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USPTO Patent Application for Compounds Targeting Pax3/Foxo1 Fusion Protein

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Summary

The USPTO has published patent application US20260083724A1, filed by Georgetown University, for novel compounds designed to target the PAX3::FOXO1 fusion protein. These compounds aim to reduce protein levels and treat fusion-positive rhabdomyosarcoma.

What changed

This document is a publication of a United States Patent and Trademark Office (USPTO) patent application, specifically US20260083724A1, filed by Georgetown University. The application describes compounds designed to bind to and reduce the levels of the PAX3::FOXO1 fusion protein, which is implicated in fusion-positive rhabdomyosarcoma (FP-RMS). The invention includes a binding moiety, a linker, and an E3 ubiquitin ligase binding moiety, with potential applications in treating FP-RMS and diseases caused by PAX3::FOXO1 overexpression.

As this is a patent application publication, it does not impose immediate regulatory obligations or compliance deadlines on entities. However, it signifies potential future developments in pharmaceutical research and development for rhabdomyosarcoma treatments. Companies involved in oncology drug development, particularly those researching targeted protein degradation therapies, should note this application as it may impact the intellectual property landscape and future therapeutic strategies in this area.

Source document (simplified)

← USPTO Patent Applications

COMPOUNDS TARGETING PAX3::FOXO1 FUSION PROTEIN

Application US20260083724A1 Kind: A1 Mar 26, 2026

Assignee

Georgetown University

Inventors

Aykut Üren, Jeffrey A. Toretsky

Abstract

Compounds comprising a PAX3::FOXO1 fusion protein binding moiety, a bivalent linker, and an E3 ubiquitin ligase binding moiety. The compounds can be used to reduce PAX3::FOXO1 protein levels in a subject with fusion-positive rhabdomyosarcoma (FP-RMS), to ubiquitinate a PAX3::FOXO1 fusion protein in a cell, to treat FP-RMS in a subject in need thereof, and/or to treat a disease caused by the overexpression of PAX3::FOXO1 protein in a subject in need thereof.

CPC Classifications

A61K 31/4709 A61P 35/00

Filing Date

2025-09-26

Application No.

19341825

View original document →

Classification

Agency
USPTO
Instrument
Notice
Legal weight
Non-binding
Stage
Final
Change scope
Minor
Document ID
US20260083724A1

Who this affects

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

Taxonomy

Primary area
Pharmaceuticals
Operational domain
Legal
Topics
Drug Development Oncology

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