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

US11649461B2 · 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
• Xuling Zhu · Cambridge, US
• Lin Tung Guey · Lexington, US
• Staci Sabnis · Somerville, US

Key dates

• Filing date: Sep 13, 2019
• Grant date: May 16, 2023
• Expiry date: Sep 13, 2039

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61K48/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.