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Excision BioTherapeutics Secures $60 Million to Advance CRISPR-Based Gene Editing Constructs Developed at Temple into Clinical Trial for People Living with Chronic HIV Infection

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Excision BioTherapeutics, Inc. (Excision) has secured $60 million in investor financing to advance CRISPR-based gene editing constructs developed by 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 the Lewis Katz School of Medicine at Temple University (LKSOM). The funding will allow these treatments to be advanced into a Phase 1/2 clinical trial in patients with chronic HIV infection. The funding secured by Excision will also provide Temple scientists and Excision the ability to research CRISPR technology on other viral diseases at LKSOM including JC Virus for Progressive Multifocal Leukoencephalopathy, Herpes Simplex Virus and other viral diseases. Those additional studies will be led by Ilker K. Sariyer, DVM, PhD, Associate Professor of Neuroscience and Neurovirology at LKSOM.

“The funding secured is a momentous occasion for Excision and Temple and the many individuals living with HIV and other viral diseases,” said John M. Daly, MD, FACS, Interim Dean & Dean Emeritus and Harry C. Donahoo Professor of Surgery at LKSOM. “These continued research and clinical trials bring us one step closer to having new technology available to suppress and eradicate multiple viral diseases.”

In research recently published by Nature Communications, Dr. Khalili along with Tricia H. Burdo, PhD, Associate Professor and Associate Chair of Education in the Department of Neuroscience at LKSOM, who is an expert on the utilization of the SIV (simian immunodeficiency virus)-infected antiretroviral therapy (ART)-treated rhesus macaque model for HIV pathogenesis and cure studies; Dr. Sariyer; Rafal Kaminski, PhD, Assistant Professor of Neuroscience and Assistant Professor in the Center for Neurovirology at LKSOM; Pietro Mancuso, PhD, an Assistant Scientist in Dr. Khalili’s laboratory; Chen Chen, a Research Technician in Dr. Khalili’s laboratory; other scientists in Dr. Khalili's, Dr. Burdo’s, Dr. Sariyer’s and Dr. Kaminski’s laboratories; and colleagues demonstrated success in editing the genome of simian immunodeficiency virus (SIV), a virus closely related to HIV, the cause of AIDS, from non-human primates. Dr. Khalili and Dr. Burdo were senior co-investigators on the study and Dr. Mancuso was first author. 

The success of Dr. Khalili’s HIV discoveries has led Temple into a partnership with Excision to support the continued development of the CRISPR technology. In this partnership Dr. Khalili serves as Co-Founder and Chief Scientific Consultant, and holds equity in Excision, which has licensed the viral gene editing technology from Temple University. Dr. Burdo contributes to preclinical research and development, serves on the Scientific Advisory Board and holds equity in Excision.

“This transformative financing will accelerate and support the research we have developed over the past decade,” said Dr. Khalili. “We have proven the technology and candidate programs in vitro and in vivo in both small animal models and primate models. We are optimistic that ongoing research will demonstrate the potential for a future therapeutic to generate functional cures for viral infectious diseases.”

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 and Rafal Kaminski are named inventors on patents that cover the viral gene editing technology. Tricia Burdo is a consultant for and holds equity in Excision BioTherapeutics. Ilker Sariyer is a consultant for Excision. Dr. Khalili , Dr. Burdo, Dr. Sariyer and Dr. Kaminski are employed by Temple University, and both they and LKSOM faculty within the department 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.