Advances and challenges with gene therapy for haemophilia

Abstract

Summary:
 Haemophilia rare inherited bleeding disorder caused by deficiency in clotting factor that affects around 6,000 people in the UK. Patients with haemophilia require regular prophylactic infusions of the deficient clotting factor intravenously, which causes a large burden on patients and their families. Gene therapy holds promise as a potential cure for Haemophilia. This article critically evaluates the existing evidence for gene therapy in Haemophilia.
 Relevance:
 There are several gene therapies in development for inherited conditions such as Haemophilia. Valoctocogene Roxaparvovec, a gene therapy for Haemophilia, is currently being assessed by regulatory authorities worldwide. Medical students and practicing physicians must be aware of the current evidence base for gene therapy for Haemophilia as well as the limitations thereof to help patients make informed decisions on whether gene therapy is a suitable treatment option for them. 
 Take Home Messages:
 The current evidence shows that gene therapy is likely to be effective in increasing clotting factor levels and reducing bleeds for at least 3 years. However, long-term data is limited, and therefore, the durability of the treatment is unknown. Additionally, there is limited evidence on the safety and efficacy of gene therapy in patients with pre-existing antibodies to the viral vector, patients with inhibitors to the clotting factor, and children.