Methods for modifying RNA splicing

US11608501B2 · 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
• Minakshi Jani · Iselin, 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: Jun 13, 2018
• Grant date: Mar 21, 2023
• Expiry date: Dec 16, 2039

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/30
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small molecule splicing modifier compound of Formula (I) provided herein or a form thereof, wherein W, X, A and B are as defined herein. In one aspect, methods for modifying RNA splicing to modulate the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene that contains an intronic REMS is modified utilizing a splicing modifier compound of Formula (I), are described herein. In another aspect, methods for modifying RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene is modified to comprise an intronic REMS utilizing a splicing modifier compound of Formula (I), are described herein.