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This technology includes the use of novel lactate dehydrogenase (LDH) inhibitors, including WO2018005807A1, for the treatment of primary hyperoxalurias (PHs). PHs are rare autosomal recessive disorders caused by overproduction of oxalate, leading to recurrent calcium oxalate kidney stone disease, and in some cases end-stage renal disease. One potential strategy to treat PHs is to reduce the production of oxalate by diminishing the activity of LDH, the proposed key enzyme responsible for converting glyoxylate to oxalate.

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.

This technology includes the identification and use of novel inhibitors of ALDH1A1 (aldehyde dehydrogenase 1 family member A1) for the treatment of multiple diseases, including cancer, inflammation, and obesity. ALDH1A1 is an enzyme that has a role in alcohol metabolism, and has been implicated in maintaining cancer stem cells. A high-throughput screen was conducted that identified novel ALDH1A1 inhibitors.

This technology includes the use of a beclin 1 inhibitor, 17-hydroxy Wortmannin, for the treatment of TRAIL-resistant colon cancer. TRAIL (TNF-related apoptosis-inducing ligand) binds to death receptors (DR4/DR5) and activates apoptosis in cancer cells. Multiple clinical trials have focused on promoting TRAIL-induced death but have had a lack of efficacy due to TRAIL resistance developing quickly in cancer cells. Recent work has found that this resistance may be mediated by a lack of activation of the apoptosis/autophagy regulator beclin 1.

This technology includes the use of pyridines for anticancer treatment. A common feature of cancer cells is a high level of reactive oxygen species with a concomitant increase of two antioxidative systems to combat the toxicity: the glutathione and thioredoxin systems. Inhibiting either, or both, of these systems is a promising avenue to target cancer cells. Thioredoxin Reductase 1 (Trxr1) is an important selenoprotein in the thioredoxin antioxidative system which has been implicated as a potential anti-cancer target.