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CRISPR-Based HIV Gene Therapy Administered To First Human Patient

The one-of-a-kind gene editing treatment has the potential to change the future of HIV therapeutics.

Johannes Van Zijl

Johannes Van Zijl

Johannes has a MSci in Neuroscience from King’s College London and serves as the Managing Director at IFLScience.

Managing Director

The human immunodeficiency viruses (HIV) circulating in the blood
The first patient to have received the new CRISPR-Based HIV gene therapy is currently under medical observation. Image Credit: Spectral-Design/

In a clinical trial, the first patient has received a single dose of a new human immunodeficiency virus (HIV) gene editing therapy, researchers at the Lewis Katz School of Medicine at Temple University and Excision BioTherapeutics, Inc have reported.

In a collaborative effort, the researchers are currently running a phase 1/2 clinical trial to evaluate the safety and efficacy of their therapy, called EBT-101, which is based on gene editing technology known as CRISPR


“Nearly 40 million people worldwide suffer from the effects of HIV, and more than 40 years after the discovery of HIV/AIDS, there still are no curative treatments,” said Professor Kamel Khalili, who helped lead the trial, in a statement

“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.”

When HIV infects, it takes long-term hold and hides from the immune system in cells, compromising the patient's immunity over time and eventually leading to the development of AIDS – the progressive failure of a patient's immune system. There is currently no cure for HIV, but various treatments and medications can help manage the infection and slow or prevent the progression of the disease.

The therapy currently being trialed, which uses CRISPR gene editing to remove HIV viral DNA from infected cells, is a major step toward finding a therapeutic cure.


“We are well-positioned to collect key data that will enable our efforts to translate the success this approach has shown in animal models to human clinical trial participants,” Khalili added. “We look forward to investigating this hypothesis through the EBT-101 clinical program and are pleased that the EBT-101 Phase 1/2 trial is proceeding as planned.”

The first patient to receive a single dose of EBT-101 in the current trial is currently under medical supervision and will soon be assessed to see if there is any viral rebound and whether the single curative treatment worked as planned. This will be a deciding factor in whether or not the patient is able to stop their current antiretroviral therapy in the future.

“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 the Katz School of Medicine. “This amazing milestone speaks to the exceptional research acumen and scientific knowledge of Drs. Khalili and Burdo and their teams – outstanding members of the Temple family.”

For further details, you can view the clinical trial information at ClinicalTrials and follow its progress.


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  • medicine,

  • hiv,

  • gene therapy,

  • gene editing,