US 12,252,704 B2
Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
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); and Staci Sabnis, Medford, MA (US)
Assigned to ModernaTX, Inc., Cambridge, MA (US)
Filed by ModernaTX, Inc., Cambridge, MA (US)
Filed on Jan. 21, 2021, as Appl. No. 17/154,309.
Application 17/154,309 is a continuation of application No. 16/302,341, granted, now 11,001,861, previously published as PCT/US2017/033420, filed on May 18, 2017.
Claims priority of provisional application 62/338,437, filed on May 18, 2016.
Prior Publication US 2021/0269830 A1, Sep. 2, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 9/51 (2006.01); A61K 48/00 (2006.01); B82Y 5/00 (2011.01); C07K 14/14 (2006.01); C12N 9/10 (2006.01); C12N 9/12 (2006.01); C12N 15/88 (2006.01); G01N 33/68 (2006.01)
CPC C12N 15/88 (2013.01) [A61K 9/51 (2013.01); A61K 48/005 (2013.01); B82Y 5/00 (2013.01); C07K 14/14 (2013.01); C12N 9/1241 (2013.01); G01N 33/6851 (2013.01); C12Y 207/0701 (2013.01)] 23 Claims
 
1. An mRNA comprising an open reading frame (ORF) encoding a galactose-1-phosphate uridylyltransferase (GALT) polypeptide, wherein the ORF has at least 85% sequence identity to a nucleic acid sequence selected from the group consisting of SEQ ID NOs: 5 to 29 and 113 to 131.