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This technology includes selective inhibitors of the human enzyme galactokinase (EC 2.7.1.6), which may be useful for the treatment of Galactosemia and other diseases caused by aberrant galactose metabolism, including cancer. These compounds inhibit the first step in galactose metabolism, thereby eliminating the build-up of toxic metabolites during the aberrant metabolism of galactose, as well as inhibitor the entry of galactose into glycolysis and other downstream assays.

This technology includes the identification and use of heterocyclic alcohol compounds, including RITA and N-BIC, for the treatment of SULT1A1-expression cancers. A high-throughput screen (qHTS) was performed using >1,000 caner cell lines identified a compound called YC-1 (also called Lificiguat) that is effective across cancer cell types that express the phase 2 detoxifying enzyme SULT1A1.

This technology includes the design, synthesis, and use of a novel chemical series for multiple treatments, including for treating cancer. A series of substituted bicyclic heteroaryl small molecules were found to be a potent inhibitor of bromodomain-containing protein 4 (BRD4) for multiple uses, including cancer. A BRD4 inhibitor is in a class of drugs known as BET inhibitors that are used broadly as anti-inflammatories and as anti-cancer agents. The chemical series exhibited less hepatocyte toxicity compared to existing treatments.

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.

3D spheroids have emerged as powerful drug discovery tools given their high-throughput screening (HTS) compatibility. The present invention presents a method for generating functional neural spheroids with differentiated human induced pluripotent stem cell (hiPSC)-derived neurons and astrocytes at cell type compositions mimicking specific regions of the human brain.

Although flaviviruses cause a great deal of human suffering and economic loss, there is a shortage of effective vaccines. The present invention is directed toward vector stage replication-defective flaviviruses that are replication-defective in mosquito vectors that transmit them to humans. The replication-defective flaviviruses of the present invention demonstrate a limited ability to replicate in the vector organisms that transmit flaviviruses from one host to another.

The invention relates to a dengue virus tetravalent vaccine containing a common 30-nucleotide deletion (delta30) in the 3'-untranslated region (UTR) of the genome of dengue virus serotypes 1, 2, 3, and 4. The previously identified delta30 attenuating mutation, created in dengue virus type 4 (DEN4) by the removal of 30 nucleotides from the 3'-UTR, is also capable of attenuating a wild-type strain of dengue virus type 1 (DEN1).

This invention relates to diphtheria toxin fusion proteins comprising a diphtheria toxin (DT) cell-killing component and a cell-binding component such as granulocyte macrophage colony-stimulating factor (GM-CSF), interleukin 2 (IL-2), or epidermal growth factor (EGF). Receptors for the latter three materials are present on many types of cancer cells; therefore, these fusion proteins bind preferentially to these cancer cells. A key feature is that these toxins are altered so as to require activation by a cell-surface protease that is overexpressed on many types of cancers.

A new conjugate vaccine for cholera has been developed. The invention includes a new method to conjugate the O-specific polysaccharide-core part of the bacterial lipopolysaccharide and protein subcomponents. Conventional technology has entailed chemical treatment of both components to introduce linkers, which made them amenable for covalent linking. The new method simplifies production by utilizing squaric acid chemistry for conjugating the free amine-containing species (e.g. polysaccharides) directly to amine-containing species (e.g.