ProQR Therapeutics (PRQR)
Develops Axiomer RNA-editing medicines, led by AX-0810 for cholestatic diseases.
Something off on this page? Send us feedback.
Develops RNA-editing medicines through the Axiomer platform, with AX-0810 in a first-in-human study for cholestatic diseases. The clinical-stage company shifted away from inherited-retinal-disease programs and maintains a liver and nervous-system collaboration with Eli Lilly.
Develops RNA-editing medicines using Axiomer, a platform that employs endogenous ADAR machinery to make targeted single-nucleotide RNA edits. The platform supports programs intended to correct or modulate RNA, following the 2022 shift away from inherited-retinal-disease programs.
AX-0810, an NTCP-targeting editing oligonucleotide for cholestatic diseases, leads the internal pipeline in a first-in-human Phase 1 study. AX-0811 is in late non-clinical development, and AX-0422 targets Hurler syndrome in non-clinical development.
Based in Leiden, the Netherlands, with a U.S. office in Cambridge, Massachusetts, the clinical-stage organization also works with Eli Lilly on potential medicines for genetic disorders in the liver and nervous system. Progress depends on clinical-trial execution and regulatory interactions.
Company facts
Target rangeAs of Sep 23, 2026
Latest analyst notes
| Date | Firm | Action | Prior | Current |
|---|---|---|---|---|
| Aug 20, 2026 | Bank of America Securities | Initiate | Rating: — | |
| Jun 25, 2026 | Chardan Capital | Target | Target: $2 | Target: $3 |
| Oct 29, 2024 | Raymond James | Upgrade |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $133.3M · Total debt $15.0M · Total assets $152.0M · Shareholders' equity $90.5M
- Revenue
- $10.1M
- Operating income
- —
- Net income
- −$10.1M
Reported in EUR, converted at the latest exchange rate
No dividends on record.
| Institutions | 57.65% | 60.74M ADS | |
|---|---|---|---|
| 71 holders · as of Jun 30, 2026 | |||
| Insiders | 2.33% | 2.46M ADS | |
| 6 holders · as of Aug 3, 2026 | |||
| Other | 40.02% | 42.17M ADS | |
| remainder | |||
105.36M ADS as of Sep 23, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Date |
|---|---|---|---|
Adage Capital Partners GP, L.L.C.Institution | 8.56% | 9.02M ADS | as of Jun 30, 2026 |
Abrdn PLCInstitution | 8.48% | 8.93M ADS | as of Jun 30, 2026 |
Adage Capital Management, L.P.Institution | 6.27% | 6.60M | filed Aug 12, 2025 |
Affinity Asset Advisors, LLCInstitution | 5.26% | 5.54M ADS | as of Jun 30, 2026 |
Privium Fund Management B.V.Institution | 5.24% | 5.52M ADS | as of Jun 30, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Jul 31, 2026 | Daniel Anton de Boer | director, officer: Chief Executive Officer | +200,000 | $1.77 |
| Jul 17, 2026 | Bart Filius | director | +150,000 | $1.72 |
| Jul 9, 2026 | Daniel Anton de Boer | director, officer: Chief Executive Officer | +150,000 | $1.74 |
| Jul 7, 2026 | Daniel Anton de Boer | director, officer: Chief Executive Officer | +150,000 | $1.59 |
| Jun 26, 2026 | Bart Filius | director | +150,000 | $1.54 |
- Wave Life Sciences
ADAR RNA editing and GalNAc liver medicines
- Korro Bio
OPERA-based ADAR editing and GalNAc liver programs
- Mirum Pharmaceuticals
Rare cholestatic-liver medicines and IBAT inhibition
CompareAfter hours
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| ProQR Therapeutics | $1.73 | +2.37% | $200.1M | — | — | 9.4 | +122.6% |
| Wave Life Sciences | $3.95 | −0.63% | $857.2M | — | — | 13.1 | −73.9% |
| Korro Bio | $9.83 | +0.10% | $102.9M | — | — | 43.2 | +2.7% |
| Mirum Pharmaceuticals | $90.00 | +0.68% | $5.57B | — | — | 9.0 | +37.9% |
| Median | — | — | 13.1 | +2.7% |