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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Bibliographic Details
Main Authors VAN SCHIE, Loes, BLOCH, Yehudi, NERINCKX, Wim, NAESSENS, Justine, VAN LANDUYT, Linde, ROOSE, Kenny, CALLEWAERT, Nico
Format Patent
LanguageEnglish
French
German
Published 27.12.2023
Subjects
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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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.
Bibliography:Application Number: EP20220706854