Hemophilia Gene Therapy: Approaching the First Licensed Product

The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product. While recent late phase clinical trials of hemophilia gene therapy have shown promising results, there remain a number of issues that require further attention with regard to both efficacy and safety of this therapeutic approach. In this review, we present information relating to the current status of the field and focus attention on the unanswered questions for hemophilia gene therapy and the future challenges that need to be overcome to enable the widespread application of this treatment paradigm.

Automated summary

The clinical potential of hemophilia gene therapy has been pursued for the past 30 years, with expectations of achieving this goal in the near future. While recent late phase clinical trials have shown promising results, there are still issues regarding efficacy and safety that need further attention. Overcoming these challenges is crucial for the widespread application of this treatment paradigm.