uniQure

QUREPhase 2

Amsterdam, NetherlandsFounded 1998uniqure.com

uniQure is a clinical-stage gene therapy company with over two decades of experience, dedicated to reimagining medicine through innovative genomic treatments. The company has built a modular AAV platform, featuring proprietary technologies like miQURE for gene silencing, and is advancing a pipeline targeting CNS and liver diseases. With a lead program, AMT-130, in late-stage development for Huntington's disease and a commercial-stage partnership for hemophilia B gene therapy, uniQure is positioned at the forefront of the gene therapy revolution.

Market Cap

$1.0B

Founded

1998

Focus

RNA & Gene Therapy

AI Company Overview

Technology Platform

A modular, industry-leading AAV gene therapy platform focused on liver and CNS delivery, featuring the AAV5 serotype and proprietary enabling technologies for gene silencing (miQURE) and targeted delivery.

Pipeline Snapshot

4 drugs in pipeline

Drug	Indication	Stage
alipogene tiparvovec + Prednisolone + Cyclosporins + Mycophenolate mofetil	LPL Deficiency	Phase 2
AMT-162	Amyotrophic Lateral Sclerosis	Phase 1/2
AMT-191	Fabry Disease	Phase 1/2
rAAV2/5-hNAGLU	Sanfilippo Syndrome B	Phase 1/2

Funding History

Total raised: $298M

Debt$150MPerceptive AdvisorsOct 15, 2020

IPO$90MUndisclosedFeb 6, 2014

Series B$36MForbionJun 15, 2006

Series A$22MAbingworthJun 15, 2004

Opportunities

The primary growth opportunity is the successful development and commercialization of AMT-130 as the first disease-modifying therapy for Huntington's disease.

Further opportunities lie in expanding the validated AAV5/miQURE platform into other large, underserved CNS indications like temporal lobe epilepsy, ALS, and Alzheimer's disease.

Risk Factors

Key risks include clinical trial failure of lead asset AMT-130, regulatory hurdles for novel CNS gene therapies, intense competition in the gene therapy space, and challenges in manufacturing and commercializing high-cost, one-time treatments.

Competitive Landscape

uniQure competes with large pharma and biotech firms in gene therapy, notably against Wave Life Sciences and Roche/IONIS in Huntington's disease. Its differentiation is based on two decades of AAV experience, the clinically validated AAV5 serotype with low pre-existing immunity, and its proprietary miQURE gene silencing technology.

Publications

Pipeline