10.30-11.30am, Mon 25th June 2018
Seminar Room 515, Level 5, 207 Bouverie Street, Carlton
Rodica Stan, PhD, Centre for Gene Therapy, City of Hope, Duarte, California, U.S.A.
We developed a novel therapeutic strategy for HIV-1 infection by engineering HIV-resistant immune cells via gene editing of autologous hematopoietic stem/progenitor cells (HSPC). A Zinc Finger Nuclease (ZFN) mRNA construct was designed to selectively disrupt the chemokine receptor 5 (CCR5), a co-factor required for HIV-1 infection of human cells, in HSPC. Thus far, we manufactured and infused eight ZFN-CCR5-modified HSPC products as autologous “transplants” after busulfan conditioning. Preliminary results are now available from the ongoing safety/feasibility clinical trial (NCT02500849). This is the first-in-human use of ZFN genome editing of HSPC, and preliminary data show that conditioning with busulfan and infusion of autologous CCR5-ZFN-modified HSPC are safe and well tolerated in HIV- infected subjects.