First human trial of HIV gene therapy: A one-time cure will be achieved if successful!
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First human trial of HIV gene therapy: A one-time cure will be achieved if successful!
A breakthrough gene-editing therapy currently in clinical trials for human immunodeficiency virus type 1 (HIV) infection has been used for the first time in an HIV-infected patient.
This trial was designed to evaluate the safety and efficacy of EBT-101.
As a one-of-a-kind gene-editing therapy, EBT-101 has the potential to change the future of HIV treatment.
AIDS (AIDS) is caused by infection with HIV, a virus that attacks the body’s immune system.
There is still a lack of effective drugs to cure HIV infection worldwide.
The current treatment goal is to maximally and lastingly inhibit viral replication in patients, so that patients can rebuild and maintain immune function, while reducing the morbidity and mortality of HIV infection and non-AIDS-related diseases.
EBT-101 Therapy
HIV resides in tissue reservoirs for a long time, evading the immune system and antiretroviral therapy, and is able to survive in the tissue host for years.
EBT-101 is a unique, clinical-stage, in vivo CRISPR-based therapeutic designed to cure HIV infection after a single intravenous infusion.
EBT-101 employs an adeno-associated virus (AAV) to deliver CRISPR-Cas9 for gene editing and uses multiple single guide RNAs targeting different regions of the HIV genome to cleave HIV DNA from the genome of human cells, thereby eliminating the opportunity for viral escape.
Dr Khalili said: “Almost 40 million people worldwide are affected by HIV, and more than 40 years after the discovery of HIV/AIDS, there is still no cure.
EBT-101 addresses HIV by removing viral DNA from cells /The long-term unmet need of people living with HIV, thereby eradicating the infection.” Removing HIV DNA from a cell’s genome is critical for treatment.
EBT-101 Phase 1/2 Clinical Trial
The Phase 1/2 clinical trial of EBT-101 is an open-label, multicenter, single-ascending-dose study.
The first patient to receive a single dose of intravenous EBT-101 is currently under observation and will soon be assessed for viral rebound if eligible to discontinue antiretroviral therapy.
If the one-time cure works as expected, patients will no longer need antiretroviral therapy – the current standard of care for HIV infection, the researchers said .
The Phase 1/2 clinical trial for EBT-101 was initiated by Dr. Kamel Khalili, Professor Laura H. Carnell and Chair of the Department of Microbiology, Immunology and Inflammation; Director of the Center for Neurovirology and Gene Editing; Director of the Center for Integrative NeuroAIDS, and Based on research led by Tricia H. Burdo, Ph.D., professor and associate chair of the Department of Microbiology, Immunology and Inflammation at Katz School of Medicine.
Dr. Khalili and colleagues at Temple University pioneered the development of CRISPR-based gene editing technology for EBT-101, and Dr. Burdo and her team have contributed to advancing EBT-101 clinical trials through EBT-101 studies in non-human primates. key role.
Amy J. Goldberg, interim dean of the Katz School of Medicine, said: “This is an amazing milestone, and the official start of the Phase 1/2 clinical trial of EBT-101 brings us an extremely close step toward creating a potential cure for HIV. an important step.”
References:
https://www.eurekalert.org/news-releases/965472
First human trial of HIV gene therapy: A one-time cure will be achieved if successful!
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