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FDA Okays One-Time HIV Treatment Clinical Trial

HIV released from cell

Illustration of HIV released from the surface of an infected cell (Bette Korber, Los Alamos National Lab)

15 Sept. 2021. A company developing a one-time treatment for HIV infections using the gene-editing technique Crispr says it received FDA clearance to begin a clinical trial. Excision BioTherapeutics Inc. in San Francisco says the Food and Drug Administration accepted the company’s investigational new drug application for its HIV treatment candidate code-named EBT-101.

Excision Bio employs gene editing to remove large sections of DNA from HIV viruses, taking out the viruses’ ability to replicate and escape. For these edits, the company uses Crispr, short for clustered regularly interspaced short palindromic repeats, a process based on bacterial defense mechanisms that use RNA to identify, monitor, and edit targeted locations in DNA. Excision Bio says one Crispr treatment disables human immunodeficiency type 1 viruses, or HIV-1 viruses, preventing their replication, thus stopping further infection.

According to Centers for Disease Control and Prevention, 36,801 Americans were diagnosed with HIV infections in 2019, a decrease of nine percent from 2015. About seven in 10 (69%) of those new cases were gay or bisexual men, with nearly a quarter (23%) heterosexual, and seven percent people who inject drugs. Globally, according to World Health Organization, nearly 38 million people were living with HIV in 2020, with 1.5 million new cases reported.

Functional cure for HIV

The standard of care for HIV infections is antiretorviral therapy, which suppresses and controls, but does not eliminate the virus. Antiretorviral medications must be taken periodically, usually each day, to prevent viral loads from rebounding, since residual levels of HIV remain in infected individuals. Excision Bio says EBT101, its one-time treatment candidate, is a functional cure for HIV.

Excision Bio says EBT-101 works in the body using Crispr to remove proviral DNA, the DNA that enters and integrates with host cells. The company says EBT-101 is guided to the HIV target with RNA, targeting three sites in the HIV genome. The Crispr edits remove the virus’s ability to replicate and minimizes its chance to escape. As reported by Science & Enterprise in December 2020, researchers from Temple University in Philadelphia used Crispr to remove genes gained from infections similar to HIV in lab monkeys. Excision Bio licenses the technology from the lab of Kamel Khalili, professor of virology and neurology at Temple and scientific founder of the company.

“EBT-101 has demonstrated removal of proviral DNA in multiple animal models,” says Excision Bio chief medical officer Lisa Danzig in a company statement released through Globe Newswire, “and offers an opportunity for individuals living with HIV to potentially cease life-long therapies.”

An investigational new drug application officially seeks permission from FDA to ship new drug candidates across state lines to clinical trial sites, in effect requesting permission for a clinical trial. Excision Bio plans a combined early- and mid-stage clinical trial testing the safety, tolerability, and efficacy of EBT-101 in individuals living with HIV, to begin later this year.

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