New Sangamo Genetic Therapy Boosts CD4s, Reduces Reservoir

To do it, researchers removed immune cells from the participants and genetically engineered them knock out genes that express the CCR5 coreceptor onto which HIV latches in order to infect the cell, thus yielding HIV-resistant CD4s without the coreceptor. For more info, click here.

“According to Sangamo Biosciences SGMO +4.65% CEO Ed Lanphier, the biotech group’s gene-therapy technology could end up disrupting the broader pharmaceutical market by providing ‘cures’ for diseases caused by certain genetic malfunctions, thereby freeing patients of cumbersome and often expensive drug regimens.

The company’s lead product, a cellular therapy called SB-728, has already shown considerable promise in treating HIV/AIDS. Sangamo is currently running Phase II trials to see if it can help rebuild the immune systems of HIV and AIDS patients, which would conceivably allow them to go off their anti-viral drug regimens. The company plans to unveil preliminary Phase II data during the first half of 2013.

'Our goal is not just to treat but to create a ‘functional cure’ for HIV,” said Lanphier, during a recent interview with MarketWatch.' ”