Groundbreaking Research at Temple Paves Way for First Trial of CRISPR-Based HIV Therapy in Human Patients

Researchers at the Lewis Katz School of Medicine at Temple University have been developing and refining CRISPR-based gene-editing technology for the treatment of human immunodeficiency virus type 1 (HIV) infection. Out of that effort has emerged a potentially revolutionizing therapy known as EBT-101, which thanks to recent acceptance as an Investigational New Drug (IND) by the U.S. Food and Drug Administration, could become the first functional cure for chronic HIV infection. The new IND approval for EBT-101 opens the way to the first Phase 1/2 clinical trials of a CRISPR-based therapy for HIV infection. The clinical trials will be initiated and managed by Excision BioTherapeutics, Inc., which has been a major collaborator with Temple on the development of CRISPR-based systems for the treatment of HIV. In preclinical studies, Kamel Khalili, PhD, Laura H. Carnell Professor and Chair of the Department of Microbiology, Immunology, and Inflammation, Director of the Center for Neurovirology and Gene Editing, and Director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine, as well as cofounder of Excision BioTherapeutics, and colleagues at Temple showed that EBT-101 can effectively excise HIV proviral DNA from the genomes of different cells and tissues, including HIV-infected human cells and cells and tissues of humanized mice. In collaboration with Tricia H. Burdo, PhD, Associate Professor and Associate Chair of Education in the Department of Microbiology, Immunology, and Inflammation, the Temple team further showed that the gene-editing technology can eliminate SIV – a virus closely related to HIV – from the genomes of non-human primates. Endpoints News covered the IND approval and included comments from Drs. Khalili and Burdo in its coverage.