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 21 UK gene therapy companies below.
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…
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…
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…
AAV gene therapies for rare neurological diseases of childhood
BlackfinBio is a biotechnology company developing AAV-based gene therapies for rare neurodegenerative diseases of the central nervous system, with an initial focus on children.…
Gene therapy platform targeting paediatric solid tumours
Kindling Bio is a biotechnology company developing a gene therapy platform designed specifically for childhood cancers. The platform engineers a biological signal that renders…
AAV gene therapies for rare and neurological diseases
Spur Therapeutics is a clinical-stage biotechnology company developing adeno-associated virus-based gene therapies for serious chronic diseases. Its lead programme is a Phase I/II…
Ex vivo haematopoietic stem cell gene therapies for rare genetic diseases
Orchard Therapeutics is a gene therapy company developing ex vivo haematopoietic stem cell therapies for rare genetic diseases, primarily severe neurometabolic and immunological…
AAV gene therapies for neurodegenerative diseases including FTD and ALS
AviadoBio is a clinical-stage gene therapy company developing AAV-based treatments for neurodegenerative diseases. Its lead programme, AVB-101, is in a Phase 1/2 trial for…
DNA-based gene therapy platform for intercellular therapeutic gene delivery
Bitrobius Genetics is a UK biotech developing a novel DNA-based gene therapy platform technology designed to deliver functional copies of mutated genes to cells within a target…
AAV gene therapy platform and clinical pipeline for genetic disease
AskBio UK Ltd is the European headquarters of AskBio Inc., a fully integrated AAV gene therapy company and Bayer AG subsidiary. Operating from Roslin Innovation Centre, the…
Bicistronic AAV gene therapies for retinal disease
Ikarovec is a near-clinical-stage gene therapy company developing bicistronic AAV-based therapeutics for vision-threatening retinal diseases. Its proprietary platform combines two…
AAV gene therapy targeting the seizure focus in refractory epilepsy
EpilepsyGTx is a biotechnology company developing gene therapies for focal refractory epilepsy, a condition in which seizures arise from a specific brain region and do not respond…