The study is an open-label Phase I clinical trial of the safety and tolerability of a single infusion of autologous CD4+ T cells genetically modified at the CCR5 gene by CCR5-specific ZFNs (SB-728-T). A total of 12 subjects with HIV will be enrolled in this trial in two treatment cohorts.
The first cohort to be treated comprises six subjects who have failed two or more highly active antiretroviral therapy regimen. The first three subjects in this cohort will be treated sequentially and monitored for the first 21-days post treatment before an additional subject is treated. After this period of evaluation and monitoring has passed successfully, the next three subjects will be treated.
The second cohort comprises six subjects who are responsive to their current therapy regimen who will be treated with CCR5-modified T-cells and undergo a structured therapy interruption or therapy ‘break’. The primary objective of the study is to evaluate the safety and tolerability of SB-728-T. In addition to safety monitoring, data will be collected on the expansion and persistence of ZFN-modified cells, CD4+ cell counts and viral load.
Edward Lanphier, president and CEO of Sangamo, said: Our zinc finger DNA-binding protein (ZFP) technology functions at the DNA level and, as this application demonstrates, enables us to address highly validated therapeutic targets that have proven difficult to drug at the protein and RNA levels. This trial is another important step in establishing our ZFP technology as a major new therapeutic product development platform.