Springfield, Illinois Patent of the Year – 2024/2025
Iris Medicine, Inc. has been awarded the 2024/2025 Patent of the Year for their groundbreaking approach to treating CAG repeat diseases. Their invention, detailed in U.S. Patent Application No. 20240425856, titled ‘Compositions and methods for treating cag repeat diseases’, introduces a novel class of double-stranded RNAs designed to target and modulate toxic RNA sequences associated with these disorders.
The patented technology involves engineered RNA molecules that specifically bind to expanded CAG repeat regions found in the RNA of affected genes. By incorporating carefully placed mismatches, these RNA strands form a double-stranded structure that disrupts the toxic RNA’s function. This mechanism aims to reduce the harmful effects of the expanded repeats, potentially alleviating symptoms and slowing disease progression.
What sets this invention apart is its precision and specificity. The design of the RNA strands allows for targeted intervention without affecting normal gene function, minimizing potential side effects. This approach holds promise for treating a range of neurological disorders caused by CAG repeat expansions, such as Huntington’s disease and certain spinocerebellar ataxias.
Iris Medicine, Inc.’s innovative RNA-based therapy represents a significant advancement in genetic medicine, offering hope for patients with previously untreatable conditions. By addressing the root cause of these diseases at the molecular level, this patent paves the way for more effective and personalized treatments in the future.
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