Gene Therapy Clinical Trials in the UK 2026 — Find Active Studies
Gene therapy is transforming medicine by addressing diseases at their root cause — the genetic code. The UK is a world leader in gene therapy research, with NHS patients among the first globally to receive approved treatments for conditions like sickle cell disease, haemophilia, and inherited retinal disorders. Clinical trials are expanding rapidly into more conditions and using increasingly sophisticated gene editing tools like CRISPR-Cas9.
Types of Gene Therapy in Clinical Trials
Delivers a functional copy of a defective gene using a viral vector (typically AAV). Used for monogenic diseases like haemophilia, spinal muscular atrophy, and inherited retinal dystrophies. The new gene produces the missing or defective protein.
Uses CRISPR-Cas9 or similar tools to directly edit the DNA in your cells. Can correct mutations, disrupt harmful genes, or insert new genetic material. Clinical trials are active for sickle cell disease, beta-thalassemia, and some cancers.
Uses RNA interference (RNAi) or antisense oligonucleotides to "turn off" disease-causing genes. Approved therapies include patisiran for hereditary ATTR amyloidosis. Trials expanding into more conditions.
Modified viruses that selectively infect and kill cancer cells while stimulating immune responses. T-VEC is approved for melanoma; next-generation oncolytic viruses are in trials for multiple cancer types.
Conditions with Gene Therapy Trials
- Sickle Cell Disease — CRISPR-edited (exagamglogene) is now approved; trials for younger patients and simpler approaches
- Haemophilia A & B — AAV gene therapy (valoctocogene roxaparvovec, etranacogene dezaparvovec) showing years of factor production from a single dose
- Macular Degeneration — Subretinal gene therapy delivering anti-VEGF genes directly to the retina
- Motor Neurone Disease (ALS) — Antisense oligonucleotides and gene therapy targeting SOD1, C9orf72 mutations
- Leukaemia — CAR-T cell therapy (a form of gene therapy) for blood cancers
- Beta-Thalassemia — Gene therapy and gene editing approaches to restore haemoglobin production
How Gene Therapy is Delivered
Gene therapy delivery methods depend on the target disease:
- Intravenous (IV) infusion — The most common method. Viral vectors are infused into the bloodstream and travel to target organs.
- Subretinal injection — For eye conditions. A tiny amount of gene therapy is injected directly under the retina in a surgical procedure.
- Ex vivo modification — Cells are removed, genetically modified in the lab, and returned (like CAR-T therapy). Used for blood disorders.
- Intrathecal injection — Direct injection into the spinal fluid for neurological conditions like SMA.
- Intrahepatic injection — Direct delivery to the liver for metabolic conditions.
Note: Many gene therapies are one-time treatments designed to provide lasting benefit from a single dose. This is fundamentally different from conventional medicines that require ongoing dosing.
Safety and What to Expect
- Gene therapy trials require extensive pre-treatment screening including genetic testing, organ function tests, and antibody screening
- Some gene therapies require immunosuppression to prevent immune reactions to the viral vector
- Long-term follow-up is required (typically 15 years) to monitor for delayed effects
- Potential risks include immune reactions, off-target genetic effects, and insertional mutagenesis (very rare with modern vectors)
- Benefits can be dramatic — some patients have gone from requiring regular transfusions to being transfusion-independent
- Your trial team includes geneticists, specialist nurses, and genetic counsellors
Find Your Matching Trials
Answer a few questions about your condition and we'll show you the most relevant actively recruiting trials near you.
Find My Matching Trials →Browse Gene Therapy Trials Search All Trials