Sangamo BioSciences, Inc.
SGMO announced new data from its program to develop a 'functional
cure' for HIV/AIDS in two presentations at the 20^th Conference on
Retroviruses and Opportunistic Infections (CROI), held in Atlanta from March 3
to 6, 2013.
The first presentation described data from the SB-728-T Phase 1 study
(SB-728-902, Cohorts 1-3) demonstrating that SB-728-T treatment of
HIV-infected subjects leads to durable reconstitution of the immune system
driven by increases in total CD4+ central memory T-cells (T[CM]) and
CCR5-protected T[CM]. T[CM] are long-lived, self-renewing cells that have the
ability to remember and react against foreign antigens including HIV. The
data also showed that certain cell surface marker and gene expression profiles
may predict which patients will likely respond best to SB-728-T treatment.
"These important data extend our understanding of why SB-728-T treatment
improves the immune system as well as the conditions required for optimal
engraftment of ZFN-modified T-cells," said Dale Ando, M.D., Sangamo's vice
president of therapeutic development and chief medical officer. "They confirm
that SB-728-T meets the key immunologic requirements for immune reconstitution
in HIV-infected individuals. In addition, analysis of cell surface marker and
gene expression profiles of immune system cells in subjects who show superior
responses to treatment in terms of increased T-cell counts provides us with
important indicators that will aid us in the optimization of our clinical
trials."
"The ability of SB-728-T to durably reconstitute the immune system in
HIV-infected subjects after a single treatment has never been observed before
with any other therapeutic approach," commented Rafick-Pierre Sekaly, Ph.D.,
Co-Director & Chief Scientific Officer, The Vaccine & Gene Therapy Institute
of Florida (VGTI Florida), whose laboratory carried out the analysis.
"Improvement in the overall health of the immune system of HIV-infected
individuals, as demonstrated by treatment with SB-728-T, is a key step along
the path to developing an immunologic approach to controlling and potentially
eliminating the virus. We have analyzed the cells that constitute this
unprecedented elevation of total CD4+ cell counts, extending our previous
observations that the increase is primarily due to durable expansion of the
central memory T-cells. Importantly, the level of ZFN-dependent CCR5 gene
disruption is sustained in these cells, which is critical for the durable
success of this approach."
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