HIV Gene Editing: EBT-001 Clears SIV in Non-Human Primates, Paving the Way for Clinical Trials

Researchers at the Lewis Katz School of Medicine at Temple University have reported a groundbreaking achievement in gene-editing technology. Their study, published in the journal Gene Therapy, details the successful use of a novel CRISPR gene-editing treatment, EBT-001, to safely and efficiently eliminate SIV—a virus related to HIV—from the genomes of non-human primates.

The preclinical trial focused on rhesus macaques and demonstrated that EBT-001 effectively removed SIV from cellular reservoirs without detectable off-target effects. These reservoirs are where viruses like SIV and HIV integrate into host DNA and remain hidden for extended periods. The findings serve as a crucial step forward in developing a cure for HIV/AIDS in humans.

Dr. Kamel Khalili, senior investigator and chair of the Department of Microbiology, Immunology, and Inflammation, emphasized the safety and efficacy of EBT-001, highlighting its potential for permanent virus inactivation across various tissues. The promising results paved the way for the FDA-authorized clinical trial of EBT-101, sponsored by Excision Biotherapeutics, Inc.

EBT-101, a unique gene-editing treatment developed through collaboration between researchers at the Lewis Katz School of Medicine and Excision BioTherapeutics, holds significant promise for the future of HIV therapeutics. Before advancing to human trials, safety data was collected from non-human primate studies using a version of EBT-101 adapted for SIV infection.

Led by Dr. Tricia H. Burdo, the animal studies involved randomized control and treatment groups, with EBT-001 administered at varying dose levels. The comprehensive analyses conducted over a lengthy timeframe confirmed the broad distribution of EBT-001 across tissues, successful gene editing of SIV proviral DNA, and the treatment’s overall well-tolerance.

The study’s impact extends beyond HIV/AIDS, with Excision Biotherapeutics planning to assess EBT-101’s safety and tolerability for potential use in gene therapies for other infectious diseases, such as herpes simplex virus and hepatitis B.

Dr. Khalili expressed excitement about the progress, emphasizing the collaborative efforts that contributed to the development of this new treatment. The study’s findings, published in Gene Therapy, offer a significant leap forward in the pursuit of effective gene-editing therapies for HIV and other infectious diseases.

Reference: Burdo TH, Chen C, Kaminski R, et al. Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates. Gene Ther. 2023. doi: 10.1038/s41434-023-00410-4