Provided herein are prodrugs of opioid receptor antagonists such as nalmefene and naltrexone, pharmaceutical compositions comprising said compounds, and methods for using said compounds for the treatment of behavioral disorders.
Breusch; Baykut, Chemische Berichte, 1957, vol. 90, p. 526,528,531
作者:Breusch、Baykut
DOI:——
日期:——
COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS
申请人:ModernaTX, Inc.
公开号:US20210198200A1
公开(公告)日:2021-07-01
The disclosure features amino lipids and compositions involving the same. Nanoparticle compositions include an amino lipid as well as additional lipids such as phospholipids, structural lipids, PEG lipids, or a combination thereof. Nanoparticle compositions further including therapeutic and/or prophylactic agents such as RNA are useful in the delivery of therapeutic and/or prophylactic agents to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
[EN] POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE<br/>[FR] POLYNUCLÉOTIDES CODANT POUR L'Α-GALACTOSIDASE A POUR LE TRAITEMENT DE LA MALADIE DE FABRY
申请人:MODERNATX INC
公开号:WO2017201328A1
公开(公告)日:2017-11-23
The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
[EN] BRANCHED TAIL LIPID COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTIC AGENTS<br/>[FR] COMPOSÉS LIPIDIQUES DE QUEUE RAMIFIÉS ET COMPOSITIONS POUR L'ADMINISTRATION INTRACELLULAIRE D'AGENTS THÉRAPEUTIQUES
申请人:MODERNATX INC
公开号:WO2021055833A1
公开(公告)日:2021-03-25
The application relates to lipids of Formula (A), Formula (B) and Formula (1-1), and to lipid nanoparticles (empty or loaded LNPs) including such a lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.