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ProQR Therapeutics
↗Leiden, Netherlands
ProQR Therapeutics N.V. is a Dutch, public, clinical-stage biotechnology company developing RNA-editing medicines through its proprietary Axiomer platform. Axiomer uses editing oligonucleotides to recruit endogenous ADAR enzymes and make targeted adenosine-to-inosine RNA edits, with potential applications in mutation correction, protein-expression modulation, and protective-variant engineering. The company is focused primarily on liver and central-nervous-system diseases with significant unmet medical need. ([sec.gov](https://www.sec.gov/Archives/edgar/data/1612940/000110465926026713/prqr-20251231x20f.htm?utm_source=openai))
ProQR's current lead program is AX-0810, a GalNAc-conjugated RNA-editing oligonucleotide targeting NTCP for cholestatic diseases, including biliary atresia. The company is also advancing AX-0811 for cholestatic disease, AX-0422 for Hurler syndrome, AX-2911 for MASH, and AX-2402 for Rett syndrome, while maintaining a partnered discovery pipeline with Eli Lilly. ([proqr.com](https://www.proqr.com/science/pipeline?utm_source=openai))
CLASSIFICATION
Company Type:Biotech
Therapeutic Areas:
Industry:Biotechnology
Sub-Industry:RNA editing and genetic medicines
SIZE & FINANCIALS
Employees:51-200
Revenue:€0-€50M annual range; Q1 2026 revenue was €2.033M. ([proqr.com](https://www.proqr.com/press-releases/proqr-announces-first-quarter-2026-operating-and-financial-results))
Founded:2012
Ownership:public
Status:operating
FUNDING
Stage:Public clinical-stage biotech
Total Raised:$275M+ in selected disclosed financings, equity investments, and partner funding since 2021; this is not a complete lifetime funding total. ([proqr.com](https://www.proqr.com/press-releases/proqr-announces-axiomerr-rna-editing-licensing-and-research-collaboration-with-lilly?utm_source=openai))
Investors:Eli Lilly and Company: strategic shareholder and Axiomer collaboration partner; purchased approximately $9.2M of shares in the June 2026 financing. ([proqr.com](https://www.proqr.com/press-releases/proqr-prices-500-million-underwritten-registered-direct-offering-and-concurrent-private-placement?utm_source=openai)), Rett Syndrome Research Trust: provided up to approximately $9.1M in research funding for the AX-2402 Rett syndrome program. ([proqr.com](https://www.proqr.com/press-releases/proqr-therapeutics-announces-81-million-in-new-funding-from-rett-syndrome-research-trust-to-expand-rna-editing-collaboration?utm_source=openai))
STOCK
Exchange:NASDAQ
Ticker:PRQR
Market Cap:$181M as of August 19, 2026; latest quoted price approximately $2.15 per share.
PIPELINE
Stage:Phase 1
Lead Drug Stage:AX-0810 is in Phase 1 in healthy volunteers, with positive target-engagement data reported from the evaluable 3 mg/kg and 6 mg/kg cohorts; additional 9 mg/kg and follow-up data were expected by year-end 2026. ([proqr.com](https://www.proqr.com/press-releases/proqr-announces-positive-phase-1-target-engagement-data-for-ax-0810-establishing-first-clinical-validation-of-the-axiomer-rna-editing-platform))
Modalities:RNA editing, Editing oligonucleotides, ADAR-mediated RNA editing, GalNAc-conjugated oligonucleotides, Antisense oligonucleotides, RNA repair
Active Trials:1
Trial Phases:Phase 1: 1
FDA Approvals:0
EMA Approvals:0
CORPORATE STRUCTURE
Subsidiaries:ProQR Therapeutics VIII B.V., a ProQR entity named in the Lilly research and collaboration agreement. ([sec.gov](https://www.sec.gov/Archives/edgar/data/1612940/000110465921113798/tm2127141d1_ex10-1.htm?utm_source=openai)), ProQR Therapeutics II B.V., identified in public patent records as a ProQR entity. ([patents.google.com](https://patents.google.com/patent/GB202405143D0/en?utm_source=openai))
Key Partnerships:Eli Lilly and Company: global licensing and research collaboration for Axiomer-based genetic medicines. The 2021 agreement covered up to five targets and included $50M in upfront payment and equity investment, up to approximately $1.25B in milestones, and tiered royalties. The 2022 expansion added targets, $75M in upfront and equity consideration, and increased aggregate potential milestones to approximately $3.75B. ([proqr.com](https://www.proqr.com/press-releases/proqr-announces-axiomerr-rna-editing-licensing-and-research-collaboration-with-lilly?utm_source=openai)), Rett Syndrome Research Trust: collaboration focused on Axiomer editing oligonucleotides targeting MECP2 mutations; expanded in December 2024 with total funding of approximately $9.1M-$9.2M. ([proqr.com](https://www.proqr.com/press-releases/proqr-therapeutics-announces-81-million-in-new-funding-from-rett-syndrome-research-trust-to-expand-rna-editing-collaboration?utm_source=openai)), Ginkgo Bioworks: collaboration providing access to Ginkgo's autonomous-lab infrastructure, including the 50-plus-instrument Nebula platform, to generate high-throughput data for ProQR's AI-enabled discovery engine. ([proqr.com](https://www.proqr.com/press-releases/proqr-announces-partnership-with-ginkgo-bioworks-and-formation-of-ai-advisory-board?utm_source=openai)), University of California, Davis: collaboration with Professor Peter Beal relating to ADAR biology and Axiomer intellectual property. ([sec.gov](https://www.sec.gov/Archives/edgar/data/1612940/000110465926026713/prqr-20251231x20f.htm)), Freie Universität Berlin: co-applicant on Axiomer-related patent work involving Professor Alexander Weng. ([sec.gov](https://www.sec.gov/Archives/edgar/data/1612940/000110465926026713/prqr-20251231x20f.htm)), University of Rochester: collaboration involving the TRIDENT pseudouridylation program. ([sec.gov](https://www.sec.gov/Archives/edgar/data/1612940/000110465926026713/prqr-20251231x20f.htm))
COMPETITION
Position:Emerging
Competitors:Korro Bio, Wave Life Sciences, ADARx Pharmaceuticals, Beam Therapeutics, Shape Therapeutics
LEADERSHIP
Key Executives:
Daniel A. de Boer - Founder and Chief Executive Officer
Gerard Platenburg - Chief Scientific Officer and Co-founder
Dennis Hom - Chief Financial Officer
Cristina Lopez Lopez, MD, PhD - Chief Medical Officer
Peter A. Beal, PhD - Chief ADAR Scientist
Sarah Kiely - Vice President, Investor Relations and Corporate Affairs
Scientific Founders:Daniel A. de Boer, Gerard Platenburg, Dinko Valerio, Henri A. Termeer, deceased 2017
Board Members:James Shannon, MD — Chairperson and non-executive director; former Chief Medical Officer of GlaxoSmithKline and former Global Head of Pharma Development at Novartis. ([proqr.com](https://www.proqr.com/leadership)), Bart Filius — Non-executive director; former President and COO of Galapagos and former CFO and M&A executive at Sanofi. ([proqr.com](https://www.proqr.com/leadership)), Begoña Carreño, PhD — Non-executive director; Chief Business Development Officer at Aspeya Switzerland and former worldwide BD&L head in Novartis Ophthalmology. ([proqr.com](https://www.proqr.com/leadership?utm_source=openai)), Martin Maier, PhD — Non-executive director; Senior Vice President, Oncology at Alnylam, with extensive RNAi, GalNAc, oligonucleotide, and lipid-nanoparticle experience. ([proqr.com](https://www.proqr.com/leadership)), Lykke Hinsch Gylvin, MD, EMBA — Non-executive director appointed in 2026; Chief Medical Officer and Head of Global Medicine at Boehringer Ingelheim. ([proqr.com](https://www.proqr.com/press-releases/proqr-announces-nomination-of-dr-lykke-hinsch-gylvin-to-its-board-of-directors?utm_source=openai)), Daniel A. de Boer — Executive director and Founder/CEO. ([proqr.com](https://www.proqr.com/leadership)), Gerard Platenburg — Executive director and Chief Scientific Officer/co-founder. ([proqr.com](https://www.proqr.com/leadership))
LINKS
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