5′-cap-trinucleotide- or higher oligonucleotide compounds and their use in stabilizing RNA, expressing proteins in therapy

US12281138B2 · kind B2 · utility

Assignee

• BIONTECH RNA PHARMACEUTICALS GMBH · DE

Inventors

• Andreas Kuhn · Mainz, DE
• Hiromi Muramatsu · Mainz, DE
• Katalin Kariko · Rydal, US
• Stephanie Fesser · Mainz, DE
• Ugur Sahin · Mainz, DE

Key dates

• Filing date: Mar 14, 2019
• Grant date: Apr 22, 2025
• Expiry date: May 3, 2041

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61K48/005
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention relates to 5′-cap compounds, in particular the stabilization of RNA by such 5′-cap compounds, and provides compositions, such as pharmaceutical compositions, and cells comprising an RNA which is modified with such a 5′-cap compound, as well as methods for producing a peptide or protein of interest using the compositions or cells according to the present invention. Furthermore, the present invention provides the RNA, compositions, or cells for use in therapy, in particular for use in a method of treating a disease or disorder by protein replacement therapy, genome engineering, genetic reprogramming, and immunotherapy; a method for increasing the stability of RNA in cells; a method for increasing the expression of RNA in cells; and a method for providing an RNA with a 5′-cap structure.