uniQure N.V.

uniQure N.V. is a gene therapy company focused on developing and commercializing treatments for severe genetic and other diseases. Its business centers on adeno-associated virus (AAV) gene therapy programs, including clinical-stage candidates and a marketed therapy, with operations spanning the United States and Europe.

−1 059,7 %

89,5 %

−1 236,0 %

−40,6 %

10.43

10.43

— uniQure N.V.
%
Clinical-stage gene therapy programs70% Research, development, and clinical testing of AAV-based therapies for rare and severe diseases.
Commercial royalty and license revenue20% Royalty and license income tied to HEMGENIX® sales under collaboration arrangements.
Collaboration and development services10% Services performed for partners under development and supply agreements.

uniQure’s direct customers are primarily pharmaceutical partners and collaborators, while the eventual end users are...

  • Pharmaceutical collaboration partnersprimary

    Partners like CSL Behring that buy development, supply, or royalty-linked rights under collaboration agreements.

  • Healthcare providers and physiciansprimary

    Specialists who recommend and administer gene therapies once products are approved.

  • Third-party payersprimary

    Insurers and reimbursement bodies that influence access and commercial uptake of approved therapies.

  • Patients with rare genetic diseasesprimary

    Patients targeted by the company’s gene therapy pipeline, including Huntington’s disease and other rare disorders.

  • Clinical and manufacturing service providerssecondary

    CROs, CMOs, and other vendors that conduct trials, manufacture materials, and support development.

uniQure operates across the United States and Europe, where it runs clinical development and supports partner-related...

  • •Operations and clinical trials span the United States and Europe
  • •U.S. regulatory and reimbursement systems are central to commercialization
  • •European operations create euro-denominated cost and cash exposure
  • •Cross-border clinical work increases regulatory and logistics complexity
  • •Foreign exchange movements affect reported results and liquidity

uniQure’s strategy is centered on advancing its lead gene therapy programs through clinical development and regulatory...

01
Advance AMT-130 for Huntington’s diseaseshort-term

The lead program is central to the company’s clinical and commercial value creation.

02
Expand and progress the gene therapy pipelinemedium-term

A broader pipeline reduces dependence on a single asset and supports long-term optionality.

03
Monetize partnered assets and royalty streamsmedium-term

Partnered programs can generate non-dilutive revenue and validate the platform.

04
Manage external execution riskshort-term

Clinical and manufacturing outcomes depend heavily on third-party service providers.

uniQure is highly dependent on the clinical success of AMT-130, so setbacks in efficacy, safety, manufacturing, or...

critical

Dependence on AMT-130 clinical success

The lead program is a major driver of future value, so failure would materially impair prospects.

Scope
Huntington’s disease program
Materiality
high
high

Third-party execution and data quality risk

CROs and other vendors conduct key studies and supply activities outside direct employee control.

Scope
Clinical trials and development programs
Materiality
high
high

Product liability and patient safety claims

Clinical and commercial gene therapies can trigger claims related to adverse events or administration procedures.

Scope
Clinical trials and approved products
Materiality
high
medium

Healthcare compliance and anti-bribery risk

Commercialization involves physicians, payers, and third parties subject to U.S. and international healthcare laws.

Scope
Sales, distribution, and payer arrangements
Materiality
medium
medium

Foreign exchange volatility

The company holds and transacts in euros and U.S. dollars, creating translation and remeasurement effects.

Scope
Cross-border cash, borrowings, and intercompany balances
Materiality
medium
License and collaboration revenue recognition
Can cause uneven quarterly revenue and comparability issues
Contract manufacturing accounting
Affects historical revenue mix and gross margin presentation
Fair value of pre-funded warrants
Can materially affect reported loss in a period
Foreign currency remeasurement
Creates gains or losses unrelated to core operating performance
Contingent consideration
Valuation changes can affect liabilities and earnings

Humacyte, Inc.

HUMA · Biological Products, (No Diagnostic Substances)

MeiraGTx Holdings plc

MGTX · Biological Products, (No Diagnostic Substances)

OnKure Therapeutics, Inc.

OKUR · Pharmaceutical Preparations

REGENXBIO Inc.

RGNX · Biological Products, (No Diagnostic Substances)

Unicycive Therapeutics, Inc.

UNCY · Pharmaceutical Preparations

Immunocore Holdings plc

IMCR · Biological Products, (No Diagnostic Substances)

: 29/04/2026