I-Corps: Delivery system for gene-based medicines

Award ID: 2120291

The broader impact/commercial potential of this I-Corps project is the development of a modular drug-delivery platform for the delivery of gene-based therapeutic medicines. The next frontier in biotechnology is focused on gene-based medicines to treat a broad spectrum of diseases, however, their success is dependent on delivery methods to enhance their efficacy and safety. Current technologies used to deliver these medicines raise concerns regarding distribution/transportation due to thermostability or portability, high costs of development in manufacturing, and safety. The successful translation of this project may enable a new paradigm in non-viral gene delivery as well as broader access to these therapeutics for a wider population through lower costs, improved distribution, and enhanced safety and efficacy. The proposed technology may provide gene therapy companies with a platform to deliver their therapeutics on a global scale to advance 5personalized medicine. This I-Corps project is based on the development of a drug delivery platform for DNA and mRNA-based therapeutics utilizing a modified electroporation technology. Electroporation is a technique for gene delivery that shocks cells with an electric pulse, allowing therapeutics and other molecules to permeate cell walls. The proposed microneedle-electroporation platform enables both gene and pulse delivery showing comparable performance to research use electroporator systems. The proposed technology delivers coated or injected therapeutics to enhance gene expression >400-fold with improved safety. Preliminary studies in vivo have shown efficacy comparable to commercial benchmarks for safety, including tests with a COVID-19 DNA vaccine. The proposed technology is designed to be handheld and weighs less than 50g, operates without access to electricity, is manufactured with existing infrastructure at a cost of less than $1, and may be operated by minimally trained personnel or self-administered. Further development is required for the identification of specific biomedical applications. This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.

NSF Program Director: Ruth Shuman