Patents Assigned to PTC Therapeutics, Inc.
-
Patent number: 11938101Abstract: 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: GrantFiled: March 10, 2022Date of Patent: March 26, 2024Assignee: PTC THERAPEUTICS, INC.Inventors: Paul Mollard, Peter Giannousis, Shazad Suchit, Mahmoud Mirmehrabi, Christopher R. Cornell, Kieron E. Wesson
-
Publication number: 20240051968Abstract: The present description relates to methods of treating spinocerebellar ataxia type 3 (SCA3) using substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof.Type: ApplicationFiled: February 2, 2022Publication date: February 15, 2024Applicant: PTC Therapeutics, Inc.Inventors: GAYAN MIRIHANA ARACHCHILAGE, MICHAEL A. ARNOLD, SCOTT BARRAZA, ANURADHA BHATTACHARYYA, GARY MITCHELL KARP
-
Publication number: 20240041886Abstract: The present description relates to methods of treating spinocerebellar ataxia type 3 (SCA3) using substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.Type: ApplicationFiled: February 2, 2022Publication date: February 8, 2024Applicant: PTC Therapeutics, Inc.Inventors: GAYAN MIRIHANA ARACHCHILAGE, MICHAEL A. ARNOLD, SCOTT BARRAZA, ANURADHA BHATTACHARYYA, GARY MITCHELL KARP
-
Publication number: 20240043444Abstract: The present description relates to methods of treating spinocerebellar ataxia type 3 (SCA3) using substituted thieno[3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.Type: ApplicationFiled: February 2, 2022Publication date: February 8, 2024Applicant: PTC Therapeutics, Inc.Inventors: GAYAN MIRIHANA ARACHCHILAGE, MICHAEL A. ARNOLD, SCOTT BARRAZA, ANURADHA BHATTACHARYYA, GARY MITCHELL KARP
-
Publication number: 20240043422Abstract: Compounds 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: ApplicationFiled: March 24, 2023Publication date: February 8, 2024Applicant: PTC Therapeutics, Inc.Inventors: Jason D. Graci, Zhengxian Gu
-
Patent number: 11858941Abstract: 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 heterocyclic and 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: GrantFiled: June 25, 2019Date of Patent: January 2, 2024Assignee: PTC THERAPEUTICS, INC.Inventors: Nadiya Sydorenko, Md Rauful Alam, Michael A. Arnold, Suresh Babu, Anuradha Bhattacharyya, Guangming Chen, Aleksey I. Gerasyuto, Gary Mitchell Karp, Andrew J. Kassick, Anthony R. Mazzotti, Young-Choon Moon, Jana Narasimhan, Jigar Patel, Anthony Turpoff, Matthew G. Woll, Wuming Yan, Nanjing Zhang
-
Patent number: 11840497Abstract: Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, and stroke (MELAS), Kearns-Sayre Syndrome (KSS), are disclosed, as well as compounds useful in the methods of the invention, such as 4-(p-quinolyl)-2-hydroxybutanamide derivatives. Methods and compounds useful in treating other disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, Parkinson's disease, and pervasive developmental disorders such as autism are also disclosed. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed. Methods of modulating, normalizing, or enhancing energy biomarkers, as well as compounds useful for such methods, are also disclosed.Type: GrantFiled: February 22, 2021Date of Patent: December 12, 2023Assignee: PTC Therapeutics, Inc.Inventors: Orion D. Jankowski, Kieron E. Wesson, Paul Mollard, William D. Shrader
-
Patent number: 11827646Abstract: The present invention provides compounds of formula (I) wherein A, R1, R2 and R3 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: GrantFiled: November 16, 2020Date of Patent: November 28, 2023Assignees: Hoffmann-La Roche Inc., PTC Therapeutics, Inc.Inventors: Hasane Ratni, Luke Green, Marla L. Weetall, Nikolai A. Naryshkin
-
Patent number: 11786486Abstract: Disclosed herein is pharmaceutical compositions of Compound 1, and/or the hydroquinone form thereof, and methods useful for treating or suppressing a disease or disorder such as an ?-synucleinpathy, a tauopathy, an autistic spectrum disorder, a pervasive developmental disorder, a liver disease, and liver damage in a subject using such pharmaceutical compositions.Type: GrantFiled: July 8, 2022Date of Patent: October 17, 2023Assignee: PTC THERAPEUTICS, INC.Inventors: Swathi Pinnamaneni, Mandar V. Dali, Dhaval Patel, Akm Nasir Uddin
-
Patent number: 11780839Abstract: 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 monocyclic heteroaryl compounds of Formula (I) or Formula (II), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.Type: GrantFiled: March 27, 2019Date of Patent: October 10, 2023Assignee: PTC THERAPEUTICS, INC.Inventors: Nadiya Sydorenko, Md Rauful Alam, Lukiana Amedzo, Suresh Babu, Ramil Y. Baiazitov, Scott J. Barraza, Anuradha Bhattacharyya, Gary Mitchell Karp, Nathaniel T. Kenton, Anthony R. Mazzotti, Young-Choon Moon, Nicholas W. Mszar, Jana Narasimhan, Sudipta Pal, Jigar S. Patel, Anthony Turpoff, Matthew G. Woll, Zhenrong Xu, Nanjing Zhang
-
Publication number: 20230310651Abstract: 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.Type: ApplicationFiled: February 3, 2023Publication date: October 5, 2023Applicant: PTC Therapeutics, Inc.Inventors: Nikolai Naryshkin, Amal Dakka
-
Patent number: 11753407Abstract: Provided herein are compounds of Formula (I): and forms thereof, wherein w1, w2, w3, w4, w5, w6 and w7 are as defined herein, including compositions thereof and uses therewith for treating spinal muscular atrophy.Type: GrantFiled: October 5, 2020Date of Patent: September 12, 2023Assignees: PTC Therapeutics, Inc., F. Hoffmann-La Roche AGInventors: Hongyan Qi, Soongyu Choi, Amal Dakka, Gary Mitchell Karp, Jana Narasimhan, Nikolai Naryshkin, Anthony A. Yurpoff, Marla L. Weetall, Ellen Welch, Matthew G. Woll, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang, Xin Zhao, Luke Green, Emmanuel Pinard, Hasane Ratni
-
Patent number: 11746077Abstract: 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: GrantFiled: January 25, 2022Date of Patent: September 5, 2023Assignee: PTC Therapeutics, Inc.Inventors: Andrew W. Hinman, Charles R. Holst, Angela Minnella, Paul Mollard, Sean Pintchovski, Jeffrey K. Trimmer, Eric Torrey
-
Publication number: 20230272367Abstract: 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: ApplicationFiled: May 12, 2023Publication date: August 31, 2023Applicant: PTC THERAPEUTICS, INC.Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
-
Patent number: 11702646Abstract: 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: GrantFiled: November 27, 2017Date of Patent: July 18, 2023Assignee: PTC THERAPEUTICS, INC.Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
-
Patent number: 11685746Abstract: 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), Formula (II), Formula (III), or Formula (IV), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.Type: GrantFiled: June 25, 2019Date of Patent: June 27, 2023Assignee: PTC Therapeutics, Inc.Inventors: Guangming Chen, Anuradha Bhattacharyya, Yao Jiang, Gary Mitchell Karp, Jana Narasimhan, Anthony Turpoff, Nanjing Zhang
-
Patent number: 11680034Abstract: Disclosed herein are compounds and methods of using such compounds for treating or suppressing oxidative stress disorders, including mitochondrial disorders, impaired energy processing disorders, neurodegenerative diseases and diseases of aging, or for modulating one or more energy biomarkers, normalizing one or more energy biomarkers, or enhancing one or more energy biomarkers, wherein the compounds are tocopherol quinone derivatives. Further disclosed are compounds, compositions, and methods for treatment of, or prophylaxis against, radiation exposure.Type: GrantFiled: April 12, 2021Date of Patent: June 20, 2023Assignee: PTC Therapeutics, Inc.Inventors: Andrew W. Hinman, Steven J. Richards
-
Publication number: 20230172915Abstract: One aspect described herein includes a method for treating coronavirus 2019 disease (COVID-19) caused by severe respiratory syndrome coronavirus 2 (SARS-CoV-2) by administering an effective amount of a DHODH inhibitor. Another aspect described herein includes a method of treating COVD-19 by administering 4-chlorophenyl (S)-6-chloro-1-(4-methoxyphenyl)-1,3,4,9-tetrahydro-2H-pyrido[3,4-b]indole-2-carboxylate (PTC299), or a pharmaceutically acceptable salt thereof.Type: ApplicationFiled: May 7, 2021Publication date: June 8, 2023Applicant: PTC THERAPEUTICS, INC.Inventors: Nikolai A. NARYSHKIN, Jason GRACI, John BAIRD, Joseph COLACINO, Marla L. WEETALL, Liangxian CAO
-
Patent number: 11667596Abstract: 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: GrantFiled: November 10, 2021Date of Patent: June 6, 2023Assignee: PTC THERAPEUTICS, INC.Inventors: Andrew W. Hinman, Charles R. Holst, Angela Minnella, Paul Mollard, Sean Pintchovski, Jeffrey K. Trimmer, Eric Torrey
-
Patent number: 11638706Abstract: 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 monocyclic 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: GrantFiled: November 9, 2020Date of Patent: May 2, 2023Assignee: PTC Therapeutics, Inc.Inventors: Suresh Babu, Anuradha Bhattacharyya, Seongwoo Hwang, Minakshi Jani, Young-choon Moon, Nadiya Sydorenko