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High Concentration Alpha-Glucosidase Compositions For The Treatment Of Pompe Disease

Application US20260083829A1 Kind: A1 Mar 26, 2026

Assignee

Amicus Therapeutics, Inc.

Inventors

Kenneth Valenzano, John Crowley, Richie Khanna, John Flanagan

Abstract

The present application provides for compositions comprising high concentrations of acid α-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.

CPC Classifications

A61K 38/47 A61K 9/0019 A61K 31/445 A61K 31/7008 A61K 47/22 C12N 9/2408 C12N 9/96 C12Y 302/0102

Filing Date

2025-09-22

Application No.

19335590