Patents by Inventor Kerstin Effenberger

Kerstin Effenberger has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Publication number: 20230272367
    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.
    Type: Application
    Filed: May 12, 2023
    Publication date: August 31, 2023
    Applicant: PTC THERAPEUTICS, INC.
    Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
  • Patent number: 11702646
    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.
    Type: Grant
    Filed: November 27, 2017
    Date of Patent: July 18, 2023
    Assignee: PTC THERAPEUTICS, INC.
    Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
  • Patent number: 11608501
    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.
    Type: Grant
    Filed: June 13, 2018
    Date of Patent: March 21, 2023
    Assignee: PTC Therapeutics, Inc.
    Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Minakshi B. Jani, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
  • Publication number: 20200370043
    Abstract: Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small en 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.
    Type: Application
    Filed: June 13, 2018
    Publication date: November 26, 2020
    Applicant: PTC THERAPEUTICS, INC.
    Inventors: Anuradha BHATTACHARYYA, Amal DAKKA, Kerstin EFFENBERGER, Vijayalakshmi GABBETA, Minakshi B. JANI, Wencheng LI, Nikolai NARYSHKIN, Christopher TROTTA, Kari WIEDINGER
  • Publication number: 20190330615
    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.
    Type: Application
    Filed: November 27, 2017
    Publication date: October 31, 2019
    Applicant: PTC THERAPEUTICS, INC.
    Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger