Unmet Need
In the United States, approximately 14.6 million Americans are estimated to suffer from diabetic retinopathy by the year 2050 (see CDC). Diabetic retinopathy is a debilitating condition defined by the loss of vision due to leaky blood vessels of the retina. The current standard of care includes prevention measures, pharmacological therapies, and surgery as a last resort. However, this strategy is limited by only providing vision management rather than fully-restored vision. To improve the current standards, recent research has focused on deploying vascular regenerative cell-therapies with conventional human induced pluripotent stem cells (hiPSC). While promising, the current approaches to generate hiPSCs are limited by high inter-line variability of differentiation and poor efficiency for generating functionally transplantable vascular progenitors. There is a dire need for a new approach that eliminates variability of differentiation and efficiently establishes functional transplantable vascular progenitors.
Technology Overview
Johns Hopkins researchers developed a novel approach to generate naïve vascular progenitors with enhanced in vivo proliferation and engraftment capacities. The inventors demonstrated that a novel class of tankyrase inhibitor-regulated naïve hiPSC (N-hiPSC) with improved functionality can significantly improve vascular cell-therapies by more efficiently reprogramming the epigenetic state of a patient’s donor cells. The naïve vascular progenitors derived from N-hiPSC have a novel embryonic phenotype and possess both improved pericytic and endothelial regenerative potential. Vascular progenitors generated from these novel N-hiPSCs strongly express vascular cell identity markers, maintain greater genomic stability, and have decreased non-lineage gene expression, while exhibiting improved migration and vascularization properties in the ischemic retina. The application of these embryonic pericytic naïve vascular progenitors has potentially high impact in vascular regenerative medicine for treating ischemic retinopathy, myocardial infarction, and cerebral vascular stroke. More broadly, this approach provides a new class of human stem cells for regenerative medicine that may extend beyond diabetic retinopathies to address other disease states.
Stage of Development
Experimental and pre-clinical data is available.
Publications
Park, Tea Soon, et al. Nature Communications 11.1 (2020): 1-20.
