Genzyme Corporation

Canwen Jiang

Seng H Cheng

Yunxiang Zhu

Luis Z Avila

Jozsef Karman

Nelson S Yew

Ronnie Rong Wei

Hongmei Zhao

Cynthia Marie Arbeeny

David J Harris

Edward R Lee

Mathieu B Lane

Ronnie Wei

Luis Z Avial

Seng H Chen

The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.

Inhibitors of T-cell activation

The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.

Oligosaccharides comprising an aminooxy group and conjugates thereof

The disclosure provides methods for treating fatty liver disease and associated conditions by inhibiting the synthesis of glucosphingolipids, as exemplified by the use of glucosylceramide synthase substrate analogs.

Methods of treating fatty liver disease

Cystic fibrosis transmembrane conductance regulator (CFTR) is a chloride (Cl,) channel regulated by phosphorylation and intracellular nucleotides. CFTR is the major Cl,transport pathway in airway epithelial cells. The abnormal transepithelial Cl,transport and subsequent defective fluid transport caused by CF is a result of the genetic mutations of the gene coding for the CFTR protein. The present invention is directed to the novel use of ionophores as artificial Cl,transport pathways into CF epithelia to treat the defective Cl,and fluid transport lonophores and in particular, small molecule ionophores, represent a potential novel means of treating CF. The invention also includes using an ionophore to generate chloride secretion on intact monolayers of airway epithelia cells and other epithelia cells by administering an ionophore to a mammal.

Small molecule chloride transport

Methods and compositions for treating CF by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.

Methods and compositions for treating cystic fibrosis

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Southborough, MA, United States of America

Canwen Jiang