Clinical Development of Advanced Gene Therapies for Hemophilia

Time: 3:50 pm
day: Day One


• Over the next 5-10 years the management and treatment of hemophilia will experience a revolution. A single intervention of gene therapy will become the treatment of choice for many patients
• Standard gene therapy can achieve a permanent and successful amelioration of the bleeding episodes in adults with hemophilia, eliminating the need for prophylactic replacement therapy
• However, the pediatric population requires gene editing to fully integrate the replacement gene into the cell. This approach maintains the production of the missing coagulation factor despite rapid turnover of liver cells