Company Directory

Small molecule drug discovery targeting rare genetic diseases and oncology

Tay Therapeutics is a drug discovery company developing targeted small molecule therapies for rare genetic diseases and cancer. Its platform uses stop codon read-through technology to restore production of functional proteins disrupted by premature stop codon mutations, with programmes targeting oncology, cystic fibrosis, Duchenne muscular dystrophy, and epidermolysis bullosa. The company previously discovered and out-licensed two BET inhibitor programmes to VYNE Therapeutics, which are advancing in clinical trials for immuno-inflammatory conditions.

Clinical-stage antifungal drug developer targeting invasive fungal infections

F2G is a clinical-stage biopharmaceutical company developing novel antifungal therapies for life-threatening invasive fungal infections. The company discovered the orotomides, a new class of antifungal agents that selectively inhibit fungal dihydroorotate dehydrogenase, a mechanism of action distinct from all approved antifungals. Its lead compound, olorofim, is an oral agent in Phase 3 development for invasive aspergillosis and has received FDA Breakthrough Therapy Designation for multiple indications.

Drug discovery platform targeting biomolecular condensates

Transition Bio is a biotechnology company developing a drug discovery platform based on the biology of biomolecular condensates, the membrane-less cellular structures implicated in a range of diseases. Using droplet microfluidics and machine learning, the company maps and modulates condensate behaviour to identify small-molecule candidates against targets previously considered undruggable. Lead programmes include a collaboration with Voyager Therapeutics targeting TDP-43 in ALS and frontotemporal dementia, and an internal oncology programme targeting MYC-driven cancers.

Transformative medicines for cystic fibrosis and serious genetic diseases

Vertex Pharmaceuticals is a global biopharmaceutical company with its European R&D headquarters at Milton Park, Oxfordshire, and its international commercial headquarters in London. The Oxford site conducts full-spectrum drug discovery encompassing human genetics, structural biology, and medicinal chemistry, and has been central to Vertex's development of approved therapies for cystic fibrosis. The company's broader pipeline addresses sickle cell disease, beta-thalassaemia, pain, and kidney disease, including gene-edited cell therapies.

UK commercial base for a rare metabolic disease biotech

Amicus Therapeutics UK is the British subsidiary of Amicus Therapeutics, a US-listed biotechnology company focused on rare metabolic diseases. The company develops enzyme replacement therapies and gene therapies targeting lysosomal storage disorders, with approved medicines for Pompe disease and Fabry disease. The UK office in Marlow serves as the commercial and medical affairs base for Great Britain and Ireland.

Clinical-stage therapeutics for advanced liver disease

Yaqrit is a clinical-stage biotechnology company developing treatments for patients with advanced liver disease, including cirrhosis and acute-on-chronic liver failure. Its pipeline includes an oral ammonia scavenger for hepatic encephalopathy, an extracorporeal liver support device, and a microbiome-based therapeutic. Multiple programmes are in Phase 2–3 development. The company's science originates from over two decades of research by Professor Rajiv Jalan and his team at University College London and the Royal Free Hospital.

AAV gene therapy platform targeting podocyte-mediated kidney disease

Purespring Therapeutics is a clinical-stage biotech developing AAV-based gene therapies for chronic kidney disease. Its platform delivers therapeutic transgenes directly to podocytes, specialised cells implicated in the majority of renal diseases, enabling disease-modifying treatment of both rare monogenic and more prevalent acquired kidney conditions. The lead programme, PS-002, targets IgA nephropathy and has received MHRA CTA approval for a Phase I/II trial.

AI-powered rare disease case finding for primary care

Mendelian is a digital health software company developing AI-driven tools to support the earlier diagnosis of rare diseases in primary care. Its core product is a Class 1 regulated medical device that analyses electronic health records to identify patients whose symptom profiles are consistent with published diagnostic criteria for hundreds of rare conditions. The platform integrates with NHS clinical workflows and is used by GPs and primary care networks across the UK.

Mitochondrial sulfide-donor therapeutics for obesity and myopathies

MitoRx Therapeutics is a company developing first-in-class small molecule therapeutics that target mitochondrial sulfide signalling, a mechanism implicated in metabolic dysfunction and muscle disease. Its lead programme is an injectable mitochondria-targeted sulfide donor designed to achieve fat loss without lean mass loss, positioning it as a differentiated alternative to GLP-1 receptor agonists. Preclinical studies have also demonstrated efficacy in models of Duchenne muscular dystrophy and Alzheimer's disease.

Specialist CRO for rare disease drug development and commercialisation

TMC Pharma Services is a contract research organisation specialising in drug development support for rare and orphan diseases. The company serves small and mid-sized biotech and biopharma clients across the full development lifecycle, providing regulatory affairs, clinical development, pharmacovigilance, medical services, quality management, and drug commercialisation services including marketing authorisation holder functions. Therapeutic areas of focus include oncology, rare neurological disorders, respiratory disease, and haematology.

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 biotechnology companies, it designs and optimises delivery strategies for gene therapies and other treatments directed at specific brain regions, bypassing the blood-brain barrier. The company develops proprietary implantable neurosurgical delivery devices and provides pre-clinical and clinical trial support for CNS drug delivery programmes in neurodegenerative, neuro-oncological, and rare neurological conditions.

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 Europe leads clinical development, regulatory affairs, and medical affairs across oncology, neuroscience, gastrointestinal disease, rare diseases, and vaccines. Takeda Cambridge, based at Cambridge Science Park, conducts target-based drug discovery research in CNS disorders and metabolic diseases.