Abstract: 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 inhibiting or reducing viral replication or the production of viral RNA or DNA or viral protein using such Compounds, and methods for treating viral infections, such as an RNA virus belonging to the family Coronaviridae, 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.
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
Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) 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 a 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 a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production.
Abstract: The present description relates to compounds useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno [3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.
Type:
Application
Filed:
December 7, 2020
Publication date:
February 23, 2023
Applicant:
PTC THERAPEUTICS, INC.
Inventors:
Nanjing ZHANG, Michael A. ARNOLD, Amal DAKKA, Gary Mitchell KARP, Tom Tuan LUONG, Christie MORRILL, Jana NARASIMHAN, Nikolai A. NARYSHKIN, Anthony TURPOFF, Jiashi WANG, Xiaoyan ZHANG
Abstract: Provided herein are methods for making an alpha-tocotrienol enriched tocol mixture, such as from a plant or plant-derived material. Also provided herein are simulated moving bed purification methods for tocotrienol compounds such as alpha tocotrienol.
Type:
Grant
Filed:
October 26, 2021
Date of Patent:
January 24, 2023
Assignee:
PTC THERAPEUTICS, INC.
Inventors:
Peter Giannousis, Paul Mollard, Noah M. Benjamin, Jeffrey D. Butler, Olivier Dapremont, James B. Falabella
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.
Abstract: A method of use for Cpd 1 as an inhibitor of dihydroorotate dehydrogenase (DHODH) function in treating or ameliorating a hematological cancer in a subject in need thereof comprising, administering an effective amount of Cpd 1 to the subject, having the structure: or a form or pharmaceutical composition thereof.
Abstract: The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
Type:
Grant
Filed:
June 5, 2018
Date of Patent:
August 9, 2022
Assignee:
PTC THERAPEUTICS, INC.
Inventors:
Matthew G. Woll, Lukiana Amedzo, Suresh Babu, Scott J. Barraza, Anuradha Bhattacharyya, Gary Mitchell Karp, Anthony R. Mazzotti, Jana Narasimhan, Jigar Patel, Anthony Turpoff, Zhenrong Xu
Abstract: The present description relates to a method or use of a compound for treating or ameliorating HD (Huntington's Disease) in a subject in need thereof comprising administering to the subject an effective amount of a compound of Formula (I): or a form thereof, wherein R1, R2, Ra, Rb and Rc are as defined herein. In particular, the present description relates to a method of use of a compound of Formula (I) or a form or composition thereof for treating or ameliorating HD in a subject in need thereof comprising, administering an effective amount of the compound or a form or composition thereof to the subject.
Type:
Grant
Filed:
June 27, 2018
Date of Patent:
July 26, 2022
Assignee:
PTC THERAPEUTICS, INC.
Inventors:
Anuradha Bhattacharyya, Minakshi B. Jani
Abstract: Provided herein are therapeutic and/or prophylactic compounds for mitochondrial or oxidative stress diseases such as cancer, amyotrophic lateral sclerosis, Creutzfeldt-Jakob disease, Machado-Joseph disease, spinocerebellar ataxia, Huntington disease, Parkinson disease, Alzheimer disease, myocardial infarction, cerebral infarction, diseases related to aging, diabetes, alcoholic liver injury, chronic obstructive pulmonary disease, mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes (MELAS), and the like, wherein the compound is represented by formula (1), or reduced forms thereof, or pharmaceutically acceptable salts thereof.
Abstract: The present description relates to a method or use of a compound for treating or ameliorating HD (Huntington's Disease) in a subject in need thereof comprising administering to the subject an effective amount of a compound of Formula (I): or a form thereof, wherein w1, w2, w3, w4, w5, w6 and w7 are as defined herein. In particular, the present description relates to a method of use of a compound of Formula (I) or a form or composition thereof for treating or ameliorating HD in a subject in need thereof comprising, administering an effective amount of the compound or a form or composition thereof to the subject.
Type:
Grant
Filed:
June 27, 2018
Date of Patent:
July 12, 2022
Assignee:
PTC THERAPEUTICS, INC.
Inventors:
Anuradha Bhattacharyya, Minakshi B. Jani, Matthew G. Woll, Nadiya Sydorenko
Abstract: An alternatively spliced intronic sequence is disclosed, the splicing of which can be induced in the presence of a small molecule, as described herein.
Type:
Application
Filed:
November 11, 2021
Publication date:
May 26, 2022
Applicant:
PTC Therapeutics, Inc.
Inventors:
Anuradha BHATTACHARYYA, Yaofeng CHENG, Kerstin Annemarie EFFENBERGER, Wencheng LI, Jana NARASIMHAN, Christopher Robert TROTTA, MARLA L. WEETALL, Matthew G. WOLL
Abstract: The present description relates to a tablet formulation of 7-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-5-fluoro-3-(piperidin-4-yl)benzo[e][1,2,4]triazine, a compound for use in treating Huntington's disease, and a method of making the same.
Abstract: The present description relates to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect described herein relates to substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.
Type:
Application
Filed:
February 10, 2020
Publication date:
May 5, 2022
Applicant:
PTC THERAPEUTICS, INC.
Inventors:
Jiashi WANG, Michael A. ARNOLD, Amal DAKKA, Gary Mitchell KARP, Jana NARASIMHAN, Nikolai A. NARYSHKIN, Nanjing ZHANG
Abstract: The present description relates' to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.
Type:
Application
Filed:
February 10, 2020
Publication date:
May 5, 2022
Applicant:
PTC THERAPEUTICS, INC.
Inventors:
Nanjing ZHANG, Michael A. ARNOLD, Amal DAKKA, Gary Mitchell KARP, Tom Tuan LUONG, Jana NARASIMHAN, Nikolai A. NARYSHKIN, Jiashi WANG, Xiaoyan ZHANG
Abstract: The invention discloses novel processes for production, enrichment and/or isolation of alpha-tocotrienol from source material comprising at least one non-alpha-tocotrienol, such as natural extracts comprising mixed tocotrienols.
Type:
Grant
Filed:
September 10, 2018
Date of Patent:
April 26, 2022
Assignee:
PTC THERAPEUTICS, INC.
Inventors:
Kieron E. Wesson, Andrew W. Hinman, Orion D. Jankowski
Abstract: Disclosed herein are polymorphic and amorphous forms of anhydrate, hydrate, and solvates of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide and methods of using such compositions for treating or suppressing oxidative stress disorders, including mitochondrial disorders, impaired energy processing disorders, neurodegenerative diseases and diseases of aging. Further disclosed are methods of making such polymorphic and amorphous forms.
Type:
Grant
Filed:
July 1, 2020
Date of Patent:
April 19, 2022
Assignee:
PTC Therapeutics, Inc.
Inventors:
Paul Mollard, Peter Giannousis, Shazad Suchit, Mahmoud Mirmehrabi
Abstract: Provided herein are methods for making an alpha-tocotrienol enriched tocol mixture, such as from a plant or plant-derived material. Also provided herein are simulated moving bed purification methods for tocotrienol compounds such as alpha tocotrienol.
Type:
Grant
Filed:
August 17, 2020
Date of Patent:
November 30, 2021
Assignee:
PTC Therapeutics, Inc.
Inventors:
Peter Giannousis, Paul Mollard, Noah M. Benjamin, Jeffrey D. Butler, Olivier Dapremont, James B. Falabella
Abstract: Amine substituted reverse pyrimidine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.
Type:
Grant
Filed:
September 26, 2019
Date of Patent:
November 23, 2021
Assignee:
PTC Therapeutics, Inc.
Inventors:
Chang-Sun Lee, Ramil Baiazitov, Liangxian Cao, Thomas W. Davis, Wu Du, Ronggang Liu, Young-Choon Moon, Steven D. Paget, Hongyu Ren, Nadiya Sydorenko, Richard Gerald Wilde
Abstract: Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of ?-synucleinopathies, tauopathies, ALS, traumatic brain injury, and ischemic-reperfusion related injuries.ury, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of the formula: or the hydroquinone form thereof; or a solvate or hydrate thereof.
Type:
Grant
Filed:
March 30, 2021
Date of Patent:
November 16, 2021
Assignee:
PTC THERAPEUTICS, INC.
Inventors:
Andrew W. Hinman, Charles R. Holst, Angela Minnella, Paul Mollard, Sean Pintchovski, Jeffrey K. Trimmer, Eric Torrey