Syndicate content

Archive - Mar 7, 2014

Date

Sangamo Presents Additional Data in Support of Enzyme-Based Functional Cure of HIV/AIDS

Sangamo BioSciences, Inc. (Nasdaq: SGMO), announced on March 6, 2014 the presentation of data from its SB-728-T program to develop a “functional cure” for HIV/AIDS at the Conference on Retroviruses and Opportunistic Infections (CROI 2014). The conference was held in Boston from March 3 to 6, 2014. Data from an earlier Phase 1 clinical study in this program were also published in the March 6, 2014 issue of the New England Journal of Medicine(NEJM) (see BioQuick story below). "The achievement of over 7 months of ongoing functional control of viral load without antiretroviral therapy and the progress that we are making in understanding how to best deploy this novel therapy are very exciting," commented Gary Blick, M.D., AAHIVS, Medical & Research Director, CIRCLE CARE Center, who presented the data at CROI and is an investigator on both studies that were reported at the meeting. "The data that have been generated over the course of the clinical investigation of SB-728-T demonstrate immune reconstitution, enhanced survival of the zinc finger nuclease-modified T-cells in the presence of the virus, and associated reductions in viral load and the levels of viral reservoir, all of which are necessary to provide functional control of the virus." At CROI, data were reported from a Phase 1 /2 clinical trial, SB-728-1101, designed to evaluate the effect of increasing doses of Cytoxan preconditioning as a method to increase the numbers of circulating T-cells, including cells that were zinc finger nuclease (ZFN) modified at the CCR5 gene (SB-728-T). The data demonstrate that increasing doses of Cytoxan preconditioning prior to a single infusion of SB-728-T led to a dose-dependent increase in both engraftment of CCR5-modified cells and notable increases in total CD4 cells above the baseline.

Treatment with Sangamo's ZFN-Modified T-Cells Provides Functional Control of HIV without Antiretroviral Drugs

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced on March 5, 2014 the publication in the March 6, 2014 issue of the New England Journal of Medicine of the first clinical study of Sangamo's proprietary zinc finger nuclease (ZFN)-based genome editing technology in humans. Data from the study, carried out in HIV-positive subjects, demonstrate that the T-cell genome can be safely engineered to mimic a naturally occurring mutation that provides resistance to HIV infection. ZFN-modified T-cells are well tolerated when reinfused and treatment is associated with decreased viral loads (VLs) in several subjects who were taken off their antiretroviral therapy (ART), including one whose viral load became undetectable. The study demonstrates the feasibility of this novel genome editing approach to achieve functional control of HIV. Additional data on the ongoing SB-728-T ongoing clinical trials in HIV will be presented at the Conference on Retroviruses and Opportunistic Infections (CROI 2014), which is taking place in Boston, March 3-6, 2014. "We have used Sangamo's ZFN technology to safely genetically engineer an HIV-infected individual's own T-cells and to make those cells resistant to infection by the virus," said Carl June, M.D., Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine at the Perelman School of Medicine at the University of Pennsylvania, and a senior author of the paper. "This study demonstrates that ZFN-modified cells can be safely administered back to the individual; are able to persist and circulate throughout the body to key reservoirs of HIV infection; and show preferential survival over unmodified cells when antiviral drugs are withdrawn, potentially keeping the virus under control without the use of drugs.