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.
Next-generation programmable gene insertion platform technology
Gene Weaver is a preclinical-stage biotechnology company developing a programmable, RNA-based gene insertion platform. Its RNA-guided enzymatic system functions as a complete molecular machine, locating the target site and inserting large DNA payloads to enable kilobase-scale gene insertion at virtually any genomic…
Biologics CDMO for development and cGMP manufacturing
Northway Biotech is a contract development and manufacturing organisation (CDMO) specialising in biologics and advanced therapies. It develops mammalian cell lines and microbial production strains, provides upstream and downstream process development, and supports analytical method development and cGMP manufacturing.…
Non-viral intracellular delivery platform based on biomolecular condensates
PartitionBio is a biotechnology company developing a non-viral intracellular delivery platform based on liquid-liquid phase separation and biomolecular condensates. Its lead product, BubbleFect, is a room-temperature-stable, non-lipid reagent designed for bench-top use without specialist equipment or cold-chain…
Near-infrared bioluminescence reagents for preclinical imaging research
Bioflares is a biotechnology company developing and supplying infraluciferin-based reagents and engineered luciferase enzymes for near-infrared bioluminescence imaging. Its patented infraluciferin substrate enables simultaneous dual-parameter imaging of two biological processes in living animals, supporting…
Global viral vector CDMO for cell and gene therapy
Oxford Biomedica (OXB) is a global contract development and manufacturing organisation specialising in viral vectors for cell and gene therapy. The company provides lentiviral, adeno-associated virus (AAV), and adenoviral vector development and manufacturing services from early-stage process development through to…
Automated bioreactor platform for cell and gene therapy manufacturing
microfluidX is a UK instruments company developing automated bioprocessing platforms for cell and gene therapy research and manufacturing. Its core product is a microfluidics-based bioreactor system designed for parallel cell culture process development and scalable cell expansion. The platform integrates online…
Generative AI platform for TCR therapy design and immune repertoire analysis
Synteny Biotechnology is a biotechnology company developing a platform that combines high-throughput TCR-antigen data generation with generative AI to design precision T cell receptor therapies for cancer. The platform addresses a critical bottleneck in the field, namely the scarcity of paired TCR-epitope interaction…
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…
Synthetic promoter platform for precision gene expression control
Chromatin Bioscience designs synthetic promoters and gene expression control systems for cell and gene therapy developers. Its platform uses bioinformatics to identify cell-type-selective regulatory elements from the human genome, which are assembled into novel synthetic promoters enabling precise, durable and…
Curative genetic medicines for peripheral nerve diseases
CureAge Therapeutics develops genetic medicines for peripheral nerve disorders, initially targeting neurofibromatosis type 1 (NF1). Its investigational platform uses locally delivered lipid nanoparticles to carry genetic material into Schwann cells, aiming to restore NF1 gene function and address surrounding tumour…
Next-generation lateral CAR-T therapies for refractory solid tumours
Leucid Bio Ltd is a clinical-stage biotechnology company developing next-generation CAR-T cell therapies for hard-to-treat solid tumours. Founded on over two decades of King's College London research, the platform redesigns the CAR signalling architecture to enhance T-cell potency, persistence, and tumour…
Engineered patient cells for sustained therapeutic antibody production
Semper-B Therapeutics is a biotechnology company developing a cell-engineering platform intended to enable patients’ own cells to produce therapeutic antibodies over prolonged periods. The approach aims to achieve durable, precise modulation of immune activity by using engineered cells as a continuing source of…
Real-time cell aggregate monitoring for stem cell manufacturing
MorphoKinetics is a life science instrumentation company developing real-time monitoring technology for stem cell manufacturing. Its process analytical platform combines automated sampling, machine vision and artificial intelligence to measure the size, shape and growth of cell aggregates in bioreactor cultures. The…
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…
Engineered immune cells as living delivery vehicles for genetic medicines
Dart Biosciences is a biotechnology company developing a platform that uses engineered immune cells as living delivery vehicles for genetic medicines. By leveraging immune cells' natural ability to localise to disease-relevant tissues, the platform aims to achieve more spatially precise genetic payload transfer with…
Gene therapy company targeting complement-mediated ocular diseases
Complement Therapeutics is a clinical-stage biotechnology company developing gene therapies and protein-based therapeutics for diseases driven by dysregulation of the complement immune pathway. A spin-out from the University of Manchester, the company's lead programme CTx001 is an AAV-based gene therapy for geographic…
Robotic automation platform for scalable cell therapy manufacturing
Cellular Origins is a biotechnology company developing robotic automation systems for the industrial-scale manufacture of cell therapies. Its platform uses mobile robotics and automated sterile fluidic interconnection to link existing bioprocess instruments into fully automated, GMP-compliant manufacturing workflows.…
Programmable cell-killing particle platform for targeted therapeutic delivery
Granza Bio is a biotechnology company developing a cell-targeted delivery platform based on supramolecular attack particles, naturally occurring structures used by cytotoxic T cells and natural killer cells to deliver lethal payloads with precision. The company is engineering programmable versions of these particles,…
AAV gene therapy for Bardet-Biedl syndrome and genetic obesity
Axovia Therapeutics is a biotechnology company developing adeno-associated viral gene therapies for inherited ciliopathies and genetic obesity. Its lead candidate delivers a functional BBS1 gene into retinal cells to investigate treatment of vision loss associated with Bardet-Biedl syndrome. A first-in-human, early…
AI-driven intron technology to enhance transgene expression
ExpressionEdits is a biotechnology company developing computational tools to improve protein expression from artificial gene constructs. Its Genetic Syntax Engine uses machine learning and biological data to predict optimal intron insertion patterns within transgenes, increasing protein production levels in mammalian…