USPTO Patent Grant for Trinucleotide Repeat Disease Treatment

ChangeBridge: Patent Grants - Organic Chemistry (C07D)

Published March 24th, 2026

Detected March 24th, 2026

Summary

The USPTO has granted a patent (US12583845B2) to The Research Foundation for The State University of New York for compositions and methods to treat diseases involving trinucleotide repeats. The patent covers novel compounds and synthesis processes aimed at inhibiting microsatellite promoted expression of deleterious expansions.

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What changed

The United States Patent and Trademark Office (USPTO) has issued patent US12583845B2 to The Research Foundation for The State University of New York. This patent covers novel compositions and methods for treating diseases associated with trinucleotide repeats, such as DM1. The disclosure includes modified polycyclic compounds designed to inhibit the expression of deleterious expansions by targeting expanded CTG DNA, with a focus on low nanomolar concentrations and negligible toxicity.

This patent grant signifies the protection of intellectual property related to a specific therapeutic approach. While not imposing direct compliance obligations on other entities, it establishes exclusive rights for the assignee. Companies operating in the pharmaceutical or biotechnology sectors, particularly those researching or developing treatments for genetic disorders involving trinucleotide repeats, should be aware of this patent's existence and scope to avoid potential infringement issues.

Source document (simplified)

← USPTO Patent Grants

Compositions and methods for treating diseases, disorders and conditions involving trinucleotide repeats

Grant US12583845B2 Kind: B2 Mar 24, 2026

Assignee

The Research Foundation for The State University of New York

Inventors

Hormoz Mazdiyasni, John Andrew Berglund

Abstract

The present disclosure provides compositions that inhibit microsatellite promoted expression of deleterious expansions by targeting expanded CTG DNA as well as methods using such compositions for use in treating or ameliorating the effects of a medical condition involving trinucleotide repeats in a subject. In embodiments, the present disclosure provides a method for treating a medical condition involving trinucleotide repeats, including a disease such as DM1, in a subject in need thereof by administering to the subject an effective amount of a modified polycyclic compound of the present disclosure. Processes for synthesizing modified polycyclic compounds that rescue mis-splicing at low nanomolar concentration with negligible toxicity are also disclosed.