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This technology includes the use of the combination of the compounds Chroman-1 and Emricasan to achieve virtually 100% cell survival during human pluripotent stem cell passaging, cryopreservation/thawing, and differentiation in 2D and 3D cultures. Human pluripotent stem cells, including ESCs and iPSCs, are highly sensitive cells and undergo apoptosis during these routine procedures. A screening approach was used to identify the combination of the two compounds in this invention.

This technology includes a robust and highly efficient protocol that differentiates human pluripotent stem cells (hPSCs) exclusively into nociceptors (also called sensory neurons) under chemically defined conditions. The use of hPSCs, including hESCs and iPSCs, holds great promise for drug screening, disease modeling, toxicology, and regenerative medicine. However, efficient and highly reproducible protocols have not been developed for most cell types that are relevant and urgently needed for translational applications.

This technology includes a robust and highly efficient protocol that differentiates induced pluripotent stem cells (iPSCs) exclusively into nociceptors (also called sensory neurons) under chemically defined conditions. The use of hPSCs, including hESCs and iPSCs, holds great promise for disease modeling, drug discovery, and cell therapy. However, efficient and highly reproducible protocols have not been developed for most cell types that are relevant and urgently needed for translational applications.

This technology includes the use of a new class of molecules (nanomolar ALK2 inhibitor) to impede bone morphogenetic proteins (BMP) signaling for the treatment of Fibrodysplasia ossificans progressiva (FOP). FOP is a rare disease, characterized by malformation of the great (big) toes during embryonic development. Individuals with FOP have an identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.

This technology includes the use of a new class of molecules (nanomolar ALK2 inhibitor) to impede bone morphogenetic proteins (BMP) signaling for the treatment of Fibrodysplasia ossificans progressiva (FOP). FOP is a rare disease, characterized by malformation of the great (big) toes during embryonic development. Individuals with FOP have identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.

This technology includes methods for screening compounds and compositions useful for inhibiting or reducing platelet deposition, adhesion, and/or aggregation. The present invention further relates to methods of treatment or prophylaxis of thrombotic disorders, including stroke, myocardial infarction, unstable angina, abrupt closure following angioplasty or stent placement, thrombosis induced by peripheral vascular surgery, peripheral vascular disease or thrombotic disorders resulting from atrial fibrillation or inflammation.

This technology includes a novel pyrazole-based compound NCGC00274266 (MLS000714501) that inhibits LDH-A with an IC50 of approximately 20 µM with low efficacy that can be used as an anti-cancer therapeutic. Structure-activity relationship studies on this compound led to hydroxypryazole-based compounds and discovery that the hydroxypyrazole compound and related analogs demonstrated a strong metal-dependent activity.

This technology includes a series of diphenylpropanamides as potent and selective RORgt inhibitors for the treatment of Th17-related autoimmune diseases. The retinoic acid-related orphan receptor RORgt plays an important role in the differentiation of thymocytes, lymphoid tissue inducer cells, and inflammatory T helper-expressing interleukin 17a (Th17) cells. Small molecule RORgt inhibitors may provide means to regulate Th17 mediated immune response. The novel molecules have potential to treat Th17-related autoimmune diseases.

This technology includes LDHA inhibitors that have been synthesized and tested for the treatment of glycolytic cancers. These compounds may have improved activity over previous compounds.

This technology includes novel systemic adeno-associated virus (AAV)-mediated CRISPR gene therapy technology. While some diseases (e.g., retinal diseases) can be treated through local gene transfer, many diseases such as Duchenne Muscular Dystrophy (DMD) require systemic therapy. The CRISPR technology has two components, the Cas9 endonuclease, and the gRNA. To explore systemic CRISPR therapy, we co-delivered the AAV.Cas9 and AAV.gRNA vector to mdx mice, a mouse DMD model. Direct delivery to muscle yielded efficient gene correction.