[EN] LNP COMPOSITIONS COMPRISING PAYLOADS FOR IN VIVO THERAPY [FR] COMPOSITIONS DE NANOPARTICULES LIPIDIQUES COMPRENANT DES CHARGES UTILES POUR LA THÉRAPIE IN VIVO
The present invention provides a technique capable of transferring an active ingredient, particularly, a nucleic acid, to a cell with excellent efficiency and a cationic lipid for use in this technique, etc. The cationic lipid of the present invention is a compound represented by the formula (I) or a salt thereof. n1 represents an integer of 2 to 6, n2 represents an integer of 0 to 2, n3 represents an integer of 0 to 2, L represents —C(O)O— or —NHC(O)O—, Ra represents a linear C
5-13
alkyl group, a linear C
13-17
alkenyl group or a linear C
17
alkadienyl group, Rb represents a linear C
2-9
alkyl group, Rc represents a hydrogen atom or a linear C
2-9
alkyl group, Rd represents a hydrogen atom or a linear C
2-9
alkyl group, Re represents a linear C
2-9
alkyl group, and Rf represents a linear C
2-9
alkyl group.
[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