LIPID ASSEMBLIES COMPRISING ANIONIC LYSOLIPIDS AND USE THEREOF
申请人:Biomedical Research Foundation of the Academy
of Athens
公开号:EP2892505A1
公开(公告)日:2015-07-15
US9730893B2
申请人:——
公开号:US9730893B2
公开(公告)日:2017-08-15
[EN] LIPID ASSEMBLIES COMPRISING ANIONIC LYSOLIPIDS AND USE THEREOF<br/>[FR] ENSEMBLES LIPIDES COMPRENANT DES LYSOLIPIDES ANIONIQUES ET LEUR UTILISATION
申请人:BIOMEDICAL RES FOUNDATION OF THE ACADEMY OF ATHENS
公开号:WO2014037436A1
公开(公告)日:2014-03-13
The present invention relates to lipid assemblies, compositions, and liposomal delivery systems comprising single chain anionic lipids, and the use thereof in diagnosis and therapy. It involves the use of anionic lysolipids for modifying the surface charge of a cationic lipid composition consisting at least one type of cationic lipid, optionally in combination with one or more neutral lipids and/or one or more anionic lipids.
[EN] POLYNUCLEOTIDES ENCODING CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS<br/>[FR] POLYNUCLÉOTIDES CODANT POUR UN RÉGULATEUR DE CONDUCTANCE TRANSMEMBRANAIRE DE LA MUCOVISCIDOSE POUR LE TRAITEMENT DE LA MUCOVISCIDOSE
申请人:[en]MODERNATX, INC.
公开号:WO2022104131A1
公开(公告)日:2022-05-19
This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA or gene editing therapeutics for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects. Nucleic acid therapeutics (e.g., mRNAs) of the disclosure further decrease abnormal accumulation of ammonia associated with deficient CFTR activity in subjects.