Invention Publication
- Patent Title: LIPID NANOPARTICLES AND POLYNUCLEOTIDES ENCODING ORNITHINE TRANSCARBAMYLASE FOR THE TREATMENT OF ORNITHINE TRANSCARBAMYLASE DEFICIENCY
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Application No.: US18282647Application Date: 2022-03-24
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Publication No.: US20240189449A1Publication Date: 2024-06-13
- 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
- Applicant: ModernaTX, Inc.
- Applicant Address: US MA Cambridge
- Assignee: ModernaTX, Inc.
- Current Assignee: ModernaTX, Inc.
- Current Assignee Address: US MA Cambridge
- International Application: PCT/US2022/021690 2022.03.24
- Date entered country: 2023-09-18
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K9/127 ; A61K9/51 ; A61K31/7105 ; A61P7/00 ; C12N9/10 ; C12N15/67
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.
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