uniQure (QURE) — Stock, Pipeline & Market Cap

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QURE · Stock Price

USD 24.15+11.36 (+88.82%)

Market Cap: $1.5B

Historical price data

Market Cap: $1.5BPipeline: 4 drugsFounded: 1998HQ: Amsterdam, Netherlands

Sector Ranking#25 in Cell & Gene Therapy #20 in Neuroscience & CNS

Overview

uniQure's mission is to reimagine the future of medicine by delivering innovative, potentially curative gene therapies for severe genetic and neurodegenerative diseases. The company's strategic foundation is its industry-leading, modular AAV5 platform, which has demonstrated a validated safety profile and holds exclusive rights for therapeutic delivery to the brain and liver. Its most advanced program, AMT-130 for Huntington's disease, represents a potential first-in-class treatment, positioning uniQure at the forefront of CNS gene therapy with a pipeline targeting high-unmet-need indications.

Neurodegenerative DiseasesGenetic Disorders

Technology Platform

Proprietary, modular AAV5-based gene therapy platform with exclusive rights for delivery to the brain/CNS and liver, complemented by enabling cargo technologies (miQURE, LinQURE, GoQURE) for gene silencing and replacement.

Pipeline

4

4 drugs in pipeline

Drug	Indication	Stage
alipogene tiparvovec + Prednisolone + Cyclosporins + Mycophe...	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

4

Total raised:$298M

Debt$150MPerceptive AdvisorsOct 15, 2020

IPO$90MUndisclosedFeb 6, 2014

Series B$36MForbionJun 15, 2006

Series A$22MAbingworthJun 15, 2004

Opportunities

AMT-130 represents a multi-billion dollar opportunity as a potential first disease-modifying therapy for Huntington's disease.

The proprietary AAV5 platform and enabling cargo technologies create multiple shots on goal across CNS and liver disorders, with royalty revenue from HEMGENIX providing non-dilutive funding.

Risk Factors

High binary risk on lead program AMT-130 clinical data; substantial cash burn requiring future financing; intense competition in neurology gene therapy; and complex regulatory and commercialization pathways for first-in-class CNS therapies.

Competitive Landscape

Faces competition from ASO developers (e.g., Roche) in Huntington's disease and approved therapies (Biogen) in SOD1-ALS. Differentiation hinges on AAV5 exclusivity, one-time administration for durable effect, and a validated safety profile. Must also compete with other gene therapy firms in Fabry and epilepsy.