New Research Provides Information for First Trial of CRISPR-Based HIV Therapy in Humans

Khalili and his colleagues have completed preclinical studies that have shown 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.

Researchers at the Lewis Katz School of Medicine at Temple University have been developing and refining CRISPR-based gene-editing technology for the past 7 years to treat HIV infection. EBT-101, a new therapy, has emerged from these data and could become the first functional cure for chronic HIV infection, according to a press release.

After the FDA approved a clinical trial for EBT-101, the treatment now heads to a phase 1/2 clinical trial of a CRISPR-based therapy for HIV infection. The trial will be managed by Excision BioTherapeutics, Inc, which has collaborated with Temple on 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, director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine, as well as cofounder of Excision BioTherapeutics, in the press release.

Khalili and his colleagues have completed preclinical studies that have shown 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.

“Temple-based preclinical studies, in both small animal and primate models, have successfully shown that CRISPR-based therapies are safe and effective,” said Tricia H. Burdo, PhD, associate professor and associate chair of Education in the department of Microbiology, Immunology, and Inflammation, in the press release. “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.”

REFERENCE

Groundbreaking research at Temple paves way for first trial of CRISPR-based HIV therapy in human patients. Temple University- Lewis Katz School of Medicine. September 15, 2021. Accessed September 20, 2021. https://medicine.temple.edu/news/groundbreaking-research-temple-paves-way-first-trial-crispr-based-hiv-therapy-human-patients