A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Subjects with Suboptimal CD4 Levels on cART
Autologous hematopoietic stem cells gene edited ex vivo to eliminate expression of HIV entry co-receptor CCR5. Cells carrying disrupted CCR5 provide a renewable, long-lasting source of HIV-1 resistant immune cells.
Single arm, non-randomised study.
Safety. Efficacy - engraftment.