SBIR Phase I: Development of an Adjustable Gene Therapy Platform Technology

The broader/commercial impacts of this Small Business Innovation Research (SBIR) Phase I project include the development of a gene therapy platform that will allow the use of genetic therapeutics in areas beyond rare diseases, tackling large unmet clinical needs such as diabetes, osteoarthritis, and autoimmune diseases. The project will also enable significant reductions in the costs of gene therapies and protein-based therapeutics.

Finally, the development of the proposed platform technology will enable the production of medical treatments that are injected less frequently, produce a potentially more optimal treatment profile, and prevent complications related to missed doses or therapeutic overdose. The societal and commercial impacts of this technology are significant, as the proposed technology could greatly expand the potential of gene therapy, while replacing other biologic-based therapies with a lower cost alternative.

The technology has significant commercial value but is, at the same time, able to greatly reduce societal medical costs associated with current treatment approaches.

This project develops a dose-adjustable gene therapy mechanism that can be used to up- or down-regulate a gene therapy dose, following initial administration. Despite recent advances and regulatory approvals, gene therapy remains limited due to its inherent shortcomings in dose adjustment – once a gene therapy dose is administered, it cannot be increased or decreased by secondary intervention.

On the other hand, many therapeutics require adjustment of the initially prescribed dose over a period of weeks or months to optimize the efficacy and side-effects profile. This project aims to develop and characterize the first, fully adjustable gene therapy, capable of predictable post-treatment dose adjustment. To accomplish this, a number of technological hurdles will be addressed as part of the project including non-viral delivery of genetic material to human cells, the ability to control the gene expression in a predictable and measurable manner, and the assurance that any adjustability is safe to the patient organs, tissues, and cells that neighbor the treatment area.

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.