UPenn team receives prestigious award for Sangamo sponsored HIV breakthrough

Sangamo Biosciences (SGMO) collaborators Carl June, Bruce Levine and Pablo Tebas are recipients of the prestigious Clinical Research Achievement Award for their personalized gene therapy work in HIV.

The Clinical Research Forum recognized the year’s 10 most outstanding research papers written by teams from across the nation at its 4th annual awards ceremony in Washington, DC, on April 16. The winning papers were chosen based on their degree of innovation from a pool of more than 50 nominations from 30 research and academic health centers nationwide.
The Penn team’s work, published in the New England Journal of Medicine in March 2014, was the first successful clinical test of any gene editing approach in humans. In the phase I study, they engineered the immune cells of 12 HIV positive patients to resist the HIV infection, by mimicking a naturally occurring mutation occuring in a very small number of people that renders their cells resistant to HIV infection. Researchers used a zinc finger nuclease technology to induce the genetic CCR5 mutation in patients’ T cells to lock out HIV. The modified T cells persisted in all patients, they found, and reduced viral loads in some taken off treatment entirely.
“This study shows that we can safely and effectively engineer an HIV patient’s own T cells to mimic a naturally occurring resistance to the virus, infuse those engineered cells, have them persist in the body, and potentially keep viral loads at bay without the use of drugs,” said June, the study’s senior author. “This reinforces our belief that modified T cells are the key that could eliminate the need for lifelong antiretroviral drug therapy and potentially lead to functionally curative approaches for HIV/AIDS.” For more on the study, read the press release here.
The Forum and its supporters believe these papers represent the best and brightest work in the field, and will lead to advancements in medicine that will change lives and patient outcomes worldwide.
The Forum advocates for increased respect for the field, which members hope will translate to sustained financial support from the National Institutes of Health, academia, foundations and other donors. “The better our clinical research, the greater our ability to diagnose, treat and prevent illness,” said Herbert Pardes, Co-Chairman of the Clinical Research Forum Board of Directors.