Methods for modulating RNA splicing

US11702646B2 · kind B2 · utility

Assignee

• PTC THERAPEUTICS, INC. · US

Inventors

• Anuradha Bhattacharyya · Edison, US
• Amal Dakka · Whitehouse Station, US
• Kerstin Effenberger · Metuchen, US
• Vijayalakshmi Gabbeta · Bound Brook Junction, US
• Wencheng Li · Mount Horeb, US
• Nikolai Naryshkin · East Brunswick Township, US
• Christopher Trotta · Somerville, US
• Kari Wiedinger · New Providence, US

Key dates

• Filing date: Nov 27, 2017
• Grant date: Jul 18, 2023
• Expiry date: Sep 16, 2040

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N15/63
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise an intronic REMS, and the use of a compound described herein to modulate protein produced from such altered endogenous genes.