POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE TYPE 1A (GSD1A)

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United States of America

APP PUB NO 20250090684A1
SERIAL NO

18292058

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Abstract

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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.

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MODERNATX INC200 TECHNOLOGY SQUARE CAMBRIDGE MA 02139

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Attarwala, Husain Billerica, US 4 31
Cao, Jingsong West Roxbury, US 8 94
Finn, Patrick Franklin, US 24 163
Mori, Simone San Diego, US 1 0

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