Compounds for Treating Genetic Diseases
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United States of America Patent
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Issued Date -
Aug 22, 2024
app pub date -
May 31, 2022
filing date -
May 28, 2021
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Abstract
Provided are 13-membered ribosome targeting compounds that can be used to treat genetic diseases, including genetic diseases that are associated with a premature termination codon mutation or other nonsense mutation. The compounds can induce and/or promote readthrough of the premature termination codon mutation. Also provided are pharmaceutical compositions containing the compounds, methods of using the compounds and processes for making the compounds.
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