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uniQure

QURE Rare disease Lead: Approved 11 Biotech Distilled articles ↓ 32 filings & releases ↓ Cash $98.4M (Sep 30, 2026) ↓ 11 verdicts ↓ 4 timing quotes ↓
HematologyMetabolicNeurology Gene Therapy

Market cap $1.52B 70.1M shares (10-Q, Nov 2, 2026) × $21.70 close Nov 6, 2026

Most recent annual filing: 2026-03-02

Catalyst timeline

3 trackedQuarter catalyst calendar →
  1. AMT-191 Fabry readout1H 2027AMT-191 Fabry readout, 1H 2027, notable
  2. AMT-130 ifezuntirgene inilparvovec PDUFA dateMay 1, 2027AMT-130 ifezuntirgene inilparvovec PDUFA date, May 1, 2027, thesis-defining
  3. Hercules term loan tranche corporateSep 30, 2027Hercules term loan tranche corporate, Sep 30, 2027, routine

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 (4 active programs)

Technology platforms

miQURE · Cold Spring Harbor Laboratory

Gene silencing platform based on exclusively licensed technology from Cold Spring Harbor Laboratory; delivers miRNA constructs via AAV to silence disease-causing genes without passenger strand off-target toxicity; secondary exosome-mediated delivery for whole-organ silencing; used in AMT-130 and other CNS programs

LinQURE

Single AAV vector carrying multiple miRNAs designed to suppress different disease genes; extension of the miQURE gene silencing strategy for CNS and other diseases

GoQURE

AAV-based platform for simultaneous silencing of a disease gene and replacement with a healthy gene; designed for CNS and other diseases with dual gene therapy needs

miQURE

Proprietary gene-silencing miRNA-based platform used to silence disease-causing genes; applied to Huntington's disease via AMT-130, incorporating a miRNA designed to silence the huntingtin gene and the toxic exon 1 protein fragment.

Targets: huntingtin gene, huntingtin

Discontinued (1)

Capital (as filed)

Cash
$98.4M cash and cash equivalents · Sep 30, 2026 (10-Q)
Operating cash flow
−$47.8M quarter Jun 30, 2026 → Sep 30, 2026, year-to-date difference (10-Q)
As stated in coverage
$768M cash, runway 2030 per the update of Nov 2, 2026 — the company's own words as read there, not the filed figure

Offering documents (1)

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 (11)

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

Verdict history (11 calls)

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

  1. 2026-02-26 Watch Uniqure (QURE): Scientific Deep Dive for AMT-130 and Pipeline Products Management narrative is decoupled from regulatory reality; the FDA rejected the 36-month AMT-130 data package as insufficient for a BLA, and the pipeline has multiple toxicity pauses, so the author recommends waiting for the FDA Type A meeting minutes before taking a position.
  2. 2026-03-08 Avoid UPDATE: UniQure (QURE) - The FDA Takes the Gloves Off and Algorithms Go Rogue The FDA formally rejected the AMT-130 data package and an anonymous senior FDA official publicly called the product a 'failed product'; the post-crash short squeeze to $21.55 is mechanically driven and not fundamental, making it a gift to exit any position.
  3. 2026-05-01 Hold UPDATE: uniQure (QURE) - The MHRA End-Around The MHRA pre-submission meeting opens a credible non-FDA path to first revenue and a 4-year dataset is coming, loosening but not closing the narrative-vs-reality trap; holders should consider trimming into strength while new money waits for the Q2 FDA Type B readout and Q3 4-year data.
  4. 2026-05-06 Hold UPDATE: uniQure (QURE) - The Pipeline Gets Smaller, the Lawsuit Gets Real AMT-162 is officially discontinued and a securities class action has surfaced, narrowing the pipeline and adding a new liability, but the MHRA optionality and AMT-130 lead asset thesis remain intact; holders should consider trimming the portion sized for broad AAV platform optionality while new money waits for Q2 catalysts.
  5. 2026-06-17 Speculative buy speculative UPDATE: uniQure (QURE) - The FDA Folds The FDA accepted the 36-month AMT-130 data package as the primary basis for an accelerated approval BLA, sham surgery is off the table, and a BLA submission is planned for Q3 2026, inverting the prior regulatory-standoff thesis; but the +127.6% move has already priced much of this, so new money should size for the Q3 four-year data readout rather than chase the announcement.
  6. 2026-06-22 Watch UPDATE: uniQure (QURE) - The Raise and the Half-Signal A $150M follow-on offering (5.4% dilution) funds the confirmatory study and launch readiness from a position of strength, but the AMT-260 first-cohort data was a split decision (3 of 6 responded) and the easy money on the regulatory pivot has been made; the four-year cUHDRS readout in Q3 remains the gate that matters most.
  7. 2026-06-24 Watch UPDATE: uniQure (QURE) - The Raise Got Bigger, and the Tape Said Yes The offering upsized to $258.7M with the greenshoe exercised same-day, confirming deep institutional demand and removing the financing overhang ahead of the Q3 BLA and four-year data readout, but at $48.85 the asymmetry has narrowed and the four-year cUHDRS readout remains the binary that matters most.
  8. 2026-07-30 Watch UPDATE: uniQure (QURE) - The Fine Print in the Minutes The official FDA Type B minutes added a condition that the confirmatory study be 'well underway, potentially fully enrolled' at the time of accelerated approval, adding friction to the BLA timeline, but the financing risk is gone with $810.3M in cash and the four-year cUHDRS readout in September is now the whole thesis.
  9. 2026-09-02 Watch UPDATE: uniQure (QURE) - The BLA Is In The AMT-130 BLA was filed in Q3 as guided with a parallel UK MAA and priority review requested, clearing the confirmatory-design alignment gate the July minutes made uncertain, but the four-year cUHDRS readout due before end of September remains the gate that matters most and is genuinely two-sided on an n=12 efficacy comparison.
  10. 2026-09-29 Watch UPDATE: uniQure (QURE) - The Coin Landed on Its Edge The four-year cUHDRS primary endpoint missed significance (44% slowing, p=0.144), the rescue relies on a post hoc control swap and nominal p-values, and the external-control problem is back at the center of the BLA review; wait for the November FDA filing decision before re-engaging.
  11. 2026-11-03 Watch UPDATE: uniQure (QURE) - Accepted, Prioritized, and Headed for a Public Trial The FDA accepted the BLA with priority review and a May 1, 2027 PDUFA date, and the confirmatory design is built around TFC (the endpoint that held), but an Advisory Committee is tentatively planned where the missed 48-month primary and external-control problem will likely be aired publicly; wait for the AdCom date and briefing documents before entering.

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 (4 quotes from 4 filings)

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 (32)

SEC filings from EDGAR and press releases captured from the company's investor-relations site, newest first. Releases captured from Jun 17, 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 →

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