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...

Full description

Saved in:
Bibliographic Details
Main Authors CLARK, Roger B, ICHIKAWA, Yoshitaka, SHI, Shuhao, WANG, Wenying, JEWETT, Ivan T, AUSTIN, Wesley Francis, LI, Xiben
Format Patent
LanguageEnglish
Published 15.08.2024
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
Online AccessGet full text

Cover

More Information
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.
Bibliography:Application Number: AU20220283443