Spark hemophilia gene therapy SPK-8011 remission rate of 97%

haemophilia A is a hereditary blood clotting disease that can cause uncontrolled bleedingIn the United States, about 20,000 people are diagnosed with haemophilia A, and there are 320,000 haemophiliacs worldwidetoday, Spark Sparks announced that 12 haemophiliacs with a type A haemophilia had a 97% remission rate after receiving gene therapy SPK-8011SPK-8011Spark developed sPK-8011 is a new bioengineered gland-related virus (AAV) vector that uses the Spark200 crust and contains the human-derived factor VIII gene optimized by the cocospandeIt can more effectively transpose liver cells and express the cocosite-optimized B-domain missing factor VIII gene, endogenous production factor VIII, used to treat haemophilia Athe study
12 patients received different doses of one-time SPK-8011 in a phase 1/2 clinicaltrial(of which) was treated with different dosesOf these, 2 cases were 5x10?11 vector genomics (vg)/weight (kg), 3 cases were 1x10?12 vg/kg, and 7 cases were 2x10?12 vg/kgin all three doses, the annual bleeding rate (ABR) decreased by 97% and the annual infusion rate (AIR) by 97% from 4 weeks after administrationThe follow-up period of the first two participants lasted more than one year and lasted 66 weeks after the activity level of factor VIII reached a stable periodIn follow-up 12-30 weeks after administration, the factor VIII levels were between 16% and 49% in 5 patients who received a dose of 2x10 to 12vg/kgBased on the average factor VIII level after 12 weeks of administration, the average factor VIII activity in these five patients was 30%Their overall ABR was reduced by 100% and their overall AIR by 100%in addition, there is evidence that the average factor VIII activity level in the three dose groups increased in dose dependence