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The high-throughput screening technology developed by Zacharon Pharmaceuticals can be applied to identify and develop small molecule drugs across a variety of glycan classes with a range of therapeutic applications. Zacharon is initially focusing its research and development activities in the following areas:
Glycosaminoglycans
Glycosaminoglycans are linear glycans that contain alternating amino sugars and uronic acids. Drugs targeting glycosaminoglycans have many potential therapeutic uses including inhibition of tumor growth and angiogenesis, repair of spinal cord injuries, and lysosomal storage diseases [1-4]. For additional information related to Zacharon’s therapeutic strategy in lysosomal storage disease, please click here.
O-linked glycans
Several classes of O-linked glycans exist, the classical type containing a glycan attached to proteins via a-N-acetylgalactosamine to the hydroxyl group of serine/threonine side chains. Drugs targeting O-linked glycans have strong promise as therapeutics for treating various forms of cancer and inflammation [1, 5-11]
Gangliosides
Gangliosides (GSLs) are compounds composed of a glycosphingolipid (ceramide and oligosaccharide) with one or more sialic acids (AKA n-acetylneuraminic acid, NANA) linked on the sugar chain. The role of GSLs in the pathogenesis of cancer and lipid storage diseases has been well-documented, making GSLs a compelling therapeutic target [12-17].
N-linked glycans
Many glycoproteins contain glycans linked through a glycosylamine bond to asparagines residues. These N-Linked glycans play central roles in protein quality control with the endoplasmic reticulum and Golgi. N-linked glycans have been implicated in cancer and other diseases [2, 18].
- Fuster, M.M. and J.D. Esko, The Sweet and Sour of Cancer: Glycans as Novel Therapeutic Targets. Nature Reviews in Cancer, 2005. 5: p. 526-542.
- Brown, J.R., B.E. Crawford, and J.D. Esko, Glycan antagonists and inhibitors: a fount for drug discovery. Crit Rev Biochem Mol Biol, 2007. 42(6): p. 481-515.
- Fuster, M.M., et al., Genetic alteration of endothelial heparan sulfate selectively inhibits tumor angiogenesis. J Cell Biol, 2007. 177(3): p. 539-49.
- Schuksz, M., Surfen, a small molecule antagonist of heparan sulfate. PNAS, 2008. 105(35): p. 13075-13080.
- Kim, Y.J., et al., P-selectin deficiency attenuates tumor growth and metastasis. Proc Natl Acad Sci U S A, 1998. 95(16): p. 9325-30.
- Kim, Y.J., et al., Distinct selectin ligands on colon carcinoma mucins can mediate pathological interactions among platelets, leukocytes, and endothelium. Am J Pathol, 1999. 155(2): p. 461-72.
- Shirota, K., Anti-metastatic effect of the sialyl Lewis-X analog GSC-150 on the human colon carcinoma derived cell line KM12-HX in the mouse. Biol Pharm Bull, 2001. 24: p. 316-319.
- Ulbrich, H.K., A novel class of potent nonglycosidic and nonpeptidid pan-selectin inhibitors. J Med Chem, 2006. 49: p. 5988-5999.
- Beeh, K.M., Bimosiamose, an inhaled small-molecule pan-selectin antagonist, attenuates late asthmatic reactions following allergen challenge in mild asthmatics: a randomized, double-blind, placebo-controlled clinical cross-over trial. Pulm Pharmacol Ther, 2006. 19: p. 233-241.
- Ikegami-Kuzuhara, A., Therapeutic potential of a novel synthetic selectin blocker, OJ-R9188, in allergic dermatitis. Br J Pharmacol, 2001. 134: p. 1498-1504.
- Brown, J.R., et al., A disaccharide-based inhibitor of glycosylation attenuates metastatic tumor cell dissemination. Clin Cancer Res, 2006. 12(9): p. 2894-901.
- Valentino, L., et al., Shed tumor gangliosides and progression of human neuroblastoma. Blood, 1990. 75(7): p. 1564-7.
- Deng, W., R. Li, and S. Ladisch, Influence of cellular ganglioside depletion on tumor formation. J Natl Cancer Inst, 2000. 92(11): p. 912-7.
- Weiss, M., et al., Inhibition of melanoma tumor growth by a novel inhibitor of glucosylceramide synthase. Cancer Res, 2003. 63(13): p. 3654-8.
- Zeng, G., et al., Alteration of ganglioside composition by stable transfection with antisense vectors against GD3-synthase gene expression. Biochemistry, 1999. 38(27): p. 8762-9.
- Zeng, G., L. Gao, and R.K. Yu, Reduced cell migration, tumor growth and experimental metastasis of rat F-11 cells whose expression of GD3-synthase is suppressed. Int J Cancer, 2000. 88(1): p. 53-7.
- Scriver, ed. The Metabolic & Molecular Bases of Inherited Disease. 8th ed. 2001.
- Granovsky, M., et al., Suppression of tumor growth and metastasis in Mgat5-deficient mice. Nat Med, 2000. 6(3): p. 306-12.
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