 02, 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 Company management is scheduled to present at the Citizens (JMP) Life Sciences Conference in New York City on Thursday, May 8 at 11:30 AM EDT. A live webcast of the Company's fireside chat will be available from the "Investors & Media" section of ProQR's website under "Events". A replay of the webcast will then be available for approximately 30 days. About Axiomer™ ProQR is pioneering a next-generation RNA base
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 01, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (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 Annual General Meeting (AGM) of Shareholders will take place on Tuesday, June 3, 2025 at 1530 CEST at the offices of Allen Overy Shearman Sterling LLP, in Amsterdam, the Netherlands. All relevant documents and information for the meeting, including the notice and agenda and explanatory notes, are or will be made available in the "Investors & Media" section of ProQR's website (www.proqr.com) under "Financials and
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 14, 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 the appointment of Dennis Hom as Chief Financial Officer (CFO) and Cristina Lopez Lopez, MD, PhD, as Chief Medical Officer (CMO). These key leadership appointments support the advancement of the Company's Axiomer platform technology and pipeline of RNA editing programs as it enters the clinical stage. "We are thrilled to welcome Dennis and Cristina to ProQR at such an important time in our evolution," said Daniel A
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expects up to four clinical data readouts in 2025 and 2026 across multiple programs, highlighted by first clinical data for AX-0810 anticipated in Q4 2025€ 149.4 million cash and cash equivalents as of end of 2024, providing runway into mid-2027, plus additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., March 13, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapie
LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 11, 2024 (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 an expansion of its collaboration with the Rett Syndrome Research Trust (RSRT). Building on the initial $1 million research grant announced in January 2024, the expanded partnership includes an additional $8.1 million in funding from the RSRT, for a total of $9.1 million. The funding will support the advancement of AX-2402 into clinical trials. Rett Syndrome, a rare and devastating neurodevelopmental disorder, affe
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Evercore ISI initiated coverage of ProQR Therapeutics with a rating of Outperform and set a new price target of $5.00
Cantor Fitzgerald resumed coverage of ProQR Therapeutics with a rating of Overweight and set a new price target of $8.00
Citigroup upgraded ProQR Therapeutics from Neutral to Buy and set a new price target of $4.00
Oppenheimer initiated coverage of ProQR Therapeutics with a rating of Outperform and set a new price target of $15.00
Raymond James upgraded ProQR Therapeutics from Outperform to Strong Buy and set a new price target of $14.00 from $6.00 previously
Chardan Capital Markets upgraded ProQR Therapeutics from Neutral to Buy and set a new price target of $2.00
JMP Securities upgraded ProQR Therapeutics from Mkt Perform to Mkt Outperform and set a new price target of $5.00 from $1.50 previously
Cantor Fitzgerald upgraded ProQR Therapeutics from Neutral to Overweight and set a new price target of $5.00 from $0.80 previously
Chardan Capital reiterated coverage of ProQR Therapeutics with a rating of Buy and set a new price target of $8.00 from $18.00 previously
Citigroup downgraded ProQR Therapeutics from Buy to Neutral and set a new price target of $1.70 from $37.00 previously
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Live Leadership Updates
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 14, 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 the appointment of Dennis Hom as Chief Financial Officer (CFO) and Cristina Lopez Lopez, MD, PhD, as Chief Medical Officer (CMO). These key leadership appointments support the advancement of the Company's Axiomer platform technology and pipeline of RNA editing programs as it enters the clinical stage. "We are thrilled to welcome Dennis and Cristina to ProQR at such an important time in our evolution," said Daniel A
LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 10, 2024 (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 the appointment of Dr. Peter Beal, a professor of Chemistry at the University of California, Davis, as Chief ADAR Scientist. Dr. Beal, one of the top experts in ADAR and RNA biology and chemistry, has been a long-term collaborator of ProQR and a valued member of ProQR's Scientific Advisory Board, where he has played an important role in shaping the Company's ADAR-based RNA editing platform. Dr. Beal's decades of res
LEIDEN, Netherlands & CAMBRIDGE, Mass., March 07, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced that John Maraganore, PhD, biopharma industry leader and the former founding CEO of Alnylam Pharmaceuticals, has joined ProQR as a strategic advisor. "It is a great honor to have John engaged with ProQR as a strategic advisor to the Supervisory Board to support us in defining the next steps for the Company, while we continue to analyze data from the Illuminate trial of sepofarsen and identify next steps for the program, if any,"
Top-line data from pivotal Phase 2/3 Illuminate trial of sepofarsen for CEP290-mediated LCA10 anticipated late Q1/early Q2 2022Five-target collaboration with Lilly highlights significant potential of ProQR's Axiomer® RNA base-editing platform and strengthens financial position with $50 million in upfront and equity and up to $1.25 billion in milestonesQR-421a Phase 2/3 Sirius and Celeste trials in Usher syndrome and retinitis pigmentosa on track to start by year end 2021Company to host an "Analyst Event" on November 18, 2021 to highlight clinical stage pipeline programs and Axiomer® RNA editing platform LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 04, 2021 (GLOBE NEWSWIRE) -- ProQR The
Senior leader with extensive global rare disease commercialization experienceTop-line data from Phase 2/3 pivotal Illuminate trial of sepofarsen for CEP290-mediated LCA10 on track for H1 2022 LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 04, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced the appointment of Theresa Heggie as Chief Commercial Officer reporting to Daniel de Boer, Founder and CEO. In this newly created leadership position, Ms. Heggie will be responsible for overseeing the Company's commercial strategy and global comme
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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 on Wednesday, December 11, 2024 from 10:00 am until approximately 12:30 pm EST, including an Analyst Q&A session with members of the ProQR Management Team. During the event, ProQR will highlight its proprietary Axiomer™ 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. Prese
ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer™ RNA Editing OligonucleotidesPreclinical proof of concept for the Company's AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT Annual MeetingManagement webinar May 9, 2024 at 8:00 am EDT LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced new preclinical data for its proprietary Axiomer™ RNA editing technology platform, incl
LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 22, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced that ProQR management will host an investor conference call and webcast to discuss the Company's Axiomer RNA editing technology platform following the recently announced partnership expansion with Lilly. ProQR will host the call on December 22, 2022, at 8:15am EST. The live and archived webcast of the presentation will be accessible through this webcast link, or to access the live call by phone please register here. A dial-in and unique PIN will be provided to join the call. The
In line with corporate strategy update in April, ProQR to focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas; update on initial pipeline targets expected in late 2022/early 2023Additional pivotal trial recommended by EMA for sepofarsen prior to a regulatory submissionCompany will seek a strategic partner for ophthalmology assets Workforce reduction to be implemented within clinical stage ophthalmology programs Cash runway extended into 2026 Conference call today at 8:15am EDT LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 11, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedica
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3Illuminate results and post-hoc analyses to be presented at the upcoming Seventh Annual Retinal Cell and Gene Therapy Innovation Summit and the ARVO 2022 Annual MeetingCorporate strategy to focus on accelerating the development of Axiomer® RNA base-editing technology platform across multiple therapeutic areas; Company to continue development of select Inherited Retinal Disease (IRD) programs, subject to regulatory feedbackCompan
Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be conducted and presented at a future medical congressSepofarsen continues to be generally well toleratedStrong cash position provides runway into mid- to late-2024ProQR Management to host conference call today at 8:15am EST LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 11, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced its pivotal Phase 2/3 Illuminate trial of sepofarsen for th
LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 08, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced that ProQR management will host an investor conference call and webcast to discuss the Company's Axiomer RNA editing technology platform following the recently announced partnership with Lilly. ProQR will host the call on September 9, 2021, at 8:15am EDT. The live and archived webcast of this presentation will be accessible through this webcast link or through the Events page of the Company's website. The dial-in details for the call are