Polynucleotides encoding citrin for the treatment of Citrullinemia type 2

US10993918B2 · kind B2 · utility

Assignee

• ModernaTX, Inc. · US

Inventors

• Paolo Martini · Boston, US
• Stephen Hoge · Brookline, US
• Kerry Benenato · Sudbury, US
• Vladimir Presnyak · Manchester, US
• Iain McFadyen · Arlington, US
• Ellalahewage Sathyajith Kumarasinghe · Harvard, US
• Jingsong Cao · Cambridge, US
• Lin Tung Guey · Lexington, US
• Staci Sabnis · Somerville, US

Key dates

• Filing date: May 18, 2017
• Grant date: May 4, 2021
• Expiry date: Jun 7, 2037

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61K38/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.