[EN] LIPID NANOPARTICLES CONTAINING POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE AND USES THEREOF [FR] NANOPARTICULES LIPIDIQUES CONTENANT DES POLYNUCLÉOTIDES CODANT POUR LA GLUCOSE-6-PHOSPHATASE ET LEURS UTILISATIONS
[EN] NANOMATERIALS COMPRISING ESTER-LINKED ACETALS<br/>[FR] NANOMATÉRIAUX COMPRENANT DES ACÉTALS À LIAISON ESTER
申请人:BEAM THERAPEUTICS INC
公开号:WO2022140252A1
公开(公告)日:2022-06-30
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
本公开说明书描述了组合物、制剂、纳米颗粒(如脂质纳米颗粒)和/或纳米材料及其使用方法。
[EN] LIPID NANOPARTICLES CONTAINING POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE AND USES THEREOF<br/>[FR] NANOPARTICULES LIPIDIQUES CONTENANT DES POLYNUCLÉOTIDES CODANT POUR LA GLUCOSE-6-PHOSPHATASE ET LEURS UTILISATIONS
申请人:MODERNATX INC
公开号:WO2022204371A1
公开(公告)日:2022-09-29
This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and/or activity in subjects and are useful for the treatment of glycogen storage disease type 1a (GSD-Ia). Lipid nanoparticle/mRNA therapies of the invention increase glucose production and reduce the abnormal accumulation of glycogen and glucose-6-phosphate associated with GSD-Ia.
[EN] LIPID NANOPARTICLES CONTAINING POLYNUCLEOTIDES ENCODING PHENYLALANINE HYDROXYLASE AND USES THEREOF<br/>[FR] NANOPARTICULES LIPIDIQUES CONTENANT DES POLYNUCLÉOTIDES CODANT POUR LA PHÉNYLALANINE HYDROXYLASE ET LEURS UTILISATIONS
申请人:MODERNATX INC
公开号:WO2022204390A1
公开(公告)日:2022-09-29
This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding phenylalanine hydroxylase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of phenylalanine hydroxylase expression and/or activity in subjects and are useful for the treatment of phenylketonuria
[EN] BRANCHED TAIL LIPID COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTIC AGENTS<br/>[FR] COMPOSÉS LIPIDIQUES À QUEUE RAMIFIÉE ET COMPOSITIONS POUR L'ADMINISTRATION INTRACELLULAIRE D'AGENTS THÉRAPEUTIQUES
申请人:MODERNATX INC
公开号:WO2022204288A1
公开(公告)日:2022-09-29
The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) 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.