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.
Convection enhanced delivery consulting and devices for CNS therapeutics
Neurochase is a contract research and consulting organisation specialising in targeted drug delivery to the CNS using convection enhanced delivery. Working with pharmaceutical and…
Takeda's UK clinical development and drug discovery operations
Takeda UK encompasses the British operations of Takeda Pharmaceutical Company, one of the world's largest biopharmaceutical companies. The London-based Takeda Development Centre…
Targeting developmentally-silent oncogenic drivers for cancer therapy
Awen Oncology is a Welsh oncology biotechnology company developing first-in-class therapeutics that target cancer-specific proteins derived from developmental genes that are…
Full-service CRO and pharmacovigilance services for biopharma
Ergomed is a global contract research organisation providing clinical development, pharmacovigilance, and regulatory compliance services to the pharmaceutical and biotechnology…
Tissue-targeted gene and RNA delivery platforms for rare and neurological disease
Dawn Therapeutics is a pre-clinical biotechnology company developing precision delivery platforms for genetic medicines targeting the central nervous system, cartilage, and bone.…
Exosome-delivered gene editing therapies for neurodegenerative diseases
Evox Therapeutics is a biotechnology company developing exosome-based gene editing therapies for rare, genetically driven neurodegenerative diseases. Its proprietary platform uses…
European specialty pharma focused on gastroenterology and hepatology
Norgine is a European specialty pharmaceutical company developing, manufacturing, and commercialising medicines across gastroenterology, hepatology, and cancer supportive care.…
Inhaled therapies for COPD and chronic respiratory diseases
Verona Pharma is a biopharmaceutical company developing and commercialising inhaled therapies for chronic respiratory diseases. Its lead product, ensifentrine, is a first-in-class…
High-throughput iPSC reprogramming and stem cell drug discovery services
Oxford StemTech is a contract research company providing induced pluripotent stem cell (iPSC) reprogramming and drug discovery services using its proprietary platform. The…
AAV gene therapies for rare neurological diseases of childhood
BlackfinBio is a biotechnology company developing AAV-based gene therapies for rare neurodegenerative diseases of the central nervous system, with an initial focus on children.…
Gene therapy platform targeting paediatric solid tumours
Kindling Bio is a biotechnology company developing a gene therapy platform designed specifically for childhood cancers. The platform engineers a biological signal that renders…
Rare and serious disease therapeutics with Cambridge R&D hub
Insmed is a US biopharmaceutical company with commercial-stage and late-pipeline products spanning respiratory, inflammatory and rare neurological diseases. Its Cambridge facility…