RNA replicon for versatile and efficient gene expression

US11168337B2 · kind B2 · utility

Assignees

• Moniech RNA Pharmecenticais GmbH · DE
• TRON—Translationale Onkologie an der Universitätsmedizin der Johannes Gutenberg-Universität Mainz · DE

Inventors

• Tim Beissert · Groß-Gerau, DE
• Ugur Sahin · Mainz, DE
• Mario Perkovic · Frankfurt, DE

Key dates

• Filing date: Mar 13, 2017
• Grant date: Nov 9, 2021
• Expiry date: Jul 6, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2770/36143
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention embraces a RNA replicon that can be replicated by a replicase of alphavirus origin. The RNA replicon comprises sequence elements required for replication by the replicase, but these sequence elements do not encode any protein or fragment thereof, such as an alphavirus non-structural protein or fragment thereof. Thus, in the RNA replicon according to the invention, sequence elements required for replication by the replicase and protein-coding region(s) are uncoupled. According to the present invention the uncoupling is achieved by the removal of at least one initiation codon compared to a native alphavirus genomic RNA. In particular, the RNA replicon comprises a 5′ replication recognition sequence, wherein the 5′ replication recognition sequence is characterized in that it comprises the removal of at least one initiation codon compared to a native alphavirus 5′ replication recognition sequence. The replicase of alphavirus origin may be encoded by an open reading frame on the RNA replicon or on a separate RNA molecule. The present invention enables efficient and safe expression of a protein of interest in a cell or organism, but is not associated with undesired …