ProQR Therapeutics N.V.
PRQR evidence brief
What we can confirm
- Market Cap:
- $262.5M
What it means
What to watch
Prepared research question
What matters most for PRQR—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · PRQR
ProQR Therapeutics N.V. is a clinical-stage biopharmaceutical company developing RNA-based therapies, including its proprietary Axiomer RNA editing platform, with a lead program in Phase 1/2 for cholestatic diseases and a cash runway into mid-2027.
Key risk: Early-stage pipeline
Market Cap
$262.5M
Cash Runway
—
Lead Asset
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Next Catalyst
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ProQR Therapeutics N.V. is a clinical-stage biopharmaceutical company developing RNA-based therapies, including its proprietary Axiomer RNA editing platform, with a lead program in Phase 1/2 for cholestatic diseases and a cash runway into mid-2027.
- ProQR ended 2025 with €92.4 million in cash and cash equivalents, providing a runway into mid-2027 [news · 2026-03-12].
- Lead program AX-0810 (targeting NTCP for cholestatic diseases) received CTA authorization and initiated Phase 1 dosing in 2025, with target engagement data expected in H1 2026 [news · 2025-11-06].
- Biliary atresia was selected as the initial Phase 2 indication for AX-0810 [news · 2026-04-09].
- First participant dosed in Phase 1 study of next-generation candidate AX-0811 in September 2026 [news · 2026-09-09].
- ProQR achieved $4.5 million in Eli Lilly collaboration milestones in 2025 [news · 2026-03-12].
What works
- ProQR's Axiomer RNA editing platform is advancing multiple candidates (AX-0810, AX-0811, AX-0422) toward the clinic, demonstrating platform breadth [news · 2026-04-09].
- The company ended 2025 with €92.4 million in cash, providing a financial runway into mid-2027 [news · 2026-03-12].
- ProQR has secured collaboration milestones from Eli Lilly, indicating external validation of its technology [news · 2026-03-12].
What to weigh
- ProQR's lead program AX-0810 is still in early clinical stages (Phase 1/2), with no approved products or revenue from product sales [news · 2025-11-06].
- The company's cash position decreased from €106.9 million at end of Q3 2025 to €92.4 million at year-end 2025, reflecting ongoing cash burn [news · 2025-11-06][news · 2026-03-12].
- ProQR relies on contract manufacturers and suppliers, which poses supply chain and operational risks [sec · filed 2023-12-08].
From the filings
Quoted directly from source documents.
Cash position and runway
“Ended 2025 with € 92.4 million cash and cash equivalents and achieved $4.5 million in Eli Lilly collaboration milestones in 2025, supporting runway into mid-2027”
News · March 12, 2026
Phase 1 initiation for AX-0810
“Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseases”
News · November 6, 2025
Phase 2 indication selection
“biliary atresia selected as initial Phase 2 indication”
News · April 9, 2026
First participant dosed in AX-0811 Phase 1
“first participant has been dosed in the Phase 1 clinical trial of AX-0811 in healthy volunteers”
News · September 9, 2026
Catalyst timeline
- 2026-12-31 · Phase 2 Readout: ax-0810 — Expected Phase 2 topline data readout for ax-0810 in biliary atresia, based on company guidance that biliary atresia was selected as the initial Phase 2 indication.
Risks & what to watch (3)›
Key risks
- Early-stage pipeline — All programs are in Phase 1 or preclinical; no approved products or commercial revenue [news · 2025-11-06].
- Cash burn rate — Cash decreased from €106.9M (Q3 2025) to €92.4M (year-end 2025), indicating ongoing operational spending [news · 2025-11-06][news · 2026-03-12].
- Supply chain reliance — Reliance on contract manufacturers and suppliers for materials, with risk of shortages or delays [sec · filed 2023-12-08].
What to watch
- Phase 2 topline data readout for AX-0810 in biliary atresia, expected by end of 2026 [catalyst].
- Target engagement data from AX-0810 Phase 1 cohorts, expected in H1 2026 [news · 2025-11-06].
- Progress of additional pipeline candidates AX-0811 and AX-0422 toward the clinic [news · 2026-04-09].
- Cash runway updates and any new partnership or financing events [news · 2026-03-12].
Sources: Catalysts · News · SEC filings · Generated October 5, 2026 · How we verify sources →
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Evidence & data
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
69
Total value held
$56.7M
| Holder | Shares | Value |
|---|---|---|
| adage capital partners gp, l.l.c. | 6,253,467 | $10.1M |
| privium fund management b.v. | 5,467,149 | $8.9M |
| aberdeen group plc | 3,316,965 | $5.4M |
| affinity asset advisors, llc | 2,454,278 | $4.0M |
| sio capital management, llc | 1,954,715 | $3.2M |
| dafna capital management llc | 1,567,561 | $2.5M |
| kynam capital management, lp | 1,566,317 | $2.5M |
| ikarian capital, llc | 1,492,229 | $2.4M |
| morgan stanley | 1,299,775 | $2.1M |
| platinum investment management ltd | 1,235,514 | $2.0M |
Showing top 10 of 69 institutional holders of ProQR Therapeutics N.V.. Open the full 13F history after sign-in.
Short Interest
as of May 22, 2026- % of float
- 1.2%
- Days to cover
- 1.0
- Shares short
- 844,972
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
PRQR short interestFrequently asked questions
What is the AI investment thesis for ProQR Therapeutics N.V.?
ProQR Therapeutics N.V. is a clinical-stage biopharmaceutical company developing RNA-based therapies, including its proprietary Axiomer RNA editing platform, with a lead program in Phase 1/2 for cholestatic diseases and a cash runway into mid-2027.
Who is the CEO of ProQR Therapeutics N.V.?
Mr. Daniel Anton de Boer is the chief executive officer of ProQR Therapeutics N.V. (PRQR).
Where is ProQR Therapeutics N.V. headquartered?
ProQR Therapeutics N.V. (PRQR) is headquartered in Leiden, Netherlands.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.