UniQure

What UniQure does

uniQure N.V. (NASDAQ: QURE) is a clinical-stage gene therapy company founded in 1998 and headquartered in Amsterdam, the Netherlands, with US operations centered on a commercially licensed cGMP manufacturing facility in Lexington, MA, and a Swiss office in Basel. The company develops adeno-associated virus (AAV)-based therapies, anchored on its AAV5 vector platform with exclusive worldwide rights for delivery to brain and liver, augmented by proprietary enabling technologies including the miQURE gene-silencing platform, LinQURE multi-microRNA delivery, GoQURE knock-down-plus-replace constructs, and Smart AAV capsids engineered for CNS and liver targeting.

The company's only approved product is HEMGENIX (etranacogene dezaparvovec), the world's first gene therapy for hemophilia B, which received FDA approval in November 2022 and EU conditional marketing authorization in February 2023, and which was awarded the Prix Galien USA Award for Best Product for Rare/Orphan Diseases in 2023. The pipeline is focused on central nervous system and metabolic rare diseases, with AMT-130 in Huntington's disease (positive Phase I/II pivotal data showing 75% slowing of disease progression at 36 months), AMT-260 in refractory mesial temporal lobe epilepsy (positive interim seizure-reduction data), AMT-191 in Fabry disease, and AMT-162 in SOD1-ALS (discontinued in 2025).

uniQure operates a hybrid commercial model. HEMGENIX is out-licensed globally to CSL Behring for commercialization under a 2021 agreement that delivered $500 million in upfront and milestone payments plus up to $400 million in monetized royalty rights, while uniQure retains global manufacturing responsibility at its Lexington facility. For AMT-130, the company is building its own commercial infrastructure for an anticipated 2026 US launch following a planned Q3 2026 BLA submission under an FDA-confirmed accelerated pathway. Revenue is currently modest (Q1 2026: $3.56 million), reflecting a pre-profitability profile supported by more than $800 million of equity and non-dilutive capital raised across 2025–2026 to fund pipeline and commercial readiness.

Name

UniQure

Legal name

uniQure N.V.

Website

https://uniqure.com

Company type

Public

Founded year

1998

Operating status

Operating

Headcount range

101–250 employees

Short description

uniQure is a publicly traded (NASDAQ: QURE) gene therapy company developing AAV5-based treatments for rare genetic and CNS diseases, with one approved product (HEMGENIX for hemophilia B, commercialized via CSL Behring) and a pipeline led by AMT-130 for Huntington's disease, targeting a planned 2026 US launch.

Ownership category

akta.pro rank

Product category

Gene Therapy Pharmaceuticals

NAICS

Pharmaceutical and Medicine Manufacturing (3254)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

Keywords

• Gene therapy
• AAV vector delivery
• Rare disease therapeutics
• Gene silencing platform
• Hemophilia treatment

Headquarters

HQ city

Amsterdam

HQ country

Netherlands

HQ region

Europe

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Licensing Royalties from HEMGENIX: UniQure receives royalties from CSL Behring on global net sales of HEMGENIX (etranacogene dezaparvovec), the approved gene therapy for hemophilia B. The company sold a portion of royalty rights to HealthCare Royalty (HCRx) and Sagard Healthcare for up to $400 million in cash.
2. Milestone Payments: UniQure is eligible to receive additional milestone payments from CSL Behring, including $25 million if 2024 net sales of HEMGENIX exceed pre-specified thresholds.
3. Manufacturing Revenue: UniQure is responsible for global manufacturing of HEMGENIX at its licensed Lexington, MA facility, providing manufacturing revenue from the CSL Behring partnership.

Go-to-market motion2 records

Distribution channels3 records

Marketing channels5 records

Core offering

UniQure develops, manufactures, and commercializes AAV5-based gene therapies for patients with rare, severe genetic and neurological diseases. Its approved product HEMGENIX is a one-time gene therapy for hemophilia B, commercialized globally by CSL Behring under an exclusive licensing agreement. The company's pipeline includes investigational gene therapies AMT-130 (Huntington's disease), AMT-260 (refractory temporal lobe epilepsy), and AMT-191 (Fabry disease). UniQure retains manufacturing responsibility at its commercially licensed cGMP facility in Lexington, MA, and receives royalties, milestone payments, and manufacturing revenue from its approved product.

Differentiator

Problem solved

Functional benefit

Brands

• HEMGENIX: World's first approved gene therapy for hemophilia B (etranacogene dezaparvovec)
• AMT-130
• AMT-260
• AMT-191
• AMT-162
• miQURE

Products and services

• HEMGENIX (etranacogene dezaparvovec) One-time administered AAV5-based gene therapy delivering a factor IX Padua transgene to enable hemophilia B patients to produce their own factor IX. Commercialized globally via exclusive licensing partnership with CSL Behring; UniQure retains manufacturing responsibility at its Lexington, MA facility.
• AMT-130 Investigational AAV5 gene therapy for Huntington's disease using uniQure's proprietary miQURE gene silencing platform to deliver microRNA that reduces production of the mutant huntingtin protein. Demonstrated 75% slowing of disease progression in high-dose cohort at 36 months. UniQure plans BLA submission in Q3 2026.
• AMT-260 Investigational AAV9 gene therapy for refractory mesial temporal lobe epilepsy (MTLE) targeting the GRIK2 gene to reduce seizure frequency. Demonstrated 79-100% seizure reduction in 3 of 6 low-dose patients during months 4-6.
• AMT-191 Investigational AAV5 gene therapy for Fabry disease delivering a GLA transgene targeting the liver to enable production of GLA protein. All 11 dosed patients in the study discontinued enzyme replacement therapy.

Quantifiable outcome

• 75% slowing of disease progression in high-dose AMT-130 patients at 36 months (p=0.003)
• +3 more outcomes

Named customers1 record

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

Four partnerships are on record, tiered core, strategic and minor.

• CSL BehringcoreChannel Partner/ Reseller/ Distributor · 1 May 2021CSL Behring acquired exclusive global license rights to commercialize HEMGENIX (etranacogene dezaparvovec), uniQure's gene therapy for hemophilia B. UniQure received $500 million in upfront payments and is eligible for up to $400 million in royalty monetization and $25 million in milestone payments. UniQure retains responsibility for global manufacturing at its Lexington, MA facility.
• 4D Molecular TherapeuticsstrategicTechnology or IntegrationCollaboration and license agreement focused on identification of novel optimized AAV variants with greatly enhanced properties for higher levels of gene expression. The collaboration aims to evolve AAV variants with improved gene delivery properties in liver and central nervous system.
• Academic and Research InstitutionsminorStrategic or Co-development PartnerUniQure collaborates with leading academic institutions and research organizations that are sponsoring preclinical and early-stage clinical trials of gene therapy product candidates to which the company holds specified rights.
• Corlieve Therapeutics SASstrategicStrategic or Co-development PartnerUniQure completed acquisition of Corlieve Therapeutics SAS in July 2021. Corlieve's gene therapy programs for epilepsy were integrated into uniQure's pipeline, including AMT-260 for refractory mesial temporal lobe epilepsy.

Scale indicators9 records

Recent moves10 records

Expansion highlights6 records

Direct peers

• BioMarin Pharmaceutical: BioMarin is a direct peer in rare disease gene therapy, most notably having received FDA approval for Roctavian, the first AAV gene therapy for hemophilia A. Both companies operate commercial-scale AAV gene therapy manufacturing and target similar one-time treatment pricing models in rare hematologic and metabolic diseases.
• Sarepta Therapeutics: Sarepta is a direct AAV gene therapy competitor focused on Duchenne muscular dystrophy (DMD) with Elevidys. Like uniQure, Sarepta operates in the rare disease AAV gene therapy space, uses similar regulatory pathways (RMAT, accelerated approval), and faces analogous pricing/reimbursement dynamics.
• Regenxbio: Regenxbio is a direct peer with a proprietary AAV gene therapy platform that licenses to multiple partners (including AbbVie, Novartis, and Roche). Both companies develop and enable AAV-based gene therapies for rare diseases, with similar technology platforms and partnership-driven business models.
• Spark Therapeutics (Roche): Spark, now part of Roche, developed and commercializes Luxturna, the first FDA-approved AAV gene therapy (for an inherited retinal disease). Both companies pioneered AAV gene therapy approvals and operate in rare inherited disease markets with similar regulatory and reimbursement challenges.
• Krystal Biotech: Krystal Biotech is a direct peer in rare disease gene therapy, having received FDA approval for Vyjuvek, a topical gene therapy for dystrophic epidermolysis bullosa. Both companies are commercial-stage gene therapy developers focused on rare genetic diseases with high unmet need and premium pricing strategies.

Emerging players

• Voyager Therapeutics: Voyager is an emerging player developing AAV-based gene therapies with a focus on CNS diseases, similar to uniQure's AMT-130 for Huntington's and AMT-260 for TLE. Both companies target neurodegenerative and neurological rare diseases using AAV vectors, though Voyager is at an earlier clinical stage.
• Taysha Gene Therapies: Taysha is an emerging player in AAV gene therapy focused on CNS and rare monogenic diseases. The company's pipeline intersects with uniQure's in CNS-targeted AAV approaches for rare neurological disorders, though Taysha operates at earlier development stages.
• LEXEO Therapeutics: LEXEO is an emerging clinical-stage gene therapy company developing AAV-based therapies for CNS and cardiac rare diseases. The company overlaps with uniQure in CNS-targeted AAV approaches and shares the rare disease gene therapy development model.

Broad incumbents

• Pfizer: Pfizer is a broad incumbent in the gene therapy space following its acquisition of Global Blood Therapeutics and gene therapy programs (and via its historical hemophilia B presence). As a major pharmaceutical company with AAV capabilities through Spark partnerships and hemophilia franchise exposure, Pfizer is a comparable incumbent operating in overlapping markets.
• CSL Behring: CSL Behring is both uniQure's commercialization partner for HEMGENIX and a broad incumbent in the rare disease/hematology space. As the global commercializer of HEMGENIX, CSL represents the established pharma-scale rare disease commercial infrastructure that uniQure relies on, and operates competing plasma-derived and recombinant therapies in adjacent indications.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/uniqure
• Xx.com/uniQure_NV

Revenue estimate

Valuation estimate

Number of profiles

Profiles15 records

Subsidiaries1 record

Funding overview

Funding rounds10 records

Investors4 records

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M&A2 records

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about UniQure