Cell and gene therapy companies develop advanced treatments that modify, replace or engineer cells and genetic material to treat disease. The field is one of the fastest-growing areas of biotechnology and precision medicine.
This category includes companies working on:
Browse 163 UK cell and gene therapy companies below.
Enzymatic synthetic DNA manufacture for gene therapy and molecular biology
NunaBio is a specialist DNA synthesis company developing a cell-free enzymatic platform for scalable production of synthetic DNA, including long oligonucleotides, whole genes and DNA fragments. Its technology avoids the plasmid and primer-dependent steps that constrain conventional phosphoramidite chemistry, enabling…
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…
Engineered T cell receptor therapies for solid tumour cancers
Adaptimmune is a clinical-stage biopharmaceutical company developing engineered T cell receptor therapies targeting solid tumours. The company's T-cell platform engineers T cell receptors with enhanced affinity for cancer-associated antigens, enabling immune recognition of tumour cells that evade conventional…
Viral vector platform for solid tumour immunotherapy
Akamis Bio is a clinical-stage oncology company developing oncolytic adenoviral therapeutics for solid tumours. Its platform delivers immunotherapeutic payloads directly to tumour sites following intravenous dosing, enabling tumour-localised expression of proteins including CD40 agonist antibodies and cytokines. Lead…
Lentiviral vector yield enhancement via HDAC inhibitors and producer lines
Synthistat is a biotechnology company developing technologies to increase lentiviral vector production for cell and gene therapy manufacturing. Its work combines designed histone deacetylase (HDAC) inhibitors with approaches to modify HEK293 producer cells. The company has reported early laboratory results from…
Synthetic polymer affinity ligands for viral vector bioprocessing
Tozaro is a biotechnology company developing synthetic polymer-based affinity ligands to improve the downstream processing of viral vectors used in cell and gene therapy. Using molecular modelling and machine learning, Tozaro rationally designs polymer-based binders from a chemical space of over 500 monomers, offering…
National facility for mouse genetics and preclinical disease modelling
The Mary Lyon Centre at MRC Harwell is the UK's national centre for mouse genetics, providing mouse model generation, phenotyping, cryopreservation, and archiving services to academic and industry researchers worldwide. The Centre offers genome engineering using CRISPR and other techniques to create genetically…
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…
Programmable gene control platform for advanced cell therapies
Laverock Therapeutics is a biotechnology company developing a programmable gene control platform for the creation of disease-responsive advanced therapies. The platform uses recoded microRNAs to deliver tunable, stable and multiplexed gene silencing within cell therapies, enabling enhanced precision and improved…
Ex-vivo organ perfusion services for preclinical drug evaluation
Pebble Bio is a contract research organisation providing ex-vivo organ and tissue perfusion services as an alternative to large animal experimentation. Using living organ systems maintained under physiological conditions, the company evaluates the safety, efficacy, and pharmacology of novel therapeutics, biomedical…
In vivo nanoparticle screening for genetic medicine delivery
Ride Therapeutics is a joint University of Cambridge and Harvard spin-out developing nanoparticle delivery systems for mRNA and gene-editing medicines. Its Molecular Logistics platform engineers and screens large libraries of non-viral carriers in living systems, measuring tissue and cell-type targeting to train an…
Mass photometry instruments for single-molecule biomolecular characterisation
Refeyn is an analytical instrumentation company and the pioneer of mass photometry, a light-scattering technique that measures the mass of individual molecules in solution without labels. Its benchtop instruments serve pharmaceutical, biotechnology, and academic customers for characterisation of proteins, nucleic…
Tissue-targeted gene and RNA delivery platforms for rare and neurological disease
Dawn Therapeutics is a pre-clinical biotechnology company developing precision delivery platforms for genetic medicines targeting the central nervous system, cartilage, and bone. Its transferrin receptor-guided system is designed to cross the blood-brain barrier and deliver RNA or DNA payloads to neuronal tissues,…
Off-the-shelf Natural Killer cell therapies for solid and blood cancers
NK:IO develops cancer immunotherapies based on human Natural Killer cells, harnessing the body's innate immune system as an alternative to T-cell-based approaches. The company's patented platform activates cord blood-derived stem cells to produce NK cells at high potency and yield, addressing manufacturing challenges…
Targeted magnetic blood filtration to enable gene therapies
MediSieve is a biotechnology company developing selective magnetic blood filtration using engineered particles that bind specific circulating biological targets. Its extracorporeal system combines target-specific magnetic beads with a capture device designed to remove bound substances before blood returns to the…
Exosome-delivered gene editing therapies for neurodegenerative diseases
Evox Therapeutics is a biotechnology company developing exosome-based gene editing therapies for rare, genetically driven neurodegenerative diseases. Its proprietary platform uses engineered exosomes to deliver CRISPR-based gene editors across the blood-brain barrier, enabling targeted genetic intervention in the…
Cell-specific mRNA delivery platform for genetic medicines
Nuntius Therapeutics is a London-based biotechnology company developing mRNA therapies using proprietary peptide dendrimer and lipid-based nanocarriers designed to deliver genetic payloads to tissues and cell types beyond the liver. The platform combines machine learning-guided carrier design with engineered mRNA…
AI vision systems for surgical workflows and cell therapy manufacturing
Appraiseye develops AI-powered vision systems for life sciences and healthcare applications across two platforms. Its operating theatre intelligence platform provides real-time instrument tracking, tray utilisation analytics and sterile processing optimisation for surgical departments. Its platform applies AI-guided…
Computational DNA design for durable genetic medicines
Houdini Bio is an early-stage biotechnology company using computational synthetic biology to redesign DNA for improved performance in genetic medicines. The company is developing approaches to enhance the durability and scalability of gene expression, with potential applications across gene therapies and DNA-based…
Cell-based regenerative therapies for heart failure
Cardiogeni is a clinical-stage biotechnology group developing regenerative cell medicines for cardiovascular disease. Its lead programme, CLXR-001, uses engineered immunomodulatory progenitor cells intended to support repair of damaged heart tissue in patients with heart failure. The group is progressing plans for…