Providing new hope for rare disease using non-viral gene therapy

Gene therapy represents one of the most promising therapeutic approaches to treat a range of devastating genetic disorders – with the goal of delivering curative therapies to patients, particularly in paediatrics where patients face a significant burden of disease and often a high risk of death. Conventional treatments for these rare diseases, where they exist, often involve chronic maintenance therapy to preserve function as long as possible or highly intensive procedures such as transplantation to reconstitute failing biological processes. Yet, the field of gene therapy has faced a number of challenges and clinical setbacks in recent years, including a lack of durable outcomes and severe immune responses to therapies using viral vectors to treat genetic diseases.¹