Patents Assigned to PTC Therapeutics, Inc.
  • Methods and agents for screening for compounds capable of modulating gene expression
    Patent number: 9476870
    Abstract: The invention relates to the fields of screening assays, compounds, and methods for altering gene expression and protein levels. In particular, the invention includes assays to screen for agents capable of modulating gene expression in a UTR-dependent manner and agents capable of modulating gene expression.
    Type: Grant
    Filed: May 22, 2015
    Date of Patent: October 25, 2016
    Assignee: PTC THERAPEUTICS, INC.
    Inventors: Liangxian Cao, Nikolai Naryshkin, Charles Romfo, Matthew C. Pellegrini, Anuradha Roy, Panayiota Trifillis, Christopher R. Trotta
  • Compositions for an orally active 1,2,4-oxadiazole for the treatment of disease
    Patent number: 9474743
    Abstract: The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.
    Type: Grant
    Filed: March 21, 2014
    Date of Patent: October 25, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Samit Hirawat, Langdon Miller
  • Compounds for Nonsense Suppression, and Methods for Their Use
    Publication number: 20160229818
    Abstract: The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
    Type: Application
    Filed: April 15, 2016
    Publication date: August 11, 2016
    Applicant: PTC Therapeutics, Inc.
    Inventors: Neil Almstead, Gary M. Karp, Richard Wilde, Ellen Welch, Hongyu Ren
  • Antibacterial compounds and methods for use
    Patent number: 9409905
    Abstract: The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens.
    Type: Grant
    Filed: August 29, 2012
    Date of Patent: August 9, 2016
    Assignee: PTC THERAPEUTICS, INC.
    Inventors: Arthur Branstrom, Vara Prasad Venkata Nagendra Josyula, Michael Andrew Arnold, Aleksey I. Gerasyuto, Gary Karp, Jiashi Wang, John David Baird, Guangming Chen, Olya Ginzburg, Jana Narasimhan, Srinivasa Peddi, Hongyan Qi, Sean Wesley Smith, Matthew G. Woll, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang
  • Compounds for treating spinal muscular atrophy
    Patent number: 9399649
    Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
    Type: Grant
    Filed: January 25, 2013
    Date of Patent: July 26, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Guangming Chen, Amal Dakka, Gary Mitchell Karp, Chunshi Li, Jana Narasimhan, Nikolai Naryshkin, Marla L. Weetall, Ellen Welch, Xin Zhao
  • Survival motor neuron gene (SMN2) mRNA constructs for post-transcription regulation
    Patent number: 9394539
    Abstract: The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of spinal muscular atrophy (SMA), in which said compounds up-regulate the post-transcriptional expression of SMN1 or SMN2.
    Type: Grant
    Filed: October 8, 2012
    Date of Patent: July 19, 2016
    Assignee: PTC THERAPEUTICS, INC.
    Inventors: Sergey V. Paushkin, Anuradha Bhattacharyya, Bansri S. Furia, Meenal Patel, Love Volkova
  • Methods of screening for compounds for treating muscular dystrophy using human ALPHA7 integrin mRNA translation regulation
    Patent number: 9376725
    Abstract: The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular mystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).
    Type: Grant
    Filed: April 24, 2014
    Date of Patent: June 28, 2016
    Assignee: PTC THERAPEUTICS, INC.
    Inventors: Westley J. Friesen, Nikolai Naryshkin, Meenal Patel, Charles Romfo, Yuki Tomizawa, Ellen Welch, Jin Zhuo
  • Compounds for treating spinal muscular atrophy
    Patent number: 9371336
    Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
    Type: Grant
    Filed: February 28, 2013
    Date of Patent: June 21, 2016
    Assignees: PTC Therapeutics, Inc., F. Hoffmann-La Roche AG
    Inventors: Chang-Sun Lee, Soongyu Choi, Gary Mitchell Karp, Hiroo Koyama, Hasane Ratni
  • Methods for treating cancer and non-neoplastic conditions
    Patent number: 9351964
    Abstract: Compounds, such as compounds of Formula (I), that selectively inhibit pathological production of human vascular endothelial growth factor (VEGF) and compositions comprising such Compounds are described. Compounds that inhibit viral replication or the production of viral RNA or DNA or viral protein and compositions comprising such Compounds are described. Also described are methods of reducing VEGF using such Compounds and methods for treating cancer and non-neoplastic conditions involving the administration of such Compounds. Further described are methods of inhibiting viral replication or the production of viral RNA or DNA or viral protein using such Compounds and methods for treating viral infections involving the administration of such Compounds. The Compounds may be administered as a single agent therapy or in combination with one or more additional therapies to a human in need of such treatments.
    Type: Grant
    Filed: May 27, 2010
    Date of Patent: May 31, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Neil Almstead, Tamil Arasu, Soongyu Choi, Liangxian Cao, Jeffrey Allen Campbell, Donald Corson, Thomas W. Davis, Jason D. Graci, Zhengxian Gu, Peter Seongwoo Hwang, William Lennox, Harry H. Miao, Langdon Miller, Young-Choon Moon, Hongyan Qi, Christopher Trotta, Marla L. Weetall
  • COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY
    Publication number: 20160145270
    Abstract: The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
    Type: Application
    Filed: June 23, 2014
    Publication date: May 26, 2016
    Applicants: F. Hoffmann-La Roche AG, PTC Therapeutics Inc.
    Inventors: Amal Dakka, Luke Green, Gary Karp, Jana Narasimhan, Nikolai Naryshkin, Emmanuel Pinard, Hongyan Qi, Hasane Ratni, Nicole Risher, Marla Weetall, Matthew Woll
  • Compounds for nonsense suppression, and methods for their use
    Patent number: 9315467
    Abstract: The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
    Type: Grant
    Filed: October 13, 2005
    Date of Patent: April 19, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Neil Almstead, Gary M. Karp, Richard Wilde, Ellen Welch, Hongyu Ren
  • Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4] oxadiazol-3-yl]-benzoic acid
    Patent number: 9309206
    Abstract: The present invention relates to crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid, pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay.
    Type: Grant
    Filed: April 25, 2014
    Date of Patent: April 12, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Neil G. Almstead, Peter Seongwoo Hwang, Young-Choon Moon
  • Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith
    Patent number: 9289398
    Abstract: The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.
    Type: Grant
    Filed: March 29, 2007
    Date of Patent: March 22, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Neil Almstead, Guangming Chen, Samit Hirawat, Peter Seongwoo Hwang, Gary M. Karp, Langdon Miller, Young-Choon Moon, Hongyu Ren, James J. Takasugi, Ellen M. Welch, Richard G. Wilde, Paul Kennedy
  • Tetrahydrocarbazoles as active agents for inhibiting VEGF production by translational control
    Patent number: 9271960
    Abstract: The present invention relates to methods, compounds, and compositions for inhibiting angiogenesis. More particularly, the present invention relates to methods, compounds, and compositions for inhibiting VEGF production.
    Type: Grant
    Filed: January 26, 2015
    Date of Patent: March 1, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: William Lennox, Hongyan Qi, Duck-Hyung Lee, Soongyu Choi, Young-Choon Moon
  • Compounds for nonsense suppression, and methods for their use
    Patent number: 9273076
    Abstract: The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.
    Type: Grant
    Filed: April 28, 2014
    Date of Patent: March 1, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Jeffrey A. Campbell, Atiyya Kahn, James Takasugi, Ellen Welch
  • Methods for dosing an orally active 1,2,4-oxadiazole
    Patent number: 9226919
    Abstract: The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.
    Type: Grant
    Filed: October 11, 2007
    Date of Patent: January 5, 2016
    Assignee: PTC Therapeutics, Inc.
    Inventors: Samit Hirawat, Langdon Miller
  • Compositions of 1,2,4-oxadiazol benzoic acid compounds and methods for their use
    Patent number: 9205088
    Abstract: Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
    Type: Grant
    Filed: January 28, 2015
    Date of Patent: December 8, 2015
    Assignee: PTC Therapeutics, Inc.
    Inventors: Gary Mitchell Karp, Seongwoo Hwang, Guangming Chen, Neil Gregory Almstead, Young-Choon Moon
  • METHODS FOR TREATING VIRAL INFECTIONS
    Publication number: 20150344877
    Abstract: The present invention relates to compounds that modulate ribosomal frameshifting and nucleic acid constructs for use in methods for identifying or validation such compounds. In particular, the present invention relates to the use of nucleic acid constructs to identify or validate compounds capable of modulating the efficiency of programmed ribosomal frameshifting and the use of said compounds to inhibit the replication or infectivity of viruses that employ programmed ribosomal frameshifting.
    Type: Application
    Filed: May 12, 2015
    Publication date: December 3, 2015
    Applicant: PTC THERAPEUTICS, INC.
    Inventors: Sergey V. Paushkin, Nikolai A. Naryshkin, Ellen Welch
  • Combinations of a HCV inhibitor such as bicyclic pyrrole derivatives and a therapeutic agent
    Patent number: 9198907
    Abstract: The present invention is directed to a combination product for treating or ameliorating hepatitis C virus (HCV) infection or disorders or symptoms associated therewith in a subject in need thereof comprising, a HCV inhibitor and one or more therapeutic agents selected from either or both a HCV protease inhibitor and one or more different therapeutic agents administered in combination in an effective amount to the subject.
    Type: Grant
    Filed: April 5, 2010
    Date of Patent: December 1, 2015
    Assignee: PTC Therapeutics, Inc.
    Inventors: Frederick C. Lahser, Zhengxian Gu
  • METHODS FOR TREATING SPINAL MUSCULAR ATROPHY
    Publication number: 20150283115
    Abstract: Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of said compounds to produce a stabilized SMN?Ex7 protein and treat Spinal Muscular Dystrophy.
    Type: Application
    Filed: February 12, 2015
    Publication date: October 8, 2015
    Applicant: PTC THERAPEUTICS, INC.
    Inventors: Sergey V. Paushkin, Nikolai A. Naryshkin, Ellen Welch