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.
CSF-1R antibody therapy for tenosynovial giant cell tumour
SynOx Therapeutics is a late-stage biopharmaceutical company developing emactuzumab, a humanised monoclonal antibody targeting CSF-1R, for the treatment of tenosynovial giant cell…
RNA-based therapeutics targeting neurodegenerative disease mechanisms
Harness Therapeutics is a Cambridge-based biotech developing oligonucleotide therapeutics that exploit post-transcriptional regulation to achieve controlled upregulation of…
DUB inhibitor therapeutics targeting mitophagy and degenerative disease
Mission Therapeutics is a clinical-stage biotechnology company developing first-in-class small molecule inhibitors of deubiquitylating enzymes to treat diseases driven by…
Non-viral gene therapy platform enabling durable nuclear DNA delivery
Gramics Bio develops a gene therapy platform solving a key limitation of lipid nanoparticle delivery: enabling therapeutic DNA to reach the cell nucleus for durable expression…
Specialist pharma acquiring and developing rare disease therapeutics
Ambrose Healthcare is a UK specialist pharmaceutical company developing treatments for rare diseases and patients in hospital and specialist care settings. The company builds its…
Oral small molecule therapies for rare hereditary diseases
KalVista Pharmaceuticals is a biopharmaceutical company specialising in the discovery and commercialisation of oral small molecule protease inhibitors for rare diseases with…
Global biopharma focused on severe neurological and immunological diseases
UCB Pharma is the UK subsidiary of UCB S.A., a Belgian biopharmaceutical company focused on the discovery and development of treatments for severe neurological and immunological…
Synthetic platform for precision delivery of biologics in tissue repair
Renovos Biologics is a biotech company developing precision delivery technologies for regenerative medicine, drawing on decades of academic research in skeletal stem cells,…
ADC pipeline targeting mutant stem cells in blood cancers
Alethio Therapeutics Limited is a preclinical-stage biotechnology company developing antibody-drug conjugates for myeloproliferative neoplasms, a group of chronic blood cancers.…
Global commercialisation of specialty, hospital and rare disease medicines
ADVANZ PHARMA is a global pharmaceutical company focused on the commercialisation of specialty, hospital and rare disease medicines, partnering with biopharma and pharmaceutical…
Targeted antibody therapeutics for cancer and autoimmune disease
Medannex is a clinical-stage biopharmaceutical company developing monoclonal antibody therapeutics targeting annexin-A1, a protein implicated in cancer progression, autoimmunity,…
Biologic therapies for autoinflammatory and autoimmune diseases
Kiniksa Pharmaceuticals International is a commercial-stage biopharmaceutical company focused on acquiring, developing and commercialising medicines for autoinflammatory and…