The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Mar. 18, 2025
Filed:
Jan. 21, 2021
Modernatx, Inc., Cambridge, MA (US);
Paolo Martini, Boston, MA (US);
Stephen Hoge, Cambridge, MA (US);
Kerry Benenato, Cambridge, MA (US);
Vladimir Presnyak, Manchester, NH (US);
Iain McFadyen, Medford, MA (US);
Ellalahewage Sathyajith Kumarasinghe, Cambridge, MA (US);
Ding An, Waban, MA (US);
Staci Sabnis, Medford, MA (US);
ModernaTX, Inc., Cambridge, MA (US);
Abstract
The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).