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 neuromodulators for neuropsychiatric disorders
Draig Therapeutics is a clinical-stage biotech developing small molecule drugs that modulate the glutamate and GABA neurotransmitter pathways to treat neuropsychiatric disorders. Its lead candidate DT-101 is an AMPA receptor positive allosteric modulator in Phase 2 development for major depressive disorder. The…
Academic cancer research institute and drug discoverer
The Institute of Cancer Research (ICR) is an independent research institute and member institution of the University of London, specialising in cancer biology, drug discovery, genetics, and precision radiotherapy. Working closely with the Royal Marsden NHS Foundation Trust, the ICR has discovered cancer medicines…
Small-molecule drugs targeting cellular ageing and senescence
Five Alarm Bio Limited is a biotechnology company discovering small-molecule therapies for diseases associated with ageing. Its research targets chemical damage that accumulates inside cells, aiming to reinforce protective mechanisms and delay cellular senescence rather than destroy senescent cells. Candidate…
Biotech venture builder co-creating and scaling drug discovery companies
Molecule to Medicine (MTM) is a biotech venture builder that co-creates, builds, and scales drug discovery companies within a shared operational ecosystem, combining drug discovery expertise with shared infrastructure to enable rapid company formation. UK operations are based at Milton Park, Abingdon, with a further…
Solid form sciences and API development for drug candidates
Veranova's Cambridge site is a centre of excellence in solid form and particle engineering, operating under the Pharmorphix brand. The facility provides polymorph screening, salt selection, co-crystallisation, chiral separations, and process scale-up services to pharmaceutical and biotechnology customers. Part of…
AI-powered drug design engine built on AlphaFold technology
Isomorphic Labs is an artificial intelligence company applying frontier AI to drug discovery, built on and beyond the Nobel Prize-winning AlphaFold protein structure prediction system developed at Google DeepMind. Its proprietary drug design engine spans target identification, molecular design, and binding prediction…
Formulation development CDMO for difficult pharmaceuticals
Agility Life Sciences is a contract development and manufacturing organisation specialising in pharmaceutical formulation development. Based in Stapleford, Nottingham, the company helps biotech and pharma clients overcome solubility, stability, and bioavailability challenges across oral, ocular, intravenous,…
Fragment and structure-based drug discovery services
Vernalis (R&D) is a Cambridge-based contract drug discovery organisation specialising in fragment-based and structure-based drug discovery. The company integrates structural biology, biophysics, NMR spectroscopy, DNA-encoded library screening, medicinal chemistry, and computational approaches to identify and progress…
G-quadruplex-targeting medicines for precision cancer treatment
Genome Therapeutics is a biotechnology company developing cancer medicines that target G-quadruplex DNA structures associated with tumour biology. Its approach builds on research into non-canonical DNA folding and how these structures influence gene regulation and cancer cell vulnerabilities. The company develops…
Small molecule drugs targeting astrocytes in neurodegenerative disease
AstronauTx is a clinical-stage UK biotech developing oral small molecule therapeutics for Alzheimer's disease and other neurodegenerative disorders. The company's approach targets astrocytes, brain support cells whose dysregulated inflammatory activity contributes to neurodegeneration, aiming to restore normal…
Structure-guided small-molecule therapeutics for antiviral and metabolic diseases
Halo Therapeutics is a University of Bristol spinout developing structure-guided small-molecule medicines for infectious and metabolic diseases. Its research combines cryo-electron microscopy, protein biophysics and computational chemistry to identify drug-binding pockets and design selective compounds. Its antiviral…
Global generics and specialty pharmaceutical manufacturer headquartered in London
Hikma Pharmaceuticals is a multinational pharmaceutical company developing, manufacturing and marketing generic, specialty and branded medicines across injectable, oral and respiratory dosage forms. The company's portfolio spans oncology, cardiovascular, respiratory, pain management and other therapeutic areas,…
AI platform targeting chromosomal instability across cancer
Tailor Bio is a Cambridge-based biotechnology company developing an AI-driven precision medicine platform for cancers with chromosomal instability, a genomic phenomenon present in over 80% of all cancers. Founded from research originating at the Cancer Research UK Cambridge Institute, the company develops both…
Small molecule therapies for cystic fibrosis and COPD
Enterprise Therapeutics is a clinical-stage biopharmaceutical company developing novel muco-regulatory therapies for respiratory diseases. Its lead programme ETD001 is a first-in-class inhaled ENaC blocker designed to hydrate airway mucus and improve mucociliary clearance, currently in a Phase 2a trial in cystic…
E3 ligase inhibitors stabilising tumour suppressor proteins
Outrun Therapeutics is a preclinical drug discovery company developing small molecule inhibitors of E3 ligase enzymes to stabilise tumour suppressor and other disease-relevant proteins. Its proprietary platform has been used to profile over 45 E3 ligases across oncology, cardiovascular, neurological, and autoimmune…
Specialist CRO for drug and agrochemical metabolite synthesis and characterisation
Hypha Discovery is a specialist contract research organisation supporting pharmaceutical and agrochemical companies worldwide. The company produces and purifies drug and agrochemical metabolites using microbial biotransformation, recombinant enzyme systems and late-stage chemical synthesis, supporting metabolite…
DUB inhibitor therapeutics targeting mitophagy and degenerative disease
Mission Therapeutics is a clinical-stage biotechnology company developing first-in-class small molecule inhibitors of deubiquitylating enzymes to treat diseases driven by mitochondrial dysfunction. By targeting a mitochondria-localised DUB, the company's drug candidates are designed to enhance mitophagy, the cellular…
Small molecule GPR65 inhibitors restoring immune response in solid tumours
Pathios Therapeutics is a clinical-stage drug discovery company developing small molecule inhibitors of GPR65, a pH-sensing G protein-coupled receptor that drives immunosuppressive polarisation of tumour-associated macrophages in the acidic tumour microenvironment. By blocking GPR65, Pathios aims to restore…
Precision oncology drugs and vaccines for cold tumours
IngenOx Therapeutics Limited is a clinical-stage biopharmaceutical company developing precision oncology drugs and cancer vaccines for difficult-to-treat tumours. The company serves oncology patients and clinical development partners through a pipeline spanning small molecule immunomodulators, PRMT5 inhibition and…
AI-driven rescue and development of failed drug candidates
Ignota Labs is a biotechnology company that identifies, in-licenses and redevelops drug candidates that have failed clinical development due to toxicity. Using deep learning applied to cheminformatics and bioinformatics data, the company resolves the mechanistic basis of safety failures and makes targeted chemical…