AMT-130
Gene Therapy Target: HTT, huntingtin, huntingtin gene
uniQure N.V.
Most recent annual filing: 2026-03-02
Pipeline (6 active programs)
AMT-260
Gene Therapy Target: GRIK2, GLUK2
AMT-191
Gene Therapy Target: GLA, alpha-galactosidase A
AMT-162
Gene Therapy Target: SOD1
HEMGENIX
etranacogene dezaparvovec · etranacogene dezaparvovec-drlb Gene Therapy Target: Factor IX
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 platform designed to silence disease-causing genes; used in AMT-130 for Huntington's disease
Targets: huntingtin gene
Biotech Distilled coverage (4)
- 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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