A Potential One-Dose Treatment to 'Functionally Cure' HIV Will Be Tested in Human Trials

Julie Mazziotta
·2 min read
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HIV treatment
HIV treatment

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A potential treatment for HIV that would "functionally cure" the virus will soon begin human trials.

In September, the Food and Drug Administration granted Excision Biotherapeutics approval to start testing their HIV treatment, known as EBT-101, in volunteers with HIV. The technology, which was discovered at Temple University in Philadelphia, uses gene editing to cut out pieces of human DNA, which researchers hope will work on the HIV genome to stop it from mutating in the body.

"If you just make a single cut, the virus can mutate around it," Excision CEO Daniel Dornbusch explained to Fierce Biotech. "We make multiple cuts to deactivate the viral genome."

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The treatment would come in the form of a single dose given by IV over one to two hours. Currently, there is no cure for HIV — the most effective way to manage the virus is with antiretroviral therapy (ART) that requires taking multiple medications every day. In Excision's trial, participants will continue taking ART for three months after getting their dose of EBT-101, and then go off the drug, Philadelphia magazine reported.

Researchers have already found EBT-101 to be effective in cutting out portions of the HIV genome in prior trials in non-human primates and lab-isolated human cells.

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They hope that the first human trial will establish that the treatment is safe and effective.

"The goal, of course, is to find the first therapeutic to create functional cures for HIV," Dornbusch told Philadelphia, adding that "the term 'functional cure' is an important distinction, as there will be no way to determine if EBT-101 will remove every viral genome from an individual, which is called a 'sterilizing cure.' "

"However, sterilizing cures are not necessary, as the goal of the therapy will be for individuals to remain HIV negative by RNA testing, maintain normal levels of immune cells, and cease taking antiretroviral treatment — achieving a functional cure," he explained.

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Along with Excision's treatment, Moderna and Johnson & Johnson are also currently running human trials on HIV vaccines to prevent the virus. Johnson & Johnson's vaccine, though, failed in its first round of trials, missing the goal of reducing the risk infection by half and instead providing around 25.2% of protection, StatNews reported.