Richmond research firm to announce HIV/AIDS clinical trial findings at national conference Saturday

Richmond-based Sangamo BioSciences, Inc. said Wednesday it is ready to take its HIV and AIDS research – which has the potential to someday provide a functional cure for HIV – to a national conference and announce the results of a clinical trial this weekend.

The research firm, located in Point Richmond, will attend the Interscience Conference on Antimicrobial Agents and Chemotherapy in Chicago on Saturday, where it will present findings on the application of gene modification as a treatment for HIV and AIDS.

Sangamo has been researching gene therapy as a new way to combat the virus since 2009, when the company began conducting studies on HIV-positive adults who were already taking standard antiretroviral medications, according to the website ClinicalTrials.gov.

The company’s approach to fighting the virus involves eliminating HIV receptors on the surface of virus-fighting white blood cells. The scientists then reintroduce the modified cells to the patient’s immune system so the HIV cells can no longer attach themselves to T-cells, which provide immune defense.

Sangamo founder and CEO Edward Lanphier said a naturally occurring mutation of the gene for the HIV receptor, which scientists had seen in some HIV and AIDS patients, led Sangamo to the premise of their research: If the gene becomes dysfunctional, the virus is rendered impotent.

“We’re replicating what’s already been shown in humans,” Lanphier said.

Sangamo’s therapeutic technique takes about six to eight weeks to prepare, according to Elizabeth Wolffe, the Sangamo director of corporate communications.

First, scientists draw the patient’s blood and separate the white blood cells. Then, they expose those cells for one to two days to a special protein that edits the white blood cells and suppresses the receptors to which the virus binds itself. After a period of evaluating and freezing the modified genes, they are then transfused back into the patient’s bloodstream, where Wolffe said they remain “forever, sort of cruising around.”

Present-day HIV and AIDS treatment generally involves patients taking a cocktail of antiretroviral medications that target the virus cells only, according to Sangamo’s website. Those treatments are prone to ineffectiveness because patients can lose T-cells at a gradual rate, making them vulnerable to other possible illnesses, such as pneumonia, Wolffe said.

Sangamo’s approach to treating HIV has a unique place in the AIDS-research industry. The Federal Drug Administration has yet to approve the sale of a gene therapy product, according to the Human Genome Project’s website. The technique can be used to treat several kinds of diseases, including herpes simplex, but the HGP notes patients can experience a short-lived effect, negative immune response, and difficulty with diseases that span several different genes.

Lanphier said the industry’s general reception of his company’s work has been extremely positive, despite the criticisms aimed at gene therapy. And Sunday’s post-conference announcement to shareholders and the public will reaffirm that notion, he said.

“When we presented the very first clinical results earlier this year, many of the comments were that this is the most exciting approach to a functional cure to HIV,” Lanphier said. “It’s not as a treatment for HIV, but as a potential functional cure for HIV.”

Wolffe said the company expects people to have a hard time understanding their approach, because it doesn’t involve a daily dosage of pills. But eventually, she said, people will come to see genetic modification as the way to fight such diseases, despite the practice’s spotty history.

Wolffe said Sangamo remains positive that the best way to fight diseases that cause gene defects is to modify existing genes to resist the virus.

“I mean, (with) genetic engineering, people get all up in arms over this when it’s about food,” Wolffe said. “But when it comes to saving a child’s life, they’re a little bit more interested.”

Lanphier said Sunday’s statement will describe the safety of the modified cells and the effect of the cells on the virus. He was unable to discuss the results, due to the upcoming conference and shareholder priority.