Non-human animals comprising a humanized TTR locus and methods of use

US12010979B2 · kind B2 · utility

Assignee

• REGENERON PHARMACEUTICALS, INC. · US

Inventors

• Meghan Drummond Samuelson · Tarrytown, US
• Jeffery Haines · New York, US
• Suzanne Hartford · Putnam Valley, US
• David Frendewey · New York, US
• Brian Zambrowicz · Diemelstraße, US
• Andrew J. Murphy · Croton-on-Hudson, US

Key dates

• Filing date: Nov 22, 2022
• Grant date: Jun 18, 2024
• Expiry date: Nov 22, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2710/10041
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus and methods of using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus express a human transthyretin protein or a chimeric transthyretin protein, fragments of which are from human transthyretin. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR. Methods are also provided for making such non-human animals comprising a humanized TTR locus.