Rare Disease Companies in the UK

Rare disease companies develop diagnostics, therapies and research technologies for conditions affecting relatively small patient populations. Their work often focuses on inherited disorders, complex diagnostic pathways and diseases with limited or no existing treatment options.

This category includes companies working on:

Orphan drugs and rare disease therapeutics
Genetic testing and molecular diagnostics
Gene, RNA and cell therapies
Biomarkers and natural history studies
Patient identification and clinical development

Browse 141 UK rare disease companies below.

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Rare Diseases

Showing 141–141 of 141

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Milner Therapeutics Institute

CambridgeEast of England

Academic-industry translational research institute

The Milner Therapeutics Institute is a University of Cambridge biomedical research institute established to bridge academic science and pharmaceutical drug discovery. Founded in 2015 and based at the Jeffrey Cheah Biomedical Centre on the Cambridge Biomedical Campus, it operates both an in-house research programme and…

Research OrganisationDrug Discovery

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