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Biotech / Medical
'functional cure' of HIV: same tech could cure more diseases
An SI Board Since November 2012
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0 0 0 SGMO
Emcee:  SanDiegoBiotech Type:  Moderated
The odds of a major advance in the fight against HIV/AIDS have improved with the recent announcement by Sangamo Biosciences (Nasdaq: SGMO) that its gene therapy treatment has achieved a “functionalcure” for some HIV patients in its Phase 1 clinical trials.

Tim Brown, the famous “Berlin patient” and first person cured of HIV, appeared at the HIV Cure Forum in Palm Springs and West Hollywood, Calif., on Nov. 13 and 14. Brown’s visit and Sangamo’s clinical trial results draw attention to an unlikely corner of human gene therapy: beneficial mutations. In Sangamo’s case, its scientists generate mutations in the CCR5 gene in human CD4 T cells that confered resistance to HIV-1, the most common strain of the virus. Brown was cured when he received donated CD4 T cells with a naturally occurring CCR5 mutation.

Sangamo has touted results at the one-year clinical trial endpoint: CD4 T cell counts persisted a year after infusion in five of nine subjects to greater than 500 cells/mm3, the accepted threshold for initiation of HAART therapy.

“The initial results in a few patients look good and compelling,” said Liana Moussatos, an analyst with Wedbush Securities. “I’d like to see the same things in Phase 2 trials with more patients. Also, I’m interested in maintenance after treatment with modified CD4 cells: how durable are those cells over time?” (The number of genetically modified CD4 T cells slowly declines over time.)
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