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 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,…
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…
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 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…
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…
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…
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…
Gene therapies targeting cancer stem cells in solid tumours
Cellinta is a pre-clinical biotechnology company developing highly selective gene therapies for solid tumour cancers. Its platform identifies transcriptional regulatory circuits…
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,…