A single injection of a novel CRISPR gene-editing treatment safely and efficiently removes SIV—a virus related to the AIDS-causing agent HIV—from the genomes of non-human primates, scientists at the Lewis Katz School of Medicine at Temple University now report. The groundbreaking work complements previous experiments as the basis for the first-ever clinical trial of an HIV gene-editing technology in human patients, which was authorized by the Food and Drug Administration (FDA) in 2022.
therapy, in rhesus macaques. The study shows that EBT-001 effectively excises SIV from reservoirs—cells and tissues where viruses like SIV and HIV integrate into host DNA and hide for years—without any detectable off-target effects in animals. The work is a significant advance in the generation of a cure for HIV/AIDS in humans.
EBT-101 is a unique gene-editing treatment that has the potential to shape the future of HIV therapeutics. Its development is the result of a collaborative effort between researchers at the Lewis Katz School of Medicine and scientists at Excision BioTherapeutics, Inc. Two additional animals were utilized in a separate study using a higher dose. Necropsy and tissue analyses were carried out at three or six months after the start of treatment. Data was collected on biodistribution, which involved histopathological investigation of sites of viral latency, including lymph node and spleen tissue, as well as other tissues, and on safety, which included off-target analyses at the different dose levels.
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