The Catholic University of America’s Bacteriophage Medical Research Center (BMRC) hosted its inaugural symposium, The Future of Gene Therapy, on May 1. Speakers and guests explored the state of gene therapy research and some of the potentially promising advances in the field, especially those happening at the BMRC and at Children’s National Hospital.
University President Peter Kilpatrick opened the symposium, remarking that gene therapy has been a dream of bioengineers for some time and that he is encouraged by the promise it holds.
“Immunotherapy can only go so far,” said Kilpatrick. “It doesn’t really treat the root cause, whereas gene therapy holds the promise to do that.”
Venigalla Rao, Ph.D., professor of biology and BMRC founder, spoke enthusiastically about the contributions he anticipates the BMRC will make to the field, noting that his confidence is based on the fundamental knowledge of bacteriophages that he and his collaborators have discovered over decades of research. Rao believes the time is ripe for breakthroughs in translational bacteriophage research.
Bacteriophages are viruses that infect bacterial cells and then replicate within them and, as it turns out, they can be engineered to deliver vaccines and gene therapies. Rao and his students and colleagues at the BMRC work with collaborators across the country to produce cutting-edge basic and translational research that could lead to the creation of a new category of biomedicines based on their success in understanding and reengineering bacteriophages.
Existing gene therapies are astronomically expensive, and the current research paradigms have led to limited progress, but Rao believes that bacteriophage-based therapies provide a promising alternative to them. The concept for bacteriophage-based therapy distilled to its most basic level is to engineer the phage in the test tube as a “delivery machine” programmed with a set of therapeutic biomolecules: DNAs, RNAs, and proteins.
The phage’s own DNA is removed from its shell; the therapeutic DNA molecules are then packaged inside it, while its outer surface is engineered with targeting and genome editing RNA and protein molecules; and the programmed phage nanoparticles can then be used to deliver the payloads into specific human cells. This versatile approach allows bioengineers to use the phage as a platform for designing many therapies: gene therapies to treat genetic diseases, vaccines against infectious diseases, and antimicrobials to combat antibiotic-resistant pathogens.
“We engineer one phage, namely T4, for many applications instead of the traditional approach that combines many natural phages for only one application,” explained Rao.
The symposium keynote was delivered by Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National Hospital (CNH). Hanley’s address, “Building a cell and gene therapy program to treat children and adults with cancer and infectious diseases,” summarized the work that CNH and its collaborators are doing, using a variety of antiviral drugs to test and treat patients.
Hanley also discussed efforts to build a hub for gene therapy research in the metropolitan Washington, D.C., area. This initiative, called CHARM (Capital Health and mid-Atlantic Regenerative Medicine), could provide a hub for networking and partnership opportunities for the BMRC, as well.
With breakthrough possibilities emerging in the field, Rao is very optimistic about the BMRC’s future, despite the fact that it is a fledgling center. His philosophy toward building the center is the same as the research philosophy he shares with his students: “Nothing is impossible,” he said. “You just haven’t figured it out . . . yet.”
For additional information, please contact the BMRC.
