First person to ever be treated with CRISPR-based HIV gene therapy

An unprecedented gene editing treatment that could lead to curing HIV.
Brittney Grimes
HIV virus.

A new gene-editing therapy for human immunodeficiency virus type 1 (HIV) is being tested on individuals. The clinical trial, led by researchers at Lewis Katz School of Medicine at Temple University and Excision BioTherapeutics, a company creating CRISPR-based therapies to ultimately cure viral infectious diseases, is unprecedented and the goal is to evaluate the safety measures of EBT-101, a unique gene-editing treatment.

Gene-editing therapy

The researchers based their trial on gene-editing therapy known as CRISPR. It is the first time such therapy has targeted an infectious disease and has been given to a patient while being evaluated for innovative treatment. Currently in the clinical stage, it’s designed to eventually cure HIV infections after a single intravenous infusion. This novel approach by the team is being used to collect data on the gene-editing therapy.

Observation and evaluation

The person who received the first single-dose intravenous infusion EBT-101 therapy is being observed and evaluated for rebound of the HIV virus. Since this treatment has never been done before, researchers want to make sure the treatment stops the virus from replicating itself in the body. The research team is hoping that the individual will no longer need to use the antiretroviral therapy, which is currently the standard in HIV treatment.

Launching the EBT-101 phase 1/2 clinical trial

The trial was launched by Kamel Khalili, PhD, Laura H. Carnell, professor and chair of the Department of Microbiology, Immunology, and Inflammation, director of the Center for Neurovirology and Gene Editing and director of the Comprehensive NeuroAIDS Center, and Tricia H. Burdo, PhD, professor and vice chair of the Department of Microbiology, Immunology, and Inflammation at the Katz School of Medicine.

Dr. Khalili mentioned that there are still no curative treatments for HIV after more than 40 years since the virus was discovered. Nearly 40 million people worldwide suffer from HIV. He said “EBT-101 can potentially address long-standing unmet needs of individuals living with HIV/AIDS by removing viral DNA from their cells, thereby eradicating infection.”

Details of the trial

The trial is a study meant to evaluate the safety and efficacy of EBT-101 in approximately nine participants living with HIV-1. These individuals undergoing the study are suppressed on antiretroviral therapy. Its goal is to assess the tolerability of a single dose of EBT-101 in participants and their response to it. There was an initial 48-week follow up period for the trial, with an intended long-term follow up.

Closer to a cure

The research team realized what must be done to move closer to curing HIV. They said it is essential to remove the HIV DNA from cells to find a cure. The virus resides in tissue reservoirs for years and avoids the immune system, making it difficult for antiretroviral therapy to work effectively. “The official start of the Phase 1/2 clinical trials for EBT-101 brings us one extremely significant step closer to creating a potential cure for HIV/AIDS,” said Dr. Amy J. Goldberg, interim dean of Katz School of Medicine.

The inaugural assessment of Phase 1/2 clinical trials for EBT-101 serves as a hopeful indicator to the team who believe they are closer to creating this cure for HIV.