A breakthrough gene editing therapy for the treatment of human immunodeficiency virus type 1 (HIV) infection, which is currently undergoing clinical trials, has been applied to a person with HIV for the first time. This trial was designed to evaluate the safety and effectiveness of EBT-101. As a unique gene editing therapy, EBT-101 will have the potential to change the future of AIDS treatment.
AIDS (AIDS) is caused by infection with HIV (HIV) and is a virus that can attack the human immune system. Currently, there is still a lack of effective drugs to cure HIV infection worldwide. The current treatment goal is to maximize and lasting inhibit viral replication in patients' bodies, enable patients to rebuild their immune function and maintain their immune function, while reducing the incidence and mortality of HIV infection and non-AIDS-related diseases.
EBT-101 Therapy
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His virus has lived in tissue repositories for a long time, avoiding the immune system and antiretroviral treatment, and can survive in tissue hosts for several years. EBT-101 is a unique, clinical-stage in vivo CRISPR-based therapeutic agent designed to cure HIV infection after a single intravenous infusion. EBT-101 uses adeno-associated virus (AAV) to deliver CRISPR-Cas9 for gene editing , and uses multiple single-guidance RNAs targeting different regions of the HIV genome to cut HIV DNA from the genome of human cells, thereby eliminating the chance of virus escape. "Nearly 40 million people worldwide suffer from HIV and there is still no curative treatment after more than 40 years of HIV/AIDS discovery. EBT-101 can eradicate infections by removing viral DNA from cells, thus eradicating infections by removing viral DNA from cells." Removal of HIV DNA from cell genome is crucial for treatment.
EBT-101 Phase 1/2 clinical trial
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EBT-101 Phase 1/2 clinical trial is an open-label, multi-center single-increment dose study. The first patient to receive a single dose of intravenous EBT-101 treatment is currently under observation and will be evaluated soon if the antiretroviral treatment is qualified. If a one-time cure treatment works as expected, patients will no longer need antiretroviral therapy —the current standard treatment for HIV infection, the researchers said. The launch of the phase 1/2 clinical trial was based on research led by Dr. Kamel Khalili, Professor Laura H. Carnell, and Director of the Department of Immunology and Inflammation; Director of the Center for Neurovirology and Gene Editing; Director of the Center for Integrated Neuro AIDS, and Professor and Associate Director of the Department of Microbiology, Immunology and Inflammation at the Katz Medical School.
Khalili and colleagues at Temper University were the first to develop EBT-101 based on CRISPR gene editing technology, while Dr. Burdo and her team played a key role in advancing EBT-101 clinical trials through EBT-101 research on non-human primates. "This is an amazing milestone, and the official start of the Phase 1/2 clinical trial of EBT-101 has taken an extremely important step towards creating potential cures for AIDS," said Amy J. Goldberg, interim dean of Katz Medical College. "This is an amazing milestone, and the official start of the Phase 1/2 clinical trial of EBT-101 has taken us to take an extremely important step towards creating potential cures for AIDS."
Reference:
https://www.eurekalert.org/news-releases/965472
