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.
Plasma fractionation and manufacture of plasma-derived therapies
Kedrion Biopharma is a biopharmaceutical manufacturer specialising in the fractionation of human blood plasma to produce albumin solutions, immunoglobulins and coagulation factors…
Native mass spectrometry platform for small molecule drug discovery
OMass Therapeutics is a biotechnology company developing small molecule therapeutics against membrane proteins, GPCRs, and intracellular complexes that have historically been…
Clinical-stage biopharma developing treatments for cancer and rare diseases
Biodexa Pharmaceuticals is a Cardiff-based clinical-stage biopharmaceutical company advancing a pipeline of proprietary drug formulations for diseases with high unmet need. Its…
Small activating RNA therapeutics for gene upregulation and disease
MiNA Therapeutics is a clinical-stage biotechnology company pioneering small activating RNA medicines, a class of oligonucleotide therapeutics that restore or boost expression of…
Synthetic glycan-targeted small molecules for next-generation immunotherapy
Anomer Bio is a biotechnology company developing synthetic monosaccharides and small molecule drugs that target glycans, the sugars coating cell surfaces that influence immune…
Small molecule therapeutics targeting PDE4 enzymes in kidney disease
Mironid is a company developing first-in-class small molecule therapeutics for autosomal dominant polycystic kidney disease, one of the most common hereditary kidney disorders.…
Specialist AAV vector manufacturer for gene therapy developers
Trisk Bio is a contract development and manufacturing organisation specialising in the production of adeno-associated virus vectors for gene therapy applications. Its intensified…
Gene therapy company targeting inherited and degenerative diseases
MeiraGTx is a vertically integrated, clinical-stage genetic medicines company developing AAV-based gene therapies for inherited retinal diseases, neurodegenerative conditions, and…
Regenerative cell therapy for sensorineural hearing loss
Rinri Therapeutics is a clinical-stage biotech developing allogeneic regenerative cell therapies for sensorineural hearing loss, a condition affecting over 400 million people…
Small molecule drugs targeting astrocytes in neurodegenerative disease
AstronauTx is a clinical-stage UK biotech developing oral small molecule therapeutics for Alzheimer's disease and other neurodegenerative disorders. The company's approach targets…
Full-service CRO for Phase I–IV pharmaceutical and device trials
Clinicology is a contract research organisation providing clinical trial management services to pharmaceutical, biotechnology, and medical device companies. Services span site…
E3 ligase inhibitors stabilising tumour suppressor proteins
Outrun Therapeutics is a preclinical drug discovery company developing small molecule inhibitors of E3 ligase enzymes to stabilise tumour suppressor and other disease-relevant…