Saturday, July 18, 2015

Comprehensive Report on the HIV Pipeline

This 100 page comprehensive report details the state of HIV research in 2015.
Here is the mention of Sangamo's work:
A development in gene therapy that made the news earlier this year was the approval by the FDA of a clinical trial involving genetic modification of stem cells. The project involves collaboration between researchers from City of Hope Medical Center in Los Angeles, the Keck School of Medicine at the University of Southern California, and Sangamo BioSciences, with support from the California Institute for Regenerative Medicine (CIRM). Stem cells will be extracted from individuals, treated with Sangamo’s zinc finger nuclease technology to disrupt the CCR5 gene, and then reinfused with the aim of generating CCR5-negative immune cells resistant to HIV. According to a press release from CIRM, the initial study population will be HIV-positive individuals responding poorly to ART.88 Although some of the headlines described the approach as a “functional cure”89 or “potential cure,”90 this is in fact only an exploratory study, and it is wildly premature to suggest that it could be curative; previous trials involving genetic modification of stem cells have generated only low levels of gene-modified CD4+ T cells.91

Research continues into the use of the Sangamo BioSciences technology to genetically modify CD4+ T cells ex vivo. The CD4+ T cells are extracted from HIV-positive individuals, exposed to the zinc finger nuclease to disrupt the CCR5 gene, then expanded and reinfused. In studies published and presented to date,94,95 an adenovirus vector was used to deliver the zinc finger nuclease into the CD4+ T cells during the process. The company is now testing a different and potentially more efficient approach in which messenger RNA encoding the zinc finger nuclease is used instead of an adenovirus vector. Over the past year, two clinical trials have opened that will deliver CD4+ T cells modified with this method; both are using transient administration of cyclophosphamide prior to the infusion to enhance the engraftment of the altered cells.

Report:
http://www.pipelinereport.org/2015/cure-and-immune-based-and-gene-therapies
PDF:
http://www.pipelinereport.org/sites/g/files/g575521/f/201507/2015%20Pipeline%20Report%20Full.pdf

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