Sangamo Therapeutics declared that the Medicines and Healthcare Products Regulatory Agency of the United Kingdom has extended Clinical Trial Authorization for enlisting subjects into the ongoing Phase 1/2 Clinical Trial assessing SB-FIX in vivo genome editing treatment for hemophilia B The CTA permits initiation of Europe’s first in vivo genome editing study.
The CTA permits evaluation of SB-FIX in both adults and adolescents. Once preliminary safety and efficacy have been demonstrated in the ongoing SB-FIX Phase 1/2 clinical trial in adults (18 years or older), Sangamo may then begin enrolling adolescents (12 – 17 years of age) into the study.
Edward Conner, M.D., Sangamo’s chief medical officer. “In vivo genome editing aims to provide a life-long therapeutic solution for certain genetic diseases. We believe the greatest value for this approach is in the treatment of children, and our goal with this study is to accumulate safety and efficacy data supporting progression of clinical trials into younger patient populations.”
To restrict editing to liver cells, the ZFNs and the corrective gene are delivered in a single intravenous infusion using AAV vectors that target the liver. The ZFNs enter the cells as inactive DNA instructions in a format designed only for liver cells to unlock. Once “unlocked”, the ZFNs then identify, bind to and cut the DNA in a specific location within the albumin gene. Using the cells’ natural DNA repair processes, liver cells can then insert the corrective gene for Factor IX at that precise location.