C10-ALKYLENE SUBSTITUTED 13-MEMBERED MACROLIDES AND USES THEREOF
申请人:Zikani Therapeutics, Inc.
公开号:EP3883647A1
公开(公告)日:2021-09-29
[EN] COMPOUNDS FOR TREATING GENETIC DISEASES<br/>[FR] COMPOSÉS POUR LE TRAITEMENT DE MALADIES GÉNÉTIQUES
申请人:ZIKANI THERAPEUTICS INC
公开号:WO2022251730A1
公开(公告)日:2022-12-01
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.