A Possible “Functional Cure” for HIV

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A biotech company is set to begin human trials on a groundbreaking new HIV treatment that could offer what they believe is a breakthrough cure for the virus.

The Food and Drug Administration (FDA) gave approval to Excision Biotherapeutics to use its innovative HIV treatment in Phase I/II human trials, according to a press release from the company. Excision Biotherapeutics specifically utilizes CRISPR gene-editing technology in its research against viruses. The Nobel Prize-winning technologyallows for the modification of human DNA and is considered revolutionary for its treatment of many diseases.

Excision’s HIV treatment, known as EBT-101, was developed in partnership with researchers at Temple University in Philadelphia and involves cutting out several pieces of the HIV genome, which the company believes will render it incapable of mutating inside the body.

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

EBT-101 has already proved effective in removing HIV proviral DNA — a latent form of the virus that inserts itself into human cells and replicates along with them — from both lab-isolated human cells and non-human primates, according to Excision. Researchers believe it, thus, has the potential to “functionally cure” HIV in humans.

A “functional cure” means there could still be small amounts of the HIV genome present in the body but not enough for an affected individual to test positive for HIV, as Dornbusch explained to Philadelphia magazine. Completely eliminating the virus from the body is referred to as a “sterilizing cure,” he said, adding: “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.”

EBT-101 is meant to be a one-time treatment with the goal of eliminating patients’ need to utilize antiretroviral therapies (ART), as Dornbusch explained. ART involves a combination of different HIV medications that decreases the amount of virus in the body and prevents it from attacking the immune system, per the National Institutes of Health (HIV). ART can even render a person’s HIV infection undetectable while they are taking the medications, which makes it almost impossible for them to transmit the disease to someone else.

While ART can significantly suppress HIV, it requires patients to stay on daily medications for life. “They require life-long treatment, cause side effects, and do not provide the possibility of a functional cure,” Dornbusch said in the press release.

The trials will include people who already have their HIV controlled using ART. Participants will receive one dose of EBT-101 and will subsequently remain on ART for three months, after which they will stop ART and be monitored for the return of the virus. The company will also be testing EBT-101 at various dose levels.

The news comes on the heels of Moderna announcing in August that it was starting human trials of its own for a vaccine against HIV. The company is using the same innovative mRNA technology it used to produce its COVID-19 vaccine. Many are hoping this new technology is the key to stopping a virus that has long eluded the antibodies produced by vaccines due to its ability to mutate rapidly.

The World Health Organization (WHO) still considers HIV to be a global epidemic. In 2020, there were 37.7 million people living with HIV worldwide, and 680,000 deaths related to the virus, according to WHO. Since the epidemic began, 79.3 million people have been infected and 36.3 million people have died. HIV is most heavily concentrated in Africa, where one in every 25 adults currently lives with it.

HIV also has massively outsized effects on transgender women. A 2014 report from the Joint United Nations Program on HIV/AIDS (UNAIDS) found that transgender women are 49 times more likely to be living with HIV than other adults. Globally, the survey also found that 19% of trans women are currently living with HIV.

The virus continues to disproportionately affect gay and bisexual men as well, especially those who are Black or Latinx, according to the Centers for Disase Control(CDC). In 2016, the CDC reported that an estimated one in two Black men who have sex with men (MSM) will be diagnosed with HIV during their lifetimes. The same is true for one in four Latinx MSMs.


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