METHODS AND COMPOSITIONS FOR INCREASING α-IDURONIDASE ACTIVITY IN CENTRAL NERVOUS SYSTEM

Title:

METHODS AND COMPOSITIONS FOR INCREASING α-IDURONIDASE ACTIVITY IN CENTRAL NERVOUS SYSTEM

Document Type and Number:

Japanese Patent JP2023099190

Kind Code:

A

Abstract:

To provide methods and compositions for treating a subject suffering from a deficiency in α-L-Iduronidase in a central nervous system.SOLUTION: The present invention provides a fusion antibody having α-L-Iduronidase bound to a carboxy terminal of a heavy chain of a Fab fragment, wherein the Fab fragment has specificity for an extracellular domain of a receptor expressed on a blood-brain barrier, and the α-L-Iduronidase has at least 85% identical to an amino acid sequence represented by SEQ ID NO: 9, and a pharmaceutical composition that comprises the fusion protein and is used in the treatment of the CNS Hurler's syndrome or Scheie syndrome.SELECTED DRAWING: Figure 12

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Inventors:

WILLIAM M PARDRIDGE
RUBEN J BOADO

Application Number:

JP2023077007A

Publication Date:

July 11, 2023

Filing Date:

May 09, 2023

Export Citation:

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Assignee:

ARMAGEN INC

International Classes:

C07K19/00; A61K38/47; A61K39/395; A61K47/68; A61P25/00; C07K14/47; C07K16/28; C12N9/14

Attorney, Agent or Firm:

Yoshiki Yamamoto

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