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.
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…
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…
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…
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…
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…
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…
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. Its lead programme is a one-time gene therapy administered directly to the brain, designed to treat a group of ultra-rare dopamine deficiency…
Lentiviral gene therapy developer for tumours and rare diseases
Santo Therapeutics develops in vivo lentiviral gene therapies for cancer and rare genetic disorders. Its proprietary vector platform supports programmes including ST002, an NF2 gene-replacement therapy for NF2-related schwannomatosis. The company announced the start of an investigator-initiated clinical trial of ST002…
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 paediatric solid tumours visible to the immune system, enabling immune-mediated destruction across multiple tumour types from a single…
AAV gene therapy targeting the seizure focus in refractory epilepsy
Focalis Bio 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 to antiseizure medicines. Its lead programme, EPY201, is an AAV9-based gene therapy delivered locally via catheter directly to the…
Targeted nanoparticles for in vivo CAR-T and gene delivery
Deliver Biosciences is a biotechnology company developing targeted nanoparticle vectors for in vivo cell and gene therapies. Its delivery platform transports genetic payloads, including DNA and mRNA, into selected cell types using programmable targeting components. Its lead programme aims to engineer T cells inside…
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 gene therapy for Gaucher disease type 1, a rare lysosomal storage disorder. Further pipeline candidates target GBA1 Parkinson's disease and…
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 disorders in children. Its platform modifies a patient's own stem cells with a functional copy of the defective gene before reinfusion. Lead…
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 frontotemporal dementia caused by GRN gene mutations, delivering a functional copy of the GRN gene via intrathalamic infusion to restore…
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 tissue or organ. Its platform is based on the principle of DNA replication and intercellular transmission, aiming to achieve broad tissue…
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 Edinburgh site develops the company's synthetic promoter technology, data-driven gene expression control sequences that complement AskBio's Pro10…
Dual-pathway AAV gene therapies for retinal degeneration
Ikarovec is a biotechnology company developing dual-pathway adeno-associated virus gene therapies for retinal diseases. Its lead candidate combines complementary mechanisms targeting complement activity, inflammation and retinal cell protection in geographic atrophy. Additional programmes target wet and intermediate…
Gene therapy platform delivering precision cancer treatments
Trogenix is a clinical-stage biotechnology company developing precision gene therapies for aggressive solid tumours. Its platform uses proprietary synthetic super-enhancers, engineered genetic constructs delivered via AAV vectors, to selectively activate in cancer cells and simultaneously trigger tumour cell death and…
Full-service CRO delivering clinical trials, regulatory and pharmacovigilance services
ProPharma is a contract research organisation providing full-service clinical trial delivery alongside regulatory affairs, pharmacovigilance, quality and compliance, and medical information services to pharmaceutical, biotechnology and medical device companies. The company supports clinical operations, site and…