Company intelligence / Profile preview
uniQure
Amsterdam, Netherlandsuniqure.com ↗
- Ownership
- Public · QURE
- Employees
- ~500
- Development stage
- Commercial
01
Overview
uniQure has achieved the world’s first approved gene therapy for hemophilia B. Its lead program in Huntington's disease has completed enrollment in multiple cohorts of a Phase I/II study and aligned with the FDA on an Accelerated Approval pathway. Other programs are advancing through early clinical development with positive interim safety data reported across several indications[7]. The company maintains strong cash reserves to fund operations into late 2027.
- Therapeutic areas
- HematologyRare DiseasesNeurology
- Modalities
- Adeno-associated virus (AAV)-based gene therapiesgene silencing via miQURE™ technology
- Industry
- Biotech
02
Drug pipeline
20 assets19 more assets in the full pipeline.
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Partnerships
CSL Behring — commercialization partnership for hemophilia B program [4].Academic collaborations on drug discovery/preclinical research [3].
04
Financials
- Market cap
- $3.2B
- Total revenue
- $5.8M
- Enterprise value
- $2.4B
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