SB-728mR-T

SB-728mR-T is an autologous cell-based gene therapy consisting of CD4+ T cells that have been genetically modified ex vivo to disrupt the CCR5 gene. Developed by Sangamo Therapeutics in collaboration with the University of Pennsylvania, the therapy uses electroporation to deliver in vitro-transcribed mRNA encoding zinc finger nucleases (ZFNs) into the patient's own T cells. These ZFNs create a double-stranded break in the CCR5 gene, leading to a permanent knockout of the CCR5 coreceptor, which is essential for most HIV-1 strains to enter T cells. By re-infusing these modified cells, the therapy aims to establish a population of HIV-resistant immune cells in patients with HIV infection, potentially allowing for long-term viral control without the need for continuous antiretroviral therapy.

Other names

ZFN Modified CD4+ T CellsSB-728mR T-CellsSB728mR T-CellsSB 728mR T-Cells