USPTO Patent Grant for Trinucleotide Repeat Disease Treatment
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.
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.
Archived snapshot
Mar 24, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
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.
CPC Classifications
C07D 405/14
Filing Date
2022-10-04
Application No.
17959826
Claims
7
Named provisions
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Source
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