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San Francisco, CA, November 30, 2020 (GLOBE NEWSWIRE) – Taking a major step forward in HIV research, scientists at Temple University’s Lewis Katz School of Medicine have successfully modified SIV, a virus closely related to HIV, the cause of AIDS – from the genomes of non-human primates. The turning point, reported in the magazine Nature Communications, marks a fundamental step towards the development of a possible cure for human HIV infection. Excision BioTherapeutics holds the exclusive license for commercial application of these advances as it works on the development and commercialization of advanced genetic modification therapies for the treatment of life-threatening diseases caused by viruses.
“For the first time, a single inoculation of our CRISPR gene modification construct, carried by an adeno-associated virus, can modify the SIV genome from infected cells in rhesus macaque monkeys,” he said. Kamel Khalili, PhD, Laura H. Carnell Professor and Chair of the Department of Neuroscience, Director of the Center for Neurovirology and Director of the Comprehensive NeuroAIDS Center at Temple University’s Lewis Katz School of Medicine (LKSOM) and the co-founder and chief scientific consultant on excision.
Of particular importance, the new work shows that the gene-editing construct can reach infected cells and tissues known to be viral reservoirs for SIV and HIV. These reservoirs, which are cells and tissues where viruses integrate into host DNA, cause the virus to emerge as soon as antiretroviral therapy (ART) is stopped.
Dr. Tricia Burdo, Associate Professor and Associate Chair of Education in the Department of Neuroscience at Temple University’s Lewis Katz School of Medicine, and a member of Excision’s Scientific Advisory Board, added: “The SIV-infected rhesus macaque model is an animal model of large size ideal for recapitulating HIV infection in humans. ”
For the new study, the researchers began by designing a CRISPR-Cas9 gene editing construct specific to SIV. Cell culture experiments confirmed that the editing tool cleaved the SIV integrated DNA in the correct position from the host cell DNA, with a limited risk of potentially damaging gene modification at off-target sites. The research team then packaged the construct into an adeno-associated virus vector 9 (AAV9), which could be injected intravenously into SIV-infected animals. Analyzes showed that in macaques treated with AAV9-CRISPR-Cas9, the gene-editing construct had been distributed to a wide range of tissues, including bone marrow, lymph nodes, and spleen, and reached CD4 + T cells. and that the SIV genome was indeed cleaved from the infected cells.
The new study is a continuation of efforts by Dr. Khalili, Dr. Burdo and colleagues to develop a new gene editing system using CRISPR-Cas9 technology – the subject of the 2020 Nobel Prize in Chemistry – to specifically remove HIV. DNA from the genomes hosting the virus. Researchers have previously shown that their system can effectively clear HIV DNA from cells and tissues in small HIV-infected animal models, including HIV-1 humanized mice.
“This work is a critical step in launching human clinical trials for a cure for HIV,” said Daniel Dornbusch, CEO of Excision. “Researchers have been looking for a cure for viruses such as HIV for decades. This important work takes us one step closer to evaluating the safety and effectiveness of a single infusion to treat infected people around the world. We are proud of the team’s work and diligent efforts and look forward to advancing this important product in human clinical trials. “
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Editor‘s Note: Kamel Khalili is co-founder and chief scientific consultant and holds interests in Excision BioTherapeutics, which licensed viral gene editing technology from Temple University. Kamel Khalili and Rafal Kaminski are named inventors of patents covering viral gene editing technology. Tricia Burdo and Jennifer Gordon hold stakes in Excision BioTherapeutics. These nominated researchers are employed by Temple University and conduct company-sponsored research. Questions regarding their affiliation with Temple University can be directed to [email protected].
In addition to owning Excision licensed viral gene editing technology, Temple University also holds a stake in Excision. As a result of these interests, Temple University could ultimately potentially benefit financially from the results 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 [email protected].
About Excision BioTherapeutics Inc.
Excision BioTherapeutics develops CRISPR-based therapies to treat viral infectious diseases. Excision is the first company in history to remove viral genomes from animals and obtain functional HIV treatment with therapy. The company’s pipeline includes potential cures for JC / PML virus, hepatitis B, Herpes Simplex virus, and SARS-CoV-2. The underlying technology was developed at Kamel Khalili’s lab at Temple University and Jennifer Doudna’s lab at Berkeley University. Excision is located in San Francisco, California and is supported by Artist Ventures, Norwest Venture Partners, Abstract business, SilverRidge Capital Partners, is Gaingels. The company is preparing to present its first IND for EBT-101, its leading HIV program in 2020. For more information, visit www.excision.bio.
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