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.
Real-world clinical data platform for life science research
Arcturis is a real-world data and evidence company that aggregates longitudinal, secondary-care patient records from a network of NHS hospitals into research-ready,…
AI-driven isoform sequencing platform for movement disorder and ageing research
Entelo Bio is an Oxford-based biotechnology company developing a proprietary single-cell isoform sequencing and AI platform to decode disease biology in conditions affecting human…
Full-service CRO specialising in oncology and rare diseases
Simbec-Orion is a full-service CRO providing Phase I to Phase IV clinical trial services, with specialist expertise in oncology, rare and orphan diseases, and clinical…
Non-surgical drug therapy for spinal cord injury recovery
NeuroSolv Therapeutics is a preclinical-stage biotechnology company developing a non-surgical two-part therapy for spinal cord injury. The proposed mechanism combines enzymatic…
Stabilised siRNA therapeutics platform targeting cardiovascular gene silencing
Argonaute RNA is a biopharmaceutical company developing small interfering RNA therapeutics for cardiovascular disease. Its siRNA platform uses proprietary chemical stabilisation…
Implantable localised chemotherapy delivery for hard-to-treat solid tumours
CRISM Therapeutics is a clinical-stage drug delivery company developing an implantable biodegradable technology that delivers chemotherapy directly into tumours or surgical…
Global population genomics platform for drug target discovery and clinical trials
Omnigen Biodata is a biotechnology company operating the Discover Me platform, a global population health and genomics research programme integrating health and genetic data from…
Rare disease partnering platform connecting patient groups with drug developers
Aspire Biosciences is a Cambridge-based company accelerating treatment development for rare and unaddressed diseases through its Systematic Serendipity methodology. The company…
Immunotherapies targeting blood cancer precursor conditions before onset
Athernal Bio is a Cambridge-based biotechnology company developing targeted immunotherapies for high-risk clonal haematopoiesis, a widespread blood stem cell mutation condition…
Specialty pharma delivering niche medicines and novel drug delivery technology
Therakind is a specialty pharmaceutical company focused on developing niche medicines for difficult-to-treat patient populations, including children and the elderly. The company…
Mitochondrial transplantation platform to restore cellular energy and regeneration
Recondria Therapeutics is a developer of a mitochondrial transplantation platform designed to restore cellular energy, accelerate tissue recovery and support regenerative…
Medical research charity translating science into treatments and diagnostics
LifeArc is a self-funded UK medical research charity focused on translating early-stage science into treatments and diagnostics for patients with rare diseases and global health…