Some 38 million people worldwide are living with HIV infection, the cause of AIDS. Thanks to advances in treatment and access to antiretroviral drugs, many of those individuals have the chance to live long, healthy lives. But medications for HIV infection are expensive, and they work by suppressing the virus – rather than by eliminating it from the body – requiring patients to make a life-long commitment to taking the drugs.
Those factors emphasize the need to find an HIV cure, and now, researchers at the Lewis Katz School of Medicine at Temple University have an unprecedented opportunity to break critical ground toward that goal, thanks to a prestigious grant from the National Institutes of Health. The grant, which is part of the Martin Delaney Collaboratories for HIV Cure Research program, will provide the Temple researchers and collaborating members with $4.8 million each year for five years.
A major aim of the Martin Delaney Collaboratories program is to expedite research for an HIV cure by fostering productive relationships between researchers at academic institutions, researchers in the private sector and government, and partners in the community. The program is funded by the National Institute of Allergy and Infectious Diseases; the National Institute of Mental Health; the Eunice Kennedy Shriver National Institute of Child Health and Human Development; the National Institute of Diabetes and Digestive and Kidney Diseases; the National Heart, Lung, and Blood Institute; the National Institute on Drug Abuse; and the National Institute of Neurological Disorders and Stroke. Temple is one of 10 academic institutions across the United States chosen to receive a portion of the $53 million in annual funding provided by these NIH Institutes on behalf of the program.
“The funding provided by the NIH and the Martin Delany Collaboratories is a momentous occasion for Dr. Kamel Khalili, Dr. Tricia Burdo, their teams and, most importantly, the many individuals living with HIV,” said Amy J. Goldberg, MD, FACS, Interim Dean and George S. Peters, MD and Louise C. Peters Chair and Professor of Surgery at the Katz School of Medicine and Surgeon-in-Chief and Senior Vice President of Perioperative Services at Temple University Health System. “I am immensely proud of this team and their work; it is exciting for Temple to be leading the way in utilizing CRISPR technology to develop novel treatments for individuals living with HIV and other viral diseases.”
“We are honored to be included among the institutions selected to receive funding through the Martin Delaney Collaboratories program,” 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 Katz School of Medicine, and a principal investigator on the new grant. “We are truly excited to be able to work with experts at other institutions and with community and industry partners toward a common goal of curing HIV.”
Dr. Khalili is joined as a principal investigator and co-director on the grant by Tricia H. Burdo, PhD, Associate Professor and Associate Chair of Education in the Department of Microbiology, Immunology and Inflammation at the Katz School of Medicine.
The grant will fund the team’s project, titled “CRISPR for Cure,” which was built based on the employment of CRISPR gene-editing technology for targeting and eliminating HIV from infected cells that was pioneered by Dr. Khalili and his Temple co-workers.
In total, the award will fund four major research laboratories at Temple, supporting their pursuit to develop and refine CRISPR gene-editing technology to inactivate or remove dormant HIV from infected cells, to enhance immune responses and to engineer immune cells to be resistant to HIV infection. The laboratories include those led by Dr. Khalili and Dr. Burdo, as well as those led by Ilker K. Sariyer, DVM, PhD, Associate Professor of Neural Sciences at the Center for Neurovirology and Gene Editing at the Katz School of Medicine, and by Rafal Kaminski, PhD, Assistant Professor of Neural Sciences at the Center for Neurovirology and Gene Editing at the Katz School of Medicine.
The researchers will be partnering with Excision BioTherapeutics, Inc., a biotech company co-founded by Dr. Khalili and for which Dr. Burdo serves on the Scientific Advisory Board.
For nearly a decade, Dr. Khalili and colleagues have been working to develop a gene-editing system using CRISPR-Cas9 technology to remove HIV DNA from genomes harboring the virus. The researchers have shown in previous studies that their system can readily eliminate HIV DNA from cells and tissues in small animal models of HIV infection, including HIV-1 humanized mice.
In 2020, Dr. Khalili’s team, in collaboration with Dr. Burdo, who specializes in the study of SIV – a virus very closely related to HIV – for the first time successfully edited SIV from the genomes of non-human primates. The breakthrough brought the Temple team closer than ever to developing a cure for human HIV infection.
“Now, because of the Collaboratories program, we can not only advance our work toward editing HIV out of the human genome but also engage with the community and corporate sectors to raise awareness and create future opportunities for therapeutic development,” Dr. Burdo said.
“Receiving this grant is a big accomplishment for Temple,” Dr. Khalili added. “We have a very strong group of researchers here who are truly devoted to finding a cure for HIV.”
The Temple University project is titled “CRISPR for Cure,” NIH grant: 1 UM1AI164568-01. For additional information on the Martin Delaney Collaboratories for HIV Cure Research program and institutions selected for funding, see: NIH Makes Substantial New Investment in HIV Cure Research.
The content of this publication is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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 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 [email protected].
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 [email protected].
