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 CLARK, Roger B, ICHIKAWA, Yoshitaka, SHI, Shuhao, WANG, Wenying, JEWETT, Ivan T, AUSTIN, Wesley Francis, LI, Xiben
Format Patent
LanguageEnglish
French
Published 01.12.2022
Subjects
CHEMISTRY
DERIVATIVES THEREOF
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SUGARS
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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. L'invention concerne des composés ciblant des ribosomes à 13 chaînons qui peuvent être utilisés pour traiter des maladies génétiques, y compris des maladies génétiques qui sont associées à une mutation de codon de terminaison prématurée ou à une autre mutation non-sens. Les composés peuvent induire et/ou favoriser la lecture de la mutation de codon de terminaison prématurée. L'invention concerne également des compositions pharmaceutiques contenant les composés, des procédés d'utilisation des composés et des procédés de fabrication des composés.
Bibliography:Application Number: WO2022US31565