公开(公告)号：US20220243182A1

公开(公告)日：2022-08-04

申请号：US17275082

申请日：2019-09-12

Applicant: ModernaTX, Inc.

Inventor： Paolo G. V. Martini ,  Vladimir Presnyak ,  Paloma Hoban Giangrande ,  Kimberly Ann Coughlan

IPC: C12N9/02 ,  A61K9/51 ,  A61P3/00 ,  C12N9/10

Abstract: This disclosure relates to mRNA therapy for the treatment of maple syrup urine disease (MSUD). mRNAs for use in the invention, when administered in vivo, encode branched chain α-ketoacid dehydrogenase complex (BCKDC) E1α, E1β, or E2mRNA therapies of the disclosure increase and/or restore deficient levels of E1α, E1β, or E2 expression and/or BCKDC activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of branched chain amino acids associated with deficient BCKDC activity in subjects.

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.