IP Library › Granted Patent US 10,494,636
Granted Patent B2
US 10,494,636 · App. 16/110,833 · Granted Dec 3, 2019

Polynucleotides encoding α-galactosidase A for the treatment of Fabry disease

Inventors: Paolo Martini (Cambridge, MA); Stephen G. Hoge (Brookline, MA); Kerry Benenato (Sudbury, MA); Vladimir Presnyak (Manchester, NH); Iain McFadyen (Arlington, MA); Ellalahewage Sathyajith Kumarasinghe (Harvard, MA); Xuling Zhu (Cambridge, MA); Lin Tung Guey (Cambridge, MA); Staci Sabnis (Cambridge, MA)
Assignee: ModernaTX, Inc.
C12N15/67A61K9/5123A61K38/47A61P3/00C12N9/2465C12Y302/01022A61K48/00
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Quick Facts
Patent No.
US 10,494,636
App. No.
16/110,833
Granted
Dec 3, 2019
Kind
B2
Abstract

The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

Claims (10)

1. A lipid nanoparticle comprising a messenger RNA (mRNA) and a compound having the formula:

wherein the mRNA comprises

an open reading frame (ORF) encoding a human α-galactosidase A (GLA) polypeptide of SEQ ID NO: 1.

2. The lipid nanoparticle of claim 1 , wherein at least 95% of uridines in the ORF are 5-methoxyuridines.

3. The lipid nanoparticle of claim 1 , further comprising a phospholipid, a structural lipid, and a PEG lipid.

4. The lipid nanoparticle of claim 1 , wherein at least 99% of uridines in the ORF are 5-methoxyuridines.

5. The lipid nanoparticle of claim 1 , wherein 100% of uridines in the ORF are 5-methoxyuridines.

6. A pharmaceutical composition comprising the lipid nanoparticle of claim 1 .

7. A pharmaceutical composition comprising the lipid nanoparticle of claim 3 .

8. A pharmaceutical composition comprising the lipid nanoparticle of claim 5 .

Assignments (2)
SECURITY INTEREST Recorded Nov 19, 2025
From: MODERNATX, INC.
To: ARES CAPITAL CORPORATION, AS AGENT
Reel/Frame 073634/0354 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 28, 2018
From: MARTINI, PAOLO; HOGE, STEPHEN G.; BENENATO, KERRY; PRESNYAK, VLADIMIR; MCFADYEN, IAIN; KUMARASINGHE, ELLALAHEWAGE SATHYAJITH; ZHU, XULING; GUEY, LIN TUNG; SABNIS, STACI
To: MODERNATX, INC.
Reel/Frame 047002/0328 →