Granted Patent
B2
US 12,239,742 · App. 17/154,325 · Granted Mar 4, 2025
Polynucleotides encoding Citrin for the treatment of Citrullinemia type 2
Inventors:
Paolo Martini (Boston, MA); Stephen Hoge (Cambridge, MA); Kerry Benenato (Cambridge, MA); Vladimir Presnyak (Manchester, NH); Iain McFadyen (Medford, MA); Ellalahewage Sathyajith Kumarasinghe (Cambridge, MA); Jingsong Cao (Cambridge, MA); Lin Tung Guey (Lexington, MA); Staci Sabnis (Cambridge, MA)
Assignee:
ModernaTX, Inc.
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Abstract
The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. 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 Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
Claims (4)
1. An mRNA comprising an open reading frame (ORF) encoding the human Citrin polypeptide of SEO ID NO: 1, wherein the ORF is 100% identical to the nucleotide sequence of SEQ ID NO:129.
2. A lipid nanoparticle comprising the mRNA of claim 1 , and a compound having the formula:
or a salt thereof.
3. A method of treating citrullinemia type 2 (CTLN2) in a human subject in need thereof, comprising administering to the human subject an effective amount of the lipid nanoparticle of claim 2 .
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 Feb 10, 2021
From: MARTINI, PAOLO; HOGE, STEPHEN; BENENATO, KERRY; PRESNYAK, VLADIMIR; MCFADYEN, IAIN; KUMARASINGHE, ELLALAHEWAGE SATHYAJITH; CAO, JINGSONG; GUEY, LIN TUNG; SABNIS, STACI
To: MODERNATX, INC.
Reel/Frame 055211/0547 →
Continuity (3)
Continuation
16302360
Provisional Application
62338479
· May 18, 2016
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