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

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Tricia H. Burdo, PhD, and Kamel Khalili, PhD

For the last seven years, 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.

“The clinical trials highlight a well-orchestrated succession of academic research findings from Temple, now with the translation [of those findings] to treatment for people living with HIV-1 infection, which is an exciting development,” said 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.

Dr. Khalili is a pioneer in the development of CRISPR-based gene-editing technology for targeting and eliminating HIV from infected cells. In preclinical studies, Dr. Khalili 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.

“Temple-based preclinical studies, in both small animal and primate models, have successfully shown that CRISPR-based therapies are safe and effective,” Dr. Burdo said. “These studies have paved the way for Excision to advance this technology with a primary focus of bringing this therapy to the HIV community to improve long-term outcomes.” Dr. Burdo serves on the Scientific Advisory Board for Excision BioTherapeutics.

“For EBT-101 to gain FDA clearance for testing in clinical trials is testament to the amazing work that Drs. Khalili and Burdo and their teams have done – and the tremendous pool of talent at Temple,” said Amy J. Goldberg, MD, FACS, Interim Dean of the Lewis Katz School of Medicine. “It takes vast scientific skill and innovation to create a therapy with life-changing potential.”

Editor’s Note: Kamel Khalili is Co-Founder and Chief Scientific Consultant and holds equity in Excision BioTherapeutics, which has licensed the viral gene-editing technology from Temple University. Kamel Khalili is a named inventor on patents that cover the viral gene-editing technology. Tricia Burdo holds equity in Excision BioTherapeutics. These named researchers are employed by Temple University and conduct research activities sponsored by the company. Questions regarding their affiliations with Temple University may be directed to coisom@temple.edu

In addition to owning the viral gene-editing technology that Excision is licensing, Temple University also holds an equity interest in Excision. As a result of these interests, Temple University could ultimately potentially benefit financially from the outcome of this research. These interests have been reviewed and approved by Temple University in accordance with its Institutional Conflict of Interest policy. Questions about this can be directed to coitemple@temple.edu