公开(公告)号：US20230233475A1

公开(公告)日：2023-07-27

申请号：US17928684

申请日：2021-06-01

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Kristine Burke ,  Jingsong Cao ,  Paloma Hoban Giangrande ,  Edward J. Hennessy ,  Stephen Hoge ,  Jaclyn Milton ,  Staci Sabnis ,  Timothy Salerno ,  Matthew Theisen

IPC: A61K9/51 ,  A61K31/7088 ,  A61K9/00 ,  C12N9/16 ,  A61P35/00

CPC classification number: A61K9/5123 ,  A61K31/7088 ,  A61K9/0019 ,  C12Y301/03009 ,  C12N9/16 ,  A61P35/00

Abstract: This disclosure relates to ionizable 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 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.