Although great advances have been made in biomedical research, scientists and clinicians are still looking for effective treatments for some of the most devastating diseases, such as diabetes, cardiovascular diseases, spinal cord injury, Parkinson’s disease, and Alzheimer’s disease. Potential treatments for these diseases include drug-based therapies and cell-based therapies. Both have their limitations. Drug-based therapies usually treat symptoms only and make patients chronically dependent on them. Cell-based therapies are hampered by the scarcity of the source, immune rejection, high manufacture and distribution costs, and high development and commercialization risks.
Our Solution: IVR Technology Platform
We are developing a new technology platform, named In Vivo Cell Reprogramming, or IVR. Using a combination of protein drugs, we can convert one type of cells (substrate cells) to another type of cells (product cells). Product cells are usually the damaged or depleted cells associated with certain diseases, and substrate cells are one type of cells that are abundant in the proximity of or in the affected organs. For example, in one IVR application, liver cells or acinar cells (substrate cells) can be converted to b cells (product cells) to treat diabetes.
IVR combines the advantages of drug-based and cell-based therapies while avoiding the shortfalls of both. Since the modality of IVR therapeutics are proteins, their risk profiles and cost structures of development and commercialization are similar to those of drug-based therapies. However, by using a cell-based mechanism, IVR therapeutics can achieve therapeutic benefits that cannot be achieved by conventional drugs. In addition, the newly created product cells are sustainable. Therefore a short course of treatment can provide long-term clinical benefits. Moreover, contrary to most situations in cell or tissue transplantation, new product cells will not cause immune injection.
We have applied IVR to several disease areas, including diabetes, autoimmune diseases, and stroke. We are collaborating with several academic institutes and have generated some exciting data.