Investigators Granted IND to Begin HIV Therapy Trials

The therapy, known as EBT-101, works by eliminating HIV-infected DNA from the genome.

The Lewis Katz School of Medicine at Temple University has developed a therapy that they hope could become a potential HIV cure.

The researchers have been refining CRISPR-based gene-editing technology to treat HIV for the last seven years. From this emerged a therapy they call EBT-101, recently declared an Investigational New Drug (IND) by the US Food and Drug Administration (FDA).

This IND approval allows EBT-101 to begin phase 1/2 clinical trials, which will be initiated and overseen by Excision BioTherapeutics.

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, director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine, and cofounder of Excision BioTherapeutics, was pivotal in the development of this therapy.

In preclinical trials, Khalili and researchers at Temple successfully demonstrated how EBT-101 eradicates HIV proviral DNA from the genomes of infected human and humanized mice cells and tissues.

Khalili’s other major collaborator was Tricia H. Burdo, PhD, associate professor and Associate Chair of Education in the Department of Microbiology, Immunology, and Inflammation at Temple, as well as a member of the Scientific Advisory Board at Excision BioTherapeutics.

Burdo and Khalili and their teams have used this gene-editing technology to eliminate SIV, a virus similar to HIV, from small animals and non-human primates.

Amy J. Goldberg, MD, FACS, interim dean of the Lewis Katz School of Medicine, emphasized the significance of the FDA granting EBT-101 clearance for clinical trials. She spoke very highly of the Temple researchers, saying, “it takes vast scientific skill and innovation to create a therapy with life-changing potential.”

As the Lewis Katz School of Medicine researchers prepare to commence clinical trials with Excision BioTherapeutics, they remain hopeful that EBT-101 will advance the fight against HIV.