Promising Results Support Continued Development of AAV-Based Human Factor VIII Gene Therapy
August 20, 2018
August 20, 2018
NEW ROCHELLE, New York, Aug. 20 -- Mary Ann Liebert issued the following news release:
Gene therapy using an optimized adeno-associated virus (AAV) to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks in some animals. These promising data combined with evidence that the optimized vector AAVhu37 is suitable for manufacturing and purification at scale, suggests that AAVhu37-base . . .
Gene therapy using an optimized adeno-associated virus (AAV) to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks in some animals. These promising data combined with evidence that the optimized vector AAVhu37 is suitable for manufacturing and purification at scale, suggests that AAVhu37-base . . .