Sangamo and the California Stem Cell Agency: Good News for Both

Sangamo Biosciences is one of the California stem cell agency award recipients that is again surfacing in the news in a way that could ultimately burnish the reputation of the $3 billion research enterprise.

Sangamo, a publicly traded company based in Richmond, Ca., popped up in a story in the New York Times dealing with the search for a cure for AIDS involving researchers at the University of Pennsylvania.

Sangamo is also involved in another AIDS research project financed by the stem cell agency at the City of Hope and USC.  The company produces a gene-editing tool and its stock price is soaring. 

The Times story by Denise Grady reported on the phase one clinical trial at the University of Pennsylvania. She wrote,

“The idea of genetically altering people’s cells to make them resist the virus that causes AIDS may seem like a pipe dream, but a new report suggests it can be done.

“The research involves the first use in humans of 'gene editing,' a treatment that zeros in on a particular gene and disables it.”

We asked the stem cell agency how the trial fits with the work financed by CIRM. Kevin McCormack, senior director for communications, replied,

“On first glance it looks as if this works with the CD4 t-cells while the work we are financing focuses on a similar approach but perhaps the best explanation of the difference comes from this section of the original application from John Zaia's team at City of Hope that is partnering with Sangamo. 

“'Recently, ZFNs have been shown to inactivate CCR5 in primary human CD4 T cells, allowing them to preferentially survive and expand in the presence of HIV. A human clinical trial evaluating this approach is on-going, in which patient T cells are re-infused after ZFN-treatment to block CCR5 expression and possibly provide an HIV-resistant reservoir of CD4 T cells (THIS IS THE PIECE IN THE NY TIMES ARTICLE). The CIRM Disease Team proposes an approach to modify a patient’s own HSC to circumvent the need to find matched donors that carry the Δ32 CCR5 mutation and yet provide a renewable and long-lasting source of HIV-resistant cells.'”

Sangamo's stock has climbed dramatically this year. On Jan. 9,  based on news of an investment in the company by Biogen Idec, Sangamo opened at $15.70, rose to $19.12 and closed at $18.88. The volume of shares traded was 10.4 million. The average volume of trading over has been well less than 1 million for most of the last 12 months.

Today the stock closed at $22.96, up from $18.92 at the Tuesday close, the day before the clinical trial news surfaced. Volume hit 11 million shares. Sangamo's 52-week low is $6.86.