Gene therapy represents a groundbreaking approach in the field of medicine, aiming to treat or prevent diseases by modifying the genetic material within a patient’s cells. The concept involves introducing, removing, or altering genetic sequences to correct or mitigate the effects of disease- causing mutations. Over the past few decades, advancements in molecular biology and genetic engineering have significantly propelled the development and application of gene therapy. Here, we provide our insights on progressing a novel gene therapy into clinical trials and various options that need to be considered along the way.

Gene therapy encompasses various strategies, including in vivo and ex vivo methods. In vivo gene therapy involves directly delivering genetic material into a patient’s body, while ex vivo techniques involve modifying cells outside the body before reintroducing them to the patient. Viral vectors, such as adenoviruses and lentiviruses, are commonly used to deliver therapeutic genes due to their efficiency in transducing cells. Non-viral methods, including liposomes and nanoparticles, offer alternative delivery mechanisms with potentially fewer immunogenic issues.

Ensuring long-term efficacy, avoiding immune responses, and addressing ethical and regulatory concerns are some of the key challenges that still remain. However, ongoing research and clinical trials continue to refine these therapies, offering hope for curing previously untreatable diseases and revolutionising modern medicine.