Small molecule drug companies develop low-molecular-weight medicines designed to interact with specific biological targets. These compounds can often enter cells and may be formulated as oral or other conventional dosage forms across a wide range of therapeutic areas.
This category includes companies working on:
Browse 221 UK small molecule drug companies below.
Small molecule CDMO delivering process chemistry and API manufacturing services
Onyx Scientific is a contract manufacturing organisation specialising in small molecule active pharmaceutical ingredients. The company supports pharma and biotech clients from discovery through commercial manufacture, offering custom synthesis, lead optimisation, process development, solid state chemistry and GMP…
CRISPR-engineered tumour-infiltrating lymphocyte therapy
Intima Bioscience is a clinical-stage biotechnology company developing CRISPR gene-edited tumour-infiltrating lymphocyte therapies for solid tumours. Its platform targets CISH, an intracellular immune checkpoint that suppresses T cell activity against tumours; CRISPR knockout of CISH in patient-derived TILs enhances…
Fragment-based drug discovery with a glioblastoma focus
IOTA Pharmaceuticals is a Cambridge drug discovery company specialising in fragment-based design, molecular screening and computational approaches to early-stage therapeutics. Its research combines transcriptomics, phenotypic assays and natural product screening to investigate candidate treatments. A major focus is…
AI-driven tRNA synthetase platform for novel small molecule therapeutics
Oxford Drug Design is an Oxford biotech developing small molecule therapeutics using an integrated platform combining aminoacyl-tRNA synthetase biology with proprietary generative AI and machine learning. The platform provides structural biology knowledge, crystallographic data, and novel chemotypes targeting tRNA…
Targeted small-molecule drugs for brain and metastatic cancers
Sentinel Oncology is a biotechnology company developing targeted small-molecule drugs for difficult-to-treat cancers. Its medicinal chemistry programmes investigate DNA damage responses and cell division. Some candidates are designed to cross the blood-brain barrier, targeting primary brain tumours and cancers that…
AI-driven robotic chemistry platform for on-demand molecule synthesis
Chemify is a UK deep-tech company that digitises chemical synthesis by combining a proprietary chemistry programming language, robotics, and artificial intelligence. Its platform translates digital molecular designs into physical compounds, drawing on a large curated database of validated chemical reactions to…
Open-source AI models for biomolecular structure and drug design
Boltz is a software and AI company building biomolecular foundation models for drug discovery. Its models predict the three-dimensional structures of proteins and other biomolecules, estimate how strongly small molecules bind their targets, and generate candidate molecules and proteins. Boltz makes models available…
Tau-targeting therapeutics and diagnostics for neurodegenerative disease
TauRx Pharmaceuticals is a clinical-stage biopharmaceutical company developing treatments and diagnostics for Alzheimer's disease and related neurodegenerative disorders. Its pipeline centres on tau aggregation inhibitors, which target the pathological accumulation of tau protein implicated in dementia. The company's…
Bioanalytical CRO supporting drug development from discovery to post-marketing
York Bioanalytical Solutions is a contract research organisation providing bioanalytical, metabolism and biomarker services to pharmaceutical and biotech companies. The company supports development of small and large molecule therapeutics through mass spectrometry-based bioanalysis, in vitro ADME studies, metabolite…
Automated drug discovery platform and CRO services
Arctoris is a technology-enabled contract research organisation that provides automated wet-lab biology and data generation services to biotech and pharmaceutical companies. The company operates a fully robotic, cloud-controlled platform covering biochemical, biophysical, cell-based and structural biology assays…
CRO-CDMO process chemistry and API manufacturing centre for global pharma partners
Sai Life Sciences operates a UK research and development site providing contract research and development and manufacturing services to global biotech and pharmaceutical companies. The Alderley Park facility serves as a centre of excellence for active pharmaceutical ingredient process and analytical development,…
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 removal of perineuronal nets on spinal cord neurons with treatment of signal-blocking glial scar tissue, enabling new synaptic connections to…
Topical formulations treating atopic dermatitis and inflammatory skin disease
Quodria is a dermatology-focused biotech developing novel topical formulations for skin diseases using nanotechnology-enabled drug delivery. Its lead programme, Q001, is a small molecule galectin-3 inhibitor licensed from University of Liverpool spin-out Galytx, in development as a first-in-class topical treatment for…
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 margins, reducing the systemic side effects of conventional treatment. Developed with the University of Birmingham, the company's lead programme…
Non-invasive small molecule eye drops for retinal vascular disease
Foventa is a biopharmaceutical company developing non-invasive, small molecule therapeutics for retinal vascular diseases that can cause vision loss. Its lead candidate targets VEGF splicing, a mechanism underlying abnormal blood vessel growth in conditions including diabetic retinopathy and diabetic macular oedema.…
Epigenetic reprogramming and senolytic therapeutics for cancer and ageing
199 Biotechnologies is a preclinical-stage biotechnology company developing epigenetic reprogramming and senolytic therapies targeting cancer and age-related disease. Its lead programme applies partial cellular reprogramming to modify the behaviour of glioblastoma cells, reducing malignancy and reactivating natural…
Preclinical orexin-1 addiction therapies and CNS drug discovery
TheraCryf is an AIM-listed biotechnology company developing small-molecule therapies for addiction and other neuropsychiatric conditions. Its lead orexin-1 receptor antagonist programme is completing preclinical toxicology and regulatory preparations for a first-in-human Phase 1 trial in Australia. An Australian CRO…
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 has obtained regulatory approvals for three products across the FDA, EMA and MHRA, and has developed a proprietary intranasal dry powder…
Regulatory affairs, quality assurance and clinical development consulting
G&L Scientific is a Belfast-founded regulatory affairs and quality assurance consultancy providing integrated product development and market access services to pharmaceutical, biopharmaceutical, and biotechnology clients globally. Services span CMC, clinical development, regulatory strategy and submissions,…
Academic drug discovery accelerator developing small molecule therapeutics
Slingshot Therapeutics is a drug discovery accelerator that identifies early-stage scientific programmes from academic institutions and advances them towards clinical development. The company provides centralised expertise, operational infrastructure, and funding to progress small molecule candidates through…