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:
Browse 128 UK rare disease companies below.
Precision calcium channel medicines for epilepsy and Parkinson's disease
Lario Therapeutics is developing first-in-class precision medicines as disease-modifying treatments for severe neurological disorders, including genetically defined epileptic…
Inhaled antifungal therapies for serious respiratory fungal infections
Pulmocide is a late-stage biopharmaceutical company developing inhaled medicines for life-threatening respiratory fungal infections. Its lead compound opelconazole is a triazole…
Global diabetes and cardiometabolic medicines leader
Novo Nordisk Limited is the UK commercial and medical affiliate of Novo Nordisk A/S, the Danish pharmaceutical company focused on diabetes, obesity, and cardiometabolic disease.…
Global biopharma focused on neurology and rare disease therapeutics
Biogen is a global biopharma company and one of the world's first biotechnology firms. The UK and Ireland headquarters in Maidenhead employs approximately 250 people across…
GPCR-targeted small molecule drugs for rare and inflammatory disease
Trio Medicines is a small pharmaceutical company developing small molecule therapeutics targeting G-protein coupled receptors for diseases with unmet clinical need. Its pipeline…
AAV gene therapy platform and clinical pipeline for genetic disease
AskBio UK Ltd is the European headquarters of AskBio Inc., a fully integrated AAV gene therapy company and Bayer AG subsidiary. Operating from Roslin Innovation Centre, the…
Antibody therapies targeting severe autoimmune diseases
argenx UK Ltd is the UK commercial subsidiary of argenx SE, a global immunology company developing antibody-based therapies for severe autoimmune diseases. The company markets…
Peptide immunotherapies for the treatment of food allergy
Aravax is a clinical-stage biotechnology company developing next-generation specific immunotherapies for food allergy. Its lead candidate, PVX108, is a synthetic peptide-based…
Clinical-stage oncology company advancing in-licensed cancer drug assets
Ellipses Pharma is a clinical-stage oncology drug development company that in-licenses cancer drug assets from academia and industry, advances them through clinical trials using a…
Precision medicine platform for patient recruitment and genetic testing
Sano Genetics is a London-based software company providing an end-to-end platform for patient recruitment, at-home genetic testing, and long-term participant engagement in…
Targeting mitochondrial dysfunction in neurodegeneration
NRG Therapeutics is a clinical-stage neuroscience company developing small molecule inhibitors of the mitochondrial permeability transition pore as disease-modifying treatments…
High-capacity viral vector manufacturing platform for gene therapy
Icosphere Biosciences is a biotechnology company developing manufacturing technology for high-capacity gene therapy vectors. Its platform enables scalable, high-yield,…