COMPOUNDS FOR TREATING GENETIC DISEASES
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 co...
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Main Authors | , , , , , , |
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Format | Patent |
Language | English French German |
Published |
01.05.2024
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Subjects | |
Online Access | Get full text |
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Summary: | 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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Bibliography: | Application Number: EP20220736067 |