Dr. John “Chip” Tilton, an associate professor and director of Immunobiology in the Center for Proteomics and Bioinformatics at the CWRU School of Medicine will be receiving $300,000 from the Dr. Ralph and Marian Falk Medical Research Trust. This funding will be used to develop a virus-based “Nano POD” (nanoscale protein delivery) platform to help treat rare genetic diseases. The seed funding, part of the Falk Catalyst Awards Program, will support one year of research into effectively packaging therapies into nanoPODS, and delivering them to specific tissues in mice affected by genetic disease.  

Tilton’s project is of dire significance because it will address one of the major challenges that nanoscale therapeutics tend to face: delivering drugs to the right parts of the body. Viruses, with their expertise in penetrating cells, also represent vehicles that can be co-opted by researchers to deliver therapeutics. nanoPODS’ main mission is to treat diseases caused by genetic mutations. Specifically those that introduce a “termination codon” that would prematurely stop protein synthesis. This form of mutation represents approximately ten percent of all genetic diseases. Some of the diseases these mutations are responsible for are cystic fibrosis, muscular dystrophy and metabolic disease. “The project is a really exciting bioengineering challenge to develop a system for delivering these drugs to the tissues where we want them in patients, while avoiding side-effects from delivering to the ‘wrong’ tissues,” Tilton said.

”While promising, these treatments cannot be used in patients yet due to a lack of a technological platform to efficiently deliver the suppressor tRNAs or genome editing machinery to the diseased organs inside of patients,” Tilton said. Tilton plans to package the therapies into nanoPODs, infuse them into mice and measure the nanoPODs’ ability to safely deliver their contents to affected tissues.

If successful, nanoPODS could be used to package a variety of emerging therapeutics. “RNA and protein therapeutics are still in their infancy, but have shown tremendous success over the past decade in providing treatments for cancer and autoimmune diseases,” Tilton said. “I think over the coming decades these drugs will take an increasingly important role in the treatment of many diseases.”