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Sangamo Biosciences (SGMO) HIV Therapy Findings

SGMO’s experimental gene/cell therapy was used safely in 6 patients infected with HIV in the first trial of a new approach aimed at blocking the virus so patients won’t need antiviral drugs.

Findings from the trial were presented 2/28/11 in Boston at the Conference on Retroviruses and Opportunistic Infections, a meeting of infectious disease doctors.

  • After a year, 5 of the 6 had “significant and largely sustained” increases in the number of infection-fighting t- cells in their system. The patients kept taking drugs to suppress the virus during the study because the research first must assess whether the therapy is safe;
  • The treatment modifies patient’s t-cells to inactivate a protein called CCR5 that HIV uses to enter the cells;
  • The theory is that without the entry point, HIV won’t be able to weaken the immune system and patients won’t get sick. If the therapy cuts HIV levels in patients who aren’t taking antiviral drugs, it may gain approval by late 2013. Big … if!

SGMO is best known for its zinc finger technology that mediates genes, said its cell therapy, called SB-728, and targets a patient’s own CCR5 gene by removing, essentially, the doorknob that HIV turns to gain access to infection-fighting white blood cells. (HWM and RLeuty)

The Bottom Line: When that data comes at the end of this year, we should have an idea whether the efficacy is durable enough and potent enough. However, any potential side effects aren’t yet fully known! SB-728 is a “functional cure” that creates an army of cells that can mount a response to the virus. It has possible drawbacks: it is a cell therapy to be given in a doctor’s office, rather than patients simply popping antiretroviral pills like those offered by Gilead Sciences. SGMO closed down $0.23 to $8.27 or (-2.71%).