	US 12,377,136 B2
	Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
Paolo Martini, Boston, MA (US); Stephen Hoge, Brookline, MA (US); Kerry Benenato, Sudbury, MA (US); Vladimir Presnyak, Manchester, NH (US); Lei Jiang, Cambridge, MA (US); Iain McFadyen, Arlington, MA (US); Ellalahewage Sathyajith Kumarasinghe, Harvard, MA (US); Antonio Fontanellas Roma, Pamplona (ES); Pedro Berraondo Lopez, Pamplona (ES); Matias Antonio Avila Zaragoza, Pamplona (ES); Lin Tung Guey, Lexington, MA (US); and Staci Sabnis, Medford, MA (US)
Assigned to ModernaTX, Inc., Cambridge, MA (US); and Fundacion Para La Investigacion Medica Aplicada, Pamplona (ES)
Filed by ModernaTX, Inc., Cambridge, MA (US); and Fundacion Para La Investigacion Medica Aplicada, Pamplona (ES)
Filed on Jul. 21, 2022, as Appl. No. 17/813,984.
Application 17/813,984 is a continuation of application No. 16/302,339, abandoned, previously published as PCT/US2017/033418, filed on May 18, 2017.
Claims priority of application No. 17382259 (EP), filed on May 9, 2017.
Prior Publication US 2023/0112986 A1, Apr. 13, 2023
Int. Cl. A61K 38/43 (2006.01); A61K 38/45 (2006.01); A61K 48/00 (2006.01); A61P 7/08 (2006.01); C12N 9/10 (2006.01); C12N 15/88 (2006.01)

CPC A61K 38/45 (2013.01) [A61K 48/0033 (2013.01); A61K 48/005 (2013.01); A61P 7/08 (2018.01); C12N 9/1085 (2013.01); C12N 15/88 (2013.01); C12Y 205/01061 (2013.01)]

11 Claims

1. A method of treating an acute Porphyria attack in a human subject in need thereof, the method comprising administering to the human subject a lipid nanoparticle comprising a therapeutically effective amount of a messenger RNA (mRNA) encoding a porphobilinogen deaminase (PBGD) polypeptide, wherein the mRNA comprises an open reading frame comprising the nucleotide sequence set forth in SEQ ID NO: 102 or SEQ ID NO: 104, and wherein the human subject has acute intermittent Porphyria.