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.
Multiplexed LC-MS proteomics for biomarker development and clinical trials
Inoviv is a bioanalytical contract research company providing multiplexed, quantitative LC-MS targeted proteomics services to pharmaceutical and biotech clients. Its platform…
Small molecule therapies for keratin-related skin disorders
Mariposa Therapeutics is a Welsh preclinical biopharma company developing disease-modifying treatments for epidermolysis bullosa simplex and other keratin-related skin disorders.…
Full-service clinical and commercial solutions across the drug development lifecycle
Syneos Health is a global biopharmaceutical solutions organisation providing integrated clinical and commercial services to pharmaceutical, biotechnology and medical device…
Global biopharma with UK R&D hub in Hertfordshire
Roche Products is the UK pharmaceutical arm of F. Hoffmann-La Roche, headquartered at Welwyn Garden City in Hertfordshire. The site employs over 1,900 people and serves as a…
Rare disease clinical trial specialist focused on patient-centred design
Actigen is a contract research organisation specialising in the design and delivery of clinical trials for rare and orphan diseases. Working in partnership with therapy developers…
Circadian hormone therapeutics for rare endocrine conditions
Neurocrine UK is the European operations of Neurocrine Biosciences, a Cardiff-headquartered specialty pharmaceutical company developing and commercialising hormone therapeutics…
AAV gene therapies for rare and neurological diseases
Spur Therapeutics is a clinical-stage biotechnology company developing adeno-associated virus-based gene therapies for serious chronic diseases. Its lead programme is a Phase I/II…
Ex vivo haematopoietic stem cell gene therapies for rare genetic diseases
Orchard Therapeutics is a gene therapy company developing ex vivo haematopoietic stem cell therapies for rare genetic diseases, primarily severe neurometabolic and immunological…
AAV gene therapies for neurodegenerative diseases including FTD and ALS
AviadoBio is a clinical-stage gene therapy company developing AAV-based treatments for neurodegenerative diseases. Its lead programme, AVB-101, is in a Phase 1/2 trial for…
Nanoparticle platform for targeted genetic medicine delivery
ViaNautis is a nanomedicine company developing a polymer nanoparticle platform for the targeted intracellular delivery of genetic therapeutic payloads across…
DNA-based gene therapy platform for intercellular therapeutic gene delivery
Bitrobius Genetics is a UK biotech developing a novel DNA-based gene therapy platform technology designed to deliver functional copies of mutated genes to cells within a target…
Integrated academic drug discovery facility for anti-infectives and innovative targets
The Drug Discovery Unit is an academic drug discovery facility at the University of Dundee, integrating medicinal chemistry, high-throughput screening, structural biology, cell…