Invention Application
- Patent Title: POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE
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Application No.: US17275053Application Date: 2019-09-12
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Publication No.: US20230009009A1Publication Date: 2023-01-12
- Inventor: Paolo G. V. Martini , Athanasios Dousis , Vladimir Presnyak , Jingsong Cao
- Applicant: ModernaTX, Inc.
- Applicant Address: US MA Cambridge
- Assignee: ModernaTX, Inc.
- Current Assignee: ModernaTX, Inc.
- Current Assignee Address: US MA Cambridge
- International Application: PCT/US19/50827 WO 20190912
- Main IPC: A61K9/51
- IPC: A61K9/51 ; A61K38/46 ; A61P3/06 ; A61K9/00 ; C12N9/16

Abstract:
This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.
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