Patent Application: Alpha-Glucosidase Compositions for Pompe Disease Treatment
Summary
The USPTO has published a new patent application (US20260083829A1) from Amicus Therapeutics, Inc. The application details compositions comprising high concentrations of acid alpha-glucosidase and active site-specific chaperones for treating Pompe disease. It also covers methods for increasing enzyme stability.
What changed
This document is a published patent application (US20260083829A1) filed by Amicus Therapeutics, Inc. It claims novel compositions containing high concentrations of acid alpha-glucosidase combined with an active site-specific chaperone, intended for the treatment of Pompe disease. The application also describes methods for enhancing the stability of acid alpha-glucosidase enzyme formulations, both in vitro and in vivo.
As this is a patent application, it does not impose direct regulatory obligations or compliance deadlines on entities. However, it signifies potential future intellectual property protection for specific therapeutic compositions and methods related to Pompe disease treatment. Companies operating in the rare disease and enzyme replacement therapy space should be aware of this filing as it may impact their research and development strategies or potential licensing opportunities.
Source document (simplified)
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
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