AMT-130
Gene Therapy Target: HTT, huntingtin, huntingtin gene
uniQure
Most recent annual filing: 2026-03-02
- HEMGENIX corporate updateMid-2026HEMGENIX corporate update, Mid-2026, routine
- AMT-130 BLA filing3Q 2026AMT-130 BLA filing, 3Q 2026, major
- Scocco litigation filingSep 14, 2026Scocco litigation filing, Sep 14, 2026, routine
- Hercules term loan tranche filingSep 30, 2027Hercules term loan tranche filing, 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)
AMT-191
Gene Therapy Target: GLA, alpha-galactosidase A
HEMGENIX
etranacogene dezaparvovec · etranacogene dezaparvovec-drlb Gene Therapy Target: Factor IX
AMT-260
Gene Therapy Target: GRIK2, GLUK2
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)
- AMT-162 — Amyotrophic Lateral Sclerosis · Discontinued following dose limiting toxicity and SAE observed in second cohort; IDMC recommended voluntary pause of enrollment
Biotech Distilled coverage (8)
- 2026-07-30 UPDATE: uniQure (QURE) - The Fine Print in the Minutes 🔒 Paid
- 2026-06-24 UPDATE: uniQure (QURE) - The Raise Got Bigger, and the Tape Said Yes 🔒 Paid
- 2026-06-22 UPDATE: uniQure (QURE) - The Raise and the Half-Signal 🔒 Paid
- 2026-06-17 UPDATE: uniQure (QURE) - The FDA Folds
- 2026-05-06 UPDATE: uniQure (QURE) - The Pipeline Gets Smaller, the Lawsuit Gets Real 🔒 Paid
- 2026-05-01 UPDATE: uniQure (QURE) - The MHRA End-Around 🔒 Paid
- 2026-03-08 UPDATE: UniQure (QURE) - The FDA Takes the Gloves Off and Algorithms Go Rogue
- 2026-02-26 Uniqure (QURE): Scientific Deep Dive for AMT-130 and Pipeline Products 🔒 Paid
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