公开(公告)号：US20240189449A1

公开(公告)日：2024-06-13

申请号：US18282647

申请日：2022-03-24

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Alicia Anne Bicknell ,  Mark Cornebise ,  Athanasios Dousis ,  Andrew Jacob Giessel ,  Edward Hennessy ,  Ruchi Jain ,  Caroline Köhrer ,  Stuart Spencer Licht ,  Kanchana Ravichandran ,  David Reid

IPC: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105 ,  A61P7/00 ,  C12N9/10 ,  C12N15/67

CPC classification number: A61K48/005 ,  A61K9/1271 ,  A61K9/5123 ,  A61K31/7105 ,  A61K48/0041 ,  A61P7/00 ,  C12N9/1018 ,  C12N15/67 ,  C12Y201/03003

Abstract: This disclosure relates to mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC). mRNA therapies of the disclosure increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the disclosure further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.