Find actively recruiting gene therapy clinical trials in the UK. From CRISPR gene editing to viral vector therapies, discover how genetic medicine is curing previously untreatable diseases.
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Gene therapy treats or cures disease by introducing, altering, or replacing genetic material within your cells. Unlike conventional drugs that manage symptoms, gene therapy addresses the root cause at the DNA level — offering the potential for one-time, curative treatments.
Gene therapy uses viral vectors (modified viruses that cannot cause disease) to deliver therapeutic genes into cells. Approaches include: Gene addition — inserting a working copy of a faulty gene. Gene editing — using CRISPR-Cas9 or zinc fingers to precisely modify DNA. Gene silencing — using RNA interference to turn off harmful genes. In vivo therapy delivers genes directly to the body; ex vivo modifies cells outside the body first.
Actively recruiting studies in the United Kingdom
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Many gene therapies are designed as one-time treatments because the therapeutic gene continues to produce the needed protein indefinitely. However, this depends on the disease, delivery method, and whether the target cells divide. Some may need repeat dosing, especially for non-dividing cells targeted by in vivo approaches.
Gene therapy is approved or in trials for haemophilia A and B, sickle cell disease, beta-thalassaemia, spinal muscular atrophy, inherited retinal diseases, certain cancers, and many rare genetic disorders. The pipeline is rapidly expanding with over 2,000 gene therapy trials worldwide.
Risks include immune reactions to the viral vector, off-target effects of gene editing, insertional mutagenesis (where the new gene disrupts a tumour suppressor gene), and unpredictable long-term effects. Trial protocols include extensive monitoring and long-term follow-up (typically 15 years).
Gene Therapy is used across multiple conditions. Select your condition for targeted results.
How does this treatment compare with alternatives?