Type A hemophilia gene therapy SB-525 achieved positive results in Phase 1/2 clinical trials

recently, Sangamo
(http://and Pfizer announced the latest results of their joint development of the type A hemophilia gene therapy SB-525 in the phase 1/2 clinicaltrial(http://The results show that SB-525 can improve the level of coagulation factor VIII on dose dependence, and has good safety and toleranceFDA(http://also grant edifised SB-525 Regenerative Medicine Advanced Therapy (RMAT)Based on these positive results, the two companies plan to push SB-525 into registered clinical trials, and Pfizer will take over the late-stage clinical development and production of the therapyAbout SB-525
SB-525 is a genetically modified gene therapy that uses AAV6 virus vector stoking expression of human coagulation factor VIIIThe viral vector design of this therapy is designed to optimize the production efficiency of viral vectors and the expression of liver-specific clotting factor VIII proteinIn this Phase 1/2 clinical trial, 10 patients were treated with an incremental dose of SB-525, of which 4 received a maximum dose of 3e13 vg/kgThe four patients treated with the highest dose of SB-525 were followed up for 24, 19, 6, and 4 weeks, respectively The level of factor VIII in the first two patients receiving the highest dose of treatment (patients 7 and 8) has reached the normal area, while the rate of factor VIII in the last two patients (patients 9 and 10) has reached the normal area, of which the factor VIII level of patient 9 has reached the normal area after 7 weeks of treatment 　　All patients treated with the highest dose of SB-525 did not experience bleeding events after treatment and no factor VIII replacement therapy was required after the initial use of prophylactic factor VIII