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Patent Application: Alpha-Glucosidase Compositions for Pompe Disease Treatment

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Published September 22nd, 2025
Detected March 27th, 2026
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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)

← USPTO Patent Applications

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

View original document →

Classification

Agency
USPTO
Published
September 22nd, 2025
Instrument
Notice
Legal weight
Non-binding
Stage
Draft
Change scope
Minor
Document ID
US20260083829A1

Who this affects

Applies to
Drug manufacturers
Industry sector
3254 Pharmaceutical Manufacturing
Activity scope
Drug Development Therapeutic Composition Manufacturing
Geographic scope
United States US

Taxonomy

Primary area
Pharmaceuticals
Operational domain
Legal
Compliance frameworks
FDA 21 CFR Part 11 GxP
Topics
Drug Development Rare Diseases

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