公开(公告)号：US20240316165A1

公开(公告)日：2024-09-26

申请号：US18578052

申请日：2022-07-12

Applicant: ModernaTX, Inc.

Inventor： Husain Attarwala ,  Lei Jiang ,  Matthew Lumley

IPC: A61K38/53 ,  A61K9/51 ,  A61K31/167 ,  A61K31/192 ,  A61K47/24 ,  A61K48/00 ,  A61P3/00

CPC classification number: A61K38/53 ,  A61K9/5123 ,  A61K31/167 ,  A61K31/192 ,  A61K47/24 ,  A61K48/005 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of propionic acidemia. mRNAs for use in the invention, when administered in vivo, encode propionyl-CoA carboxy lase alpha (PCCA) and propionyl-CoA carboxy lase beta (PCCB). mRNA therapies of the disclosure increase and/or restore deficient levels of PCCA and/or PCCB expression and/or activity in subjects.

公开(公告)号：US20250090684A1

公开(公告)日：2025-03-20

申请号：US18292058

申请日：2022-07-26

Applicant: ModernaTX, Inc.

Inventor： Husain Attarwala ,  Jingsong Cao ,  Patrick Finn ,  Simone Mori

IPC: A61K48/00 ,  A61K38/46 ,  A61P3/08 ,  C12N9/16

Abstract: This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a (GSD1a). mRNAs for use in the invention, when administered in vivo, encode glucose-6-phosphatase (G6PC). mRNA therapies of the disclosure increase and/or restore deficient levels of G6PC expression and/or activity in subjects.

公开(公告)号：US20240269248A1

公开(公告)日：2024-08-15

申请号：US18560498

申请日：2022-05-19

Applicant: ModernaTX, Inc.

Inventor： Husain Attarwala ,  Kimberly Ann Coughlan ,  Ruchi Jain ,  Matthew Lumley ,  Vladimir Presnyak

IPC: A61K38/52 ,  A61K9/00 ,  A61K9/51 ,  A61K31/167 ,  A61K48/00 ,  A61P3/00

CPC classification number: A61K38/52 ,  A61K9/0019 ,  A61K9/5123 ,  A61K31/167 ,  A61K48/0033 ,  A61P3/00

Abstract: This disclosure relates to mRNA therapy for the treatment of methylmalonic acidemia (MMA). mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase (MUT). mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.