The number of Advanced Therapy Medicinal Products (ATMPs) approved by the Medicines and Healthcare products Regulatory Agency (MHRA) is expected to rise significantly in the coming years. The MHRA has only approved an average of two ATMPs annually but the UK is leading the way with clinical trials in this area despite uncertainty remaining about how long the benefits of ATMPs might persist. The potential benefits are such that the investment is vital. AMTPs including cell and gene therapies offer hope for diseases previously considered untreatable. ATMPs are already being used to treat some rare conditions, including haemophilia and spinal muscular atrophy. In some cases, these therapies can transform people’s lives with just a single treatment. Therapies now in development are aiming to address conditions that affect larger patient populations, including certain types of dementia and Parkinson’s disease.