The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Mar. 21, 2023
Filed:
Jun. 13, 2018
Ptc Therapeutics, Inc., South Plainfield, NJ (US);
Anuradha Bhattacharyya, Edison, NJ (US);
Amal Dakka, Whitehouse Station, NJ (US);
Kerstin Effenberger, Metuchen, NJ (US);
Vijayalakshmi Gabbeta, Bridgewater, NJ (US);
Minakshi B. Jani, Iselin, NJ (US);
Wencheng Li, Martinsville, NJ (US);
Nikolai Naryshkin, East Brunswick, NJ (US);
Christopher Trotta, Somerset, NJ (US);
Kari Wiedinger, New Providence, NJ (US);
PTC Therapeutics, Inc., South Plainfield, NJ (US);
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.