Mei He, Ph.D., an associate professor of pharmaceutics in the University of Florida College of Pharmacy, has received a $1.3 million award from the National Institutes of Health to support a new extracellular vesicle platform used to develop gene therapy.
Extracellular vesicles, or EVs, are a relatively new approach to delivering genome editing therapeutics — the small, membrane-bound vesicles act as envelopes delivering intercellular communications that influence a cell’s behavior or function. By optimizing and validating their EV platform, He’s team will be able to accelerate the clinical translation of EV-based therapeutic products, a task that currently poses a significant challenge in the field.
“With this support, our team will be able to develop a scalable manufacturing pipeline and establish quality control criteria for EV-based genome editing products,” He said. “The outcome will have a significant impact on accelerating the translation of novel, safe and effective therapeutics and genome editing strategies into first-in-human clinical trials.”
As part of the award, He and her research team will be introduced into the NIH Common Fund’s Somatic Cell Genome Editing Consortium, which provides recourse and expertise from large testing facilities and regulatory office to facilitate the clinical development and evaluation of novel genome editing therapeutics.
“Being selected to the Somatic Cell Genome Editing Consortium could leverage synergistic resources and regulatory support, in addition to the NIH’s funding support, to accelerate the project’s development and clinical translation,” He said.
In creating this research project, “Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs,” He collaborated with Xiaoshu Pan, Ph.D., a postdoctoral researcher in her lab; UF Clinical and Translational Science Institute Good Manufacturing Practice Facility Director Eugenio Simon Manso, Ph.D.; UF Gene Therapy Center Toxicology Core Director Kirsten E. Coleman and UF Gene Therapy Center Director Barry Byrne, M.D., Ph.D.
The project began in 2023 when He’s team received a $100,000 UF Research seed fund to establish the Regional Center for Development of Advanced Therapeutics Based on Cell and Gene Therapy, the first-of-its-kind center in the Southeast to bring novel treatments in advanced therapeutics to market. Byrne said the far-reaching potential of this research would not be possible without interdisciplinary collaboration from a diverse team of experts.
“The collaborative effort of the Powell Gene Therapy Center and the UF College of Pharmacy team has been a model for the value of early investment in innovative research like the work of Dr. He,” Byrne said. “The transformative potential of gene editing will be evaluated in this program, and we are delighted to facilitate the clinical translation of this innovative approach.”