US 11,939,601 B2
Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
Raj Rajendran, Sharon, MA (US); Patrick Finn, Franklin, MA (US); Paolo G. V. Martini, Boston, MA (US); Ding An, Waban, MA (US); Athanasios Dousis, Boston, MA (US); and Kanchana Ravichandran, Cambridge, MA (US)
Assigned to ModernaTX, Inc., Cambridge, MA (US)
Appl. No. 16/765,656
Filed by ModernaTX, Inc., Cambridge, MA (US)
PCT Filed Nov. 21, 2018, PCT No. PCT/US2018/062237
§ 371(c)(1), (2) Date May 20, 2020,
PCT Pub. No. WO2019/104160, PCT Pub. Date May 31, 2019.
Claims priority of provisional application 62/590,128, filed on Nov. 22, 2017.
Claims priority of provisional application 62/679,081, filed on Jun. 1, 2018.
Prior Publication US 2021/0040456 A1, Feb. 11, 2021
Int. Cl. A61P 3/00 (2006.01); A61K 9/00 (2006.01); A61K 9/51 (2006.01); A61K 48/00 (2006.01); C12N 9/02 (2006.01)
CPC C12N 9/0071 (2013.01) [A61K 9/0019 (2013.01); A61K 9/5123 (2013.01); A61K 48/0075 (2013.01); A61P 3/00 (2018.01); C12Y 114/16001 (2013.01)] 19 Claims
 
1. A method of treating phenylketonuria in a human subject that has phenylketonuria, comprising administering to the human subject an effective amount of a pharmaceutical composition comprising a lipid nanoparticle comprising

OG Complex Work Unit Chemistry
or a salt thereof, wherein the pharmaceutical composition comprises an mRNA comprising an open reading frame encoding a phenylalanine hydroxylase polypeptide.