Actelion Pharmaceuticals Ltd

Monsanto Company

The Chancellor, Masters and Scholars of the University of Oxford

Terence D Butters

Raymond A Dwek

Frances M Platt

Michael Glen Orchard

George W J Fleet

Martin L Bryant

Gunilla B Karlsson

Simon J Davis

Mylvaganam Jeyakumar

David Priestman

Frances Mary Platt

George Fleet

Pharmaceutically active piperidine derivatives

A method for treating a glycolipid storage-related disorder, comprising administering a therapeutically effective amount of an inhibitor of glycolipid synthesis in combination with an agent capable of increasing the rate of glycolipid degradation or in combination with bone marrow transplantation. Inhibitors of glycolipid synthesis include N-butyldeoxynojirimycin (NB-DNJ), N-butyldeoxygalactonojirimycin (NB-DGJ) or N-nonyldeoxynojirimycin (NN-DNJ). Glycolipid storage-related disorders include Gaucher disease, Sandhoff's disease, Fabry's disease, Tay-Sach's disease, Niemann-Pick C storage disease, GM1 gangliosidosis, genetic disorders in which neuronal glycolipid accumulation contributes to disease pathology.

Therapeutic compositions and methods of treating glycolipid storage related disorders

A method is disclosed for modifying the carbohydrate moiety on glycoproteins to facilitate the structural and functional analysis of said glycoproteins such as by NMR spectroscopic analysis and crystallography which comprises treating glycoprotein-secreting mammalian cells having low endomannosidase activity under cell culture maintenance conditions with a glucosidase I inhibitor, and after secretion and purification, subsequent treatment of the active glycoprotein with endoglycosidase H to thereby provide a glycoprotein with a single GlcNAc residue at each glycosylation sequon. The preferred mammalian cells are CHO cells and the preferred glucosidase I inhibitor is N-butyl deoxynojirimycin.

Method for carbohydrate engineering of glycoproteins

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Oxford, United Kingdom

Terence D Butters