Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome

US12128113B2 · kind B2 · utility

Assignee

• ModernaTX, Inc. · US

Inventors

• Kerry Benenato · Sudbury, US
• Stephen Hoge · Brookline, US
• Paolo Martini · Boston, US
• Iain McFadyen · Arlington, US
• Vladimir Presnyak · Manchester, US
• Ding An · Newton, US
• Ellalahewage Sathyajith Kumarasinghe · Harvard, US

Key dates

• Filing date: May 18, 2017
• Grant date: Oct 29, 2024
• Expiry date: Apr 30, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C07K14/705
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The invention relates to mRNA therapy for the treatment of Alagille syndrome (ALGS), mRNAs for use in the invention, when administered in vivo, encode JAGGED 1 (JAG1), isoforms thereof functional fragments thereof, and fusion proteins comprising JAG1, 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 JAG1 expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient JAG1 activity in subjects.