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This technology includes a precise measurement assay of cellular FMRP levels in patients, which can assist in the diagnosis and assess the severity of Fragile X syndrome (FXS). FXS is an X-linked disorder that produces intellectual disability, cognitive impairment, epilepsy, depression and anxiety. FXS is caused by mutations in the Fragile X Mental Retardation-1 (FMR1) gene that result in the absence or a loss of function of its protein product, FMRP.

This technology includes a group of eight AMPK activating compounds to be further developed for the treatment of Niemann Pick Type C (NPC) disease. Through the recent molecular biology and pharmacological experiments, we have identified the cyclodextrin which directly binds to beta-subunits of AMP-activated protein kinase (AMP), resulting in subsequently activations of AMPK and AMPK linked autophagy, and restoration of autophagy function that is impaired in the NPC cells.

This technology includes newly identified best-in-class inhibitors of the p53-S100B interaction that plays a role in malignant melanoma. S100B contributes to cancer cell proliferation (particularly malignant melanoma) by binding to p53 and inhibiting its tumor suppressor function. A high-throughput screen was used to find p53-S100B inhibitors, leading to the identification of a putative inhibitor, which was then subjected to medicinal chemistry optimization. Structure-based design was then used to develop compounds with significantly improved potency.

This technology includes a cell line that expresses two reporters (a secreted luciferase, secNLuc, and GFP) in a pattern that recapitulates the endogenous expression of the peripheral myelin protein 22 (Pmp22) gene. Pmp22 is mainly expressed in the Schwann cells of the peripheral nervous system. Many neurological disorders are associated with aberrations in Schwann cells, including the most common inherited peripheral neuropathy known as Charcot-Marie-Tooth (CMT) disease. This cell line will permit the study of the regulatory elements behind the gene.

This technology includes the generation and use of human induced pluripotent stem cell (iPSC) lines that can be used to study and screen potential therapeutics for lysosomal storage diseases (LSDs). LSDs are a group of 50 genetic disorders caused by mutations in the genes encoding lysosomal enzymes and proteins. Although various therapeutic approaches exist, most cases of LSDs are not effectively treated due to a lack of therapeutics (including stem cells and recombinant proteins).

This technology includes the use of a novel formulation for an engineered version of Fibroblast Growth Factor 1 (FGF1), TTHX1114, that can be used to accelerate regeneration of the corneal endothelium after surgical lesions. FGFs are well-established regulators of migration and proliferation of corneal endothelial cells (CECs).

This technology includes a series of imidazo[1,2-b]pyridazines that display potent inhibition of FLT3, as well as potent binding and activity against FLT3 tyrosine kinase domain and gatekeeper mutations. This chemotype exhibits superior anti-leukemic activity against the common clinically-relevant FLT3-mutant acute myeloid leukemia (AML) in vitro and in vivo. Tyrosine kinase domain mutations are a common cause of acquired resistance to FLT3 inhibitors used to treat FLT3-mutant AML.

This technology includes the identification and use of a novel small molecule, NCGC1481, to inhibit both the FLT3 and IRAK1/4 kinase pathways for treating acute myeloid leukemia (AML). An activating mutation of the FMS-like receptor kinase 3 (FMT3) occurs in approximately 25% of AML cases. Consequently, FLT3 inhibitors (FLT3i) have a good initial clinical response, however patients relapse with FLT3i-resistance. This adaptive resistance following FLT3i treatment is partially conferred by activation of the IRAK1/4 kinase complex.

The technology includes the creation of a high-throughput assay and the identification and use of small molecules that inhibit the SIX/EYA interaction as a treatment for cancer. The Eya proteins are phosphatases that form a complex and are activated by the Six family of homeobox transcription factors. The interaction of Eya and Six mediates breast cancer cell transformation, migration, invasion and metastasis. An assay was designed to screen a large collection of compounds to identify inhibitors of the SIX/EYA interaction.

This technology includes a novel computational algorithm and software implementation to map and display biological pathways and their relationship on the surface of a globe in a three-dimensional space. Currently, biological pathways and genes are represented as two-dimensional networks, which is not effective for displaying complicated relationships between pathways and genes.