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ProQR Therapeutics

PRQR Rare disease Lead: Phase 1 2 Biotech Distilled articles ↓ 28 filings & releases ↓ Cash €92.4M (Dec 31, 2025) ↓ 2 verdicts ↓ 2 timing quotes ↓
MetabolicNeurology Nucleic Acid

Market cap $191M 141.1M shares (vendor count, Nov 7, 2026) × $1.35 close Nov 6, 2026 · share count from the market-data vendor, not the SEC cover page

Most recent annual filing: 2026-03-12

Catalyst timeline

8 trackedQuarter catalyst calendar →
  1. AX-0810 Ph1 readoutDec 9, 2026AX-0810 Ph1 readout, Dec 9, 2026, major
  2. AX-0811 Ph1 interimJan 2027AX-0811 Ph1 interim, Jan 2027, major
  3. AX-0810/biliary atresia IIT readout1H 2027AX-0810/biliary atresia IIT readout, 1H 2027, material
  4. AX-0422 readout1H 2027AX-0422 readout, 1H 2027, material
  5. AX-0422 filingearly 2027AX-0422 filing, early 2027, material
  6. AX-2911 trial start1H 2027AX-2911 trial start, 1H 2027, routine
  7. AX-0810/biliary atresia Ph2 startMid-2027AX-0810/biliary atresia Ph2 start, Mid-2027, notable
  8. AX-0810/biliary atresia Phase 2 Ph2mid-2028AX-0810/biliary atresia Phase 2 Ph2, mid-2028, major

Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.

Pipeline (7 active programs)

Technology platforms

Axiomer

Next-generation RNA base editing technology using ADAR (Adenosine Deaminase Acting on RNA) machinery; Editing Oligonucleotides (EONs) mediate single nucleotide A-to-I changes in RNA to correct disease-causing mutations, modulate protein expression, or alter protein function; applicable to both rare and prevalent diseases with unmet need.

Targets: ADAR, NTCP, IDUA, PNPLA3

Axiomer · Eli Lilly

Next-generation RNA base editing technology using endogenous ADAR machinery to make single nucleotide A-to-I edits in RNA; applicable to both rare and prevalent diseases with unmet need

Targets: ADAR

Axiomer · Eli Lilly and Company

Next-generation RNA base editing technology using endogenous ADAR machinery to make A-to-I single nucleotide edits in RNA; designed to correct disease-causing mutations, modulate protein expression, or alter protein function; applicable to both rare and prevalent diseases including cholestatic liver diseases, MPS I Hurler syndrome, and MASH.

Targets: ADAR, NTCP, IDUA, PNPLA3, B4GALT1

Axiomer · Ginkgo Bioworks

Next-generation RNA base editing technology using ADAR (Adenosine Deaminase Acting on RNA) to make single nucleotide edits in RNA via Editing Oligonucleotides (EONs); applications across multiple tissues and disease areas including rare and prevalent diseases. Integration of AI-enabled discovery and high-throughput screening to accelerate design and optimization.

Targets: ADAR

Capital (as filed)

Cash
€92.4M cash and cash equivalents · Dec 31, 2025 (20-F)
Operating cash flow
−€13.2M annual ÷ 4, year to Dec 31, 2025 (20-F)
As stated in coverage
€136M cash, runway mid-2028 per the deep dive of Aug 31, 2026 — the company's own words as read there, not the filed figure; stated in EUR

Cash, investments and operating cash flow are the company's own XBRL facts as filed with the SEC, each shown with its period end and tag. As-stated figures come from the company's own words as read in Biotech Distilled coverage, dated, and routinely count securities and planned financing that a balance sheet does not. Offering rows are the SEC cover pages as filed. Not investment advice. Figures as of Nov 7, 2026.

Biotech Distilled coverage (2)

Articles open on Substack. Some are subscriber-only; those marked ★ Distilled Pro are for Distilled Pro members.

Verdict history (2 calls)

Every verdict Biotech Distilled has published on ProQR Therapeutics, oldest first — the full history, never a selection. How calls are scored: the track record.

  1. 2026-09-01 Watch ProQR Therapeutics (PRQR) - Scientific Deep Dive for AX-0810, AX-0811 and the Axiomer RNA Editing Pipeline The editor cleared its pre-declared bar and the balance sheet is funded through mid-2028, but the lead indication's mechanistic neighborhood just failed two Phase 3s, editing percentage is modeled not measured, and the capital structure is in open conflict with a 10.9% holder.
  2. 2026-09-10 Speculative buy speculative UPDATE: ProQR Therapeutics (PRQR) - The Gate Opened, and the Clock Moved The CTA authorization gate — the explicit condition set for engaging with this name — has cleared, retiring a binary administrative risk, warranting a small speculative position sized at 1–1.5% of the book into the January AX-0811 print.

Every verdict Biotech Distilled has published on this company, in order, with the post it came from — the full history, never a selection. Position figures are the same ones shown on /track-record, computed by the same method. Editorial opinion, not investment advice.

Timing, in the company's words (2 quotes from 1 filing)

Sentences quoted verbatim from the company's SEC filings, newest filing first. Each sits under the filing it appeared in; the "matched to" tag names the calendar row it was read for.

Timing sentences quoted from the company's own SEC filings, each under the filing it appeared in. The 'matched to' tag is the calendar row the sentence was read for; the filing is the source. Not investment advice.

Filings & releases (28)

SEC filings from EDGAR and press releases captured from the company's investor-relations site, newest first. Releases captured from Sep 2, 2026; filings from March 2026. Summaries come from the BD update that read the document; "BD extracted" counts point at the pipeline table above. All companies →

4 more filings (13G holder notices, S-8, proxy material, SEC correspondence …)