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 141 UK rare disease companies below.
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 biology, and DMPK in a single purpose-built facility. The DDU conducts small molecule drug discovery programmes across two areas:…
Precision calcium channel medicines for epilepsy and Parkinson's disease
Lario Therapeutics is developing first-in-class precision medicines as disease-modifying treatments for severe neurological disorders, including genetically defined epileptic encephalopathies and Parkinson's disease. The company's pipeline centres on neuronal calcium channel inhibitors, beginning with genetically…
Inhaled antifungal therapies for serious respiratory fungal infections
Pulmocide is a late-stage biopharmaceutical company developing inhaled medicines for life-threatening respiratory fungal infections. Its lead compound opelconazole is a triazole antifungal designed for inhaled delivery to maximise pulmonary drug concentration while minimising systemic exposure and drug interactions.…
Global diabetes and cardiometabolic medicines leader
Novo Nordisk Limited is the UK commercial and medical affiliate of Novo Nordisk A/S, the Danish pharmaceutical company focused on diabetes, obesity, and cardiometabolic disease. The UK operation covers commercialisation, medical affairs, and clinical trial activities. Separately, Novo Nordisk Research Centre Oxford…
Global biopharma focused on neurology and rare disease therapeutics
Biogen is a global biopharma company and one of the world's first biotechnology firms. The UK and Ireland headquarters in Maidenhead employs approximately 250 people across clinical operations, regulatory affairs, drug safety, and medical affairs. The company develops and commercialises therapies for neurological,…
GPCR-targeted small molecule drugs for rare and inflammatory disease
Trio Medicines is a small pharmaceutical company developing small molecule therapeutics targeting G-protein coupled receptors for diseases with unmet clinical need. Its pipeline includes gastrin/CCK2 receptor antagonists for gastric neuroendocrine tumours, a selective β2-adrenoceptor antagonist, and a formyl peptide…
AAV gene therapy platform and clinical pipeline for genetic disease
AskBio UK Ltd is the European headquarters of AskBio Inc., a fully integrated AAV gene therapy company and Bayer AG subsidiary. Operating from Roslin Innovation Centre, the Edinburgh site develops the company's synthetic promoter technology, data-driven gene expression control sequences that complement AskBio's Pro10…
Antibody therapies targeting severe autoimmune diseases
argenx UK Ltd is the UK commercial subsidiary of argenx SE, a global immunology company developing antibody-based therapies for severe autoimmune diseases. The company markets VYVGART (efgartigimod), the first approved FcRn blocker in the UK, indicated for generalised myasthenia gravis and chronic inflammatory…
Peptide immunotherapies for the treatment of food allergy
Aravax is a clinical-stage biotechnology company developing next-generation specific immunotherapies for food allergy. Its lead candidate, PVX108, is a synthetic peptide-based therapy designed to desensitise peanut-allergic patients by targeting the T cells that drive allergic responses, without triggering acute…
Clinical-stage oncology company advancing in-licensed cancer drug assets
Ellipses Pharma is a clinical-stage oncology drug development company that in-licenses cancer drug assets from academia and industry, advances them through clinical trials using a continuous funding model, and out-licenses at value-inflection points. Its pipeline spans small molecule kinase inhibitors, selective…
Precision medicine platform for patient recruitment and genetic testing
Sano Genetics is a London-based software company providing an end-to-end platform for patient recruitment, at-home genetic testing, and long-term participant engagement in precision medicine clinical trials and population genomics programmes. The platform integrates DNA testing logistics, medical history retrieval,…
Targeting mitochondrial dysfunction in neurodegeneration
NRG Therapeutics is a clinical-stage neuroscience company developing small molecule inhibitors of the mitochondrial permeability transition pore as disease-modifying treatments for neurodegenerative disorders. Its lead candidate, NRG5051, entered Phase 1 clinical trials in January 2026 for ALS and Parkinson's disease.…
High-capacity viral vector manufacturing platform for gene therapy
Icosphere Biosciences is a biotechnology company developing manufacturing technology for high-capacity gene therapy vectors. Its platform enables scalable, high-yield, contamination-free production of high capacity adenoviral vectors capable of carrying substantially more genetic material than conventional viral…
Personalised cancer treatment selection using in vivo drug screening
Vivan Therapeutics is a London-based biotech developing a personalised cancer therapeutics platform. The company's Personal Discovery Process sequences a patient's tumour genome, engineers those mutations into a fruit fly avatar model, and screens thousands of approved drug combinations at high throughput to identify…
Dual-pathway AAV gene therapies for retinal degeneration
Ikarovec is a biotechnology company developing dual-pathway adeno-associated virus gene therapies for retinal diseases. Its lead candidate combines complementary mechanisms targeting complement activity, inflammation and retinal cell protection in geographic atrophy. Additional programmes target wet and intermediate…
Human iPSC models and AI phenomics for drug discovery
Pixl Bio combines human induced pluripotent stem cell (iPSC) models with AI-driven image analysis for drug discovery and safety research. Its portfolio includes liver cells, stellate cells and disease models, alongside CRISPR engineering, high-content Cell Painting and contract studies. These tools help researchers…
Gene therapies targeting the ageing process and age-related disease
Genflow Biosciences is a longevity-focused biotechnology company developing gene therapies designed to slow or halt the biological ageing process and delay age-related disease. Its lead compound uses an adeno-associated viral vector to deliver a centenarian variant of the SIRT6 gene, with a pipeline spanning human and…
Clinical genomic decision support software for rare disease and oncology
Congenica is a clinical genomic interpretation platform spun out of the Wellcome Sanger Institute. Its software supports variant analysis and clinical reporting across rare disease, oncology and infectious disease, integrating whole genome sequencing data with phenotypic information to generate actionable diagnostic…
ADC oncology company developing novel spliceosome-modulating payloads
Akari Therapeutics is a clinical-stage biopharma company developing next-generation bi-functional antibody-drug conjugates for cancer treatment. Its proprietary ADC platform centres on PH1, a novel spliceosome-modulating small molecule payload designed to disrupt RNA splicing in tumour cells and activate anti-tumour…
CAR-T and bispecific antibody therapies for blood cancers
Hemogenyx Pharmaceuticals is a clinical-stage biopharmaceutical company developing targeted therapies for haematological malignancies and autoimmune diseases. Its lead asset, HG-CT-1, is a third-generation anti-FLT3 CAR-T cell therapy for relapsed and refractory acute myeloid leukaemia, currently in a Phase 1…