Gene therapy research for HIV awarded $14.6 million NIH grant

On September 2, 2020, it was announced that an HIV research program led by scientist at USC Keck School of Medicine and the Fred Hutchinson Cancer Research Center in Seattle received a five-year, $14.6 million grant from the National Institutes of Health. The team is advancing a gene therapy approach to control the virus without the need for daily medicines.

The program’s co-directors are Paula Cannon, PhD, Distinguished Professor of Molecular Microbiology and Immunology at the Keck School of Medicine of USC, and Hans-Peter Kiem, MD, PhD, the Stephanus Family Endowed Chair for Cell and Gene Therapy at Fred Hutch. Other key partners are David Scadden, MD, a professor at Harvard University, and the biotechnology company Magenta Therapeutics.

The NIH award will support preclinical studies that combine gene editing against HIV with technologies for safer and more effective hematopoietic stem cell transplants. Such transplants, also known as bone marrow transplants, are currently used for severe blood cancers. They renew a patient’s immune system, which can be damaged by cancer therapies, by infusing healthy donor blood stem cells that can grow into any type of blood or immune cell.

The researchers’ goal is to build a therapy that prepares patients for a stem cell transplantation using their own cells with little to no toxicity, engineers their own stem cells to fight HIV and stimulates those cells to quickly produce new and engineered immune cells once they’re reintroduced into the patient.

“This grant funds a team with an overarching goal of developing what our perfect HIV gene therapy would look like,” Cannon said. “All of these pieces could happen separately, but the fact that the NIH has funded us as a team means that the sum will be so much bigger than the parts.”

You can read the full announcement here.