Gene therapy companies develop medicines that introduce, replace or modify genetic material to treat disease. Their programmes span inherited disorders, rare diseases, cancer and other conditions where addressing the underlying genetic cause may provide a lasting therapeutic effect.
This category includes companies working on:
Browse 30 UK gene therapy companies below.
Next-generation viral vectors engineered for safer gene therapy delivery
Sandbox Bioscience is a biotechnology company developing next-generation viral delivery vectors for gene therapy. Founded by a team of virologists with extensive experience in gene therapy delivery systems and platform development, the company is engineering vectors designed to improve on existing market solutions…
Gene therapy immune regulators for infection, cancer and autoimmunity
Virothera develops gene therapies intended to regulate immune responses in persistent diseases. Its approach draws on human versions of immune-modifying proteins that viruses have evolved to influence immune cells. The company aims to use these proteins to create targeted treatments with potential advantages over some…
Gene therapy biotech targeting neural circuit dysfunction
Sania Therapeutics is a biotechnology company developing genetic medicines for diseases caused by dysfunctional neural circuits. Its approach combines precision AAV delivery to selected neural circuits with tuneable neuromodulation, using human iPSC-derived neural models to direct the evolution of human-centric…
Gene therapy company targeting inherited and degenerative diseases
MeiraGTx is a vertically integrated, clinical-stage genetic medicines company developing AAV-based gene therapies for inherited retinal diseases, neurodegenerative conditions, and salivary gland disorders. The company operates end-to-end capabilities spanning viral vector design, GMP manufacturing, and clinical…
RNA trans-splicing gene therapies for aggressive cancers
Spliceor is a University of Cambridge spinout developing RNA trans-splicing gene therapies for aggressive cancers. Its platform is designed to produce therapeutic messenger RNA selectively in targeted cells by joining segments from different RNA molecules. Machine learning helps refine the constructs and optimise…
Non-viral gene therapy platform enabling durable nuclear DNA delivery
Gramics Bio develops a gene therapy platform solving a key limitation of lipid nanoparticle delivery: enabling therapeutic DNA to reach the cell nucleus for durable expression without viral vectors. By programming specificity into synthetic DNA sequences rather than new delivery chemistries, the technology combines…
RNA-based gene control systems for safer cell and gene therapies
Concinnity Genetics is a biotechnology company developing RNA-based control systems that enable precise regulation of gene therapies after administration. Its technology allows therapeutic gene expression to be modulated or reduced in response to side effects or clinical need, addressing a fundamental safety…
Historical gene therapy platform targeting cancer stem cells
Cellinta was a University of Edinburgh-linked biotechnology company established to develop selective gene therapies targeting cancer stem cells in solid tumours. Its approach used stem-cell-specific regulatory sequences to restrict therapeutic gene expression to tumour-driving cell populations. The technology…
AAV gene therapy company targeting inherited and common retinal diseases
Beacon Therapeutics is a clinical-stage UK biopharma company developing adeno-associated virus (AAV) gene therapies for retinal diseases causing blindness. Its lead programme, laru-zova (AGTC-501), is in Phase II/III clinical trials for X-linked retinitis pigmentosa. The pipeline also includes preclinical programmes…
Inhaled lentiviral gene therapies for rare respiratory diseases
AlveoGene is a biotechnology company developing inhaled gene therapies for rare respiratory disorders. It uses a lentiviral vector platform engineered to deliver therapeutic genes efficiently to the cells lining the airways and lungs. Its lead programme targets alpha-1 antitrypsin deficiency, an inherited condition…