This Small Business Innovation Research (SBIR) Phase I project will develop a platform technology that improves drug delivery. Many drugs, such as blood factors, require frequent dosing because they do not last long in the body and/or require delivery via a needle into the vein because they are not available to the body when given as an injection into the skin. This project is aimed at developing a way to modify the blood factors so that they can access one of the body?s natural transport pathways to deliver them into the blood where they are needed, and allow them to last longer in the body. In this project, the modification will be tested for its ability to enhance the properties of blood factors, expanding on previous results. The blood factors will be tested to make sure they are still functional following modification. If successful, the proposed Phase II studies would test the effectiveness and improved properties of the modified blood factors in the appropriate models. This novel approach could improve the delivery efficiency of numerous other drugs by allowing them to be administered through the skin, and dosed on a less-frequent basis.
The broader impact/commercial potential of this project is the development of a drug delivery platform that can improve a drug?s properties such that the drug requires less-frequent dosing, and can now be administered by the patient at home. This technology has significant commercial potential in that it can be broadly applied to numerous emerging and existing drugs in the $140B global biotherapeutics market. As an example, current treatments for hemophilia require frequent intravenous infusions of blood factors over the course of a patient?s lifetime. Use of this technology may have a significant societal impact by providing more efficient medicines delivered in a more patient-friendly way that will reduce healthcare cost and increase patient compliance. Development of this technology will further scientific understanding of drug delivery. This technology will advance the field in that it has significant advantages over the current state-of-the-art, including the ability to allow for less-frequent dosing, and by increasing the amount of drug available to the body, which decreases the dose. These beneficial effects are obtained with one simple modification that does not interfere with its function.
The technology being developed under the NSF SBIR Phase I award will enable the rapid development of peptide-based drugs. Currently, peptide-based drugs have an extremely short residence time in the body that prevents the drugs from having a long acting therapeutic effect. The platform technology being developed will allow this class of drugs to be retained in the body for a longer period of time thus allowing for the therapeutic effects to be meaningful. When fully developed, this technology will enable this class of drugs to be developed more rapidly, allowing for new medicines to come to market. The technical objectives for SBIR Phase I were met and feasibility of the approach was demonstrated with one promising peptide-based therapeutic during the grant award period. We proposed two objectives: (1) broaden the platform and perhaps improve upon the original design, and (2) show additional examples of the utility of the platform. Feasibility of the approach was demonstrated by showing that (1) we improved the properties over the first-generation design, and (2) we showed that a modified peptide had a greatly extended residence time in vivo. The positive results justify continuing on in SBIR Phase II to develop the long-acting peptide as a potential treatment for a cardiopulmonary indication.
