CATION-INDEPENDENT MANNOSE-6-PHOSPHATE RECEPTOR BINDERS
The present invention relates to protein binding agents specifically binding the human cation- independent mannose-6-phosphate receptor, more specifically agents comprising an immunoglobulin single variable domain (ISVD) which allow internalisation upon binding to the extracellular N-terminal domain...
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Main Authors | , , , , , , |
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Format | Patent |
Language | English French German |
Published |
27.12.2023
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Subjects | |
Online Access | Get full text |
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Summary: | The present invention relates to protein binding agents specifically binding the human cation- independent mannose-6-phosphate receptor, more specifically agents comprising an immunoglobulin single variable domain (ISVD) which allow internalisation upon binding to the extracellular N-terminal domains 1, 2 and / or 3 in monovalent format. More specifically said ISVD provides for means and methods for lysosomal targeting, especially when fused to further proteins such as enzymes relevant for treatment of diseases caused by a lysosomal storage phenotype or lysosomal storage diseases. Finally the binding agents of the invention provide for use in therapeutic treatments, such as in Enzyme-replacement therapy, more specifically, when fused to human acid ot-glucosidase (hGAA) or human cathepsin D proteases for treatment of Pompe disease or sporadic inclusion body myositis or neuronal ceroid lipofuscinosis 10 (CLN10), respectively. |
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Bibliography: | Application Number: EP20220706854 |