公开(公告)号：US11667596B2

公开(公告)日：2023-06-06

申请号：US17523880

申请日：2021-11-10

Applicant: PTC THERAPEUTICS, INC.

Inventor： Andrew W. Hinman ,  Charles R. Holst ,  Angela Minnella ,  Paul Mollard ,  Sean Pintchovski ,  Jeffrey K. Trimmer ,  Eric Torrey

IPC: C07C50/04 ,  A61P25/16 ,  C07C46/10

CPC classification number: C07C50/04 ,  A61P25/16 ,  C07C46/10 ,  C07B2200/13

Abstract: Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of α-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.

公开(公告)号：US11186559B2

公开(公告)日：2021-11-30

申请号：US16995754

申请日：2020-08-17

Applicant: PTC Therapeutics, Inc.

Inventor： Peter Giannousis ,  Paul Mollard ,  Noah M. Benjamin ,  Jeffrey D. Butler ,  Olivier Dapremont ,  James B. Falabella

IPC: C07D311/72

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.

公开(公告)号：US10745371B2

公开(公告)日：2020-08-18

申请号：US16063035

申请日：2016-12-16

Applicant: PTC Therapeutics, Inc.

Inventor： Peter Giannousis ,  Paul Mollard ,  Noah M. Benjamin ,  Jeffrey D. Butler ,  Olivier Dapremont ,  James B. Falabella

IPC: C07D311/72

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.

公开(公告)号：US11304914B2

公开(公告)日：2022-04-19

申请号：US16919044

申请日：2020-07-01

Applicant: PTC Therapeutics, Inc.

Inventor： Paul Mollard ,  Peter Giannousis ,  Shazad Suchit ,  Mahmoud Mirmehrabi

IPC: C07C235/78 ,  C07C215/28 ,  C07C215/30 ,  C07C231/12 ,  C07C215/08 ,  A61K31/122 ,  C07B57/00 ,  A61K31/164

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.

公开(公告)号：US20220106248A1

公开(公告)日：2022-04-07

申请号：US17523880

申请日：2021-11-10

Applicant: PTC THERAPEUTICS, INC.

Inventor： Andrew W. Hinman ,  Charles R. Holst ,  Angela Minnella ,  Paul Mollard ,  Sean Pintchovski ,  Jeffrey K. Trimmer ,  Eric Torrey

IPC: C07C50/04 ,  A61P25/16 ,  C07C46/10

Abstract: Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of α-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.

公开(公告)号：US20210276937A1

公开(公告)日：2021-09-09

申请号：US17218042

申请日：2021-03-30

Applicant: PTC THERAPEUTICS, INC.

Inventor： Andrew W. Hinman ,  Charles R. Holst ,  Angela Minella ,  Paul Mollard ,  Sean Pintchovski ,  Jeffrey K. Trimmer ,  Eric Torrey

IPC: C07C50/04 ,  C07C46/10 ,  A61P25/16

Abstract: Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of α-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.

公开(公告)号：US11938101B2

公开(公告)日：2024-03-26

申请号：US17692133

申请日：2022-03-10

Applicant: PTC Therapeutics, Inc.

Inventor： Paul Mollard ,  Peter Giannousis ,  Shazad Suchit ,  Mahmoud Mirmehrabi ,  Christopher R. Cornell ,  Kieron E. Wesson

IPC: C07C235/78 ,  A61K31/122 ,  A61K31/164 ,  C07B57/00 ,  C07C215/08 ,  C07C215/28 ,  C07C215/30 ,  C07C231/12 ,  C07D215/28

CPC classification number: A61K31/122 ,  A61K31/164 ,  C07B57/00 ,  C07C215/08 ,  C07C215/28 ,  C07C215/30 ,  C07C231/12 ,  C07C235/78 ,  C07B2200/13 ,  C07C2601/16 ,  C07C231/12 ,  C07C235/78

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.

公开(公告)号：US20230339835A1

公开(公告)日：2023-10-26

申请号：US18347068

申请日：2023-07-05

Applicant: PTC THERAPEUTICS, INC.

Inventor： Andrew W. Hinman ,  Charles R. Holst ,  Angela Minnella ,  Paul Mollard ,  Sean Pintchovski ,  Jeffrey K. Trimmer ,  Eric Torrey

IPC: C07C50/04 ,  A61P25/16 ,  C07C46/10

CPC classification number: C07C50/04 ,  A61P25/16 ,  C07C46/10 ,  C07B2200/13

Abstract: Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of α-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.

公开(公告)号：US11560364B2

公开(公告)日：2023-01-24

申请号：US17511432

申请日：2021-10-26

Applicant: PTC Therapeutics, Inc.

Inventor： Peter Giannousis ,  Paul Mollard ,  Noah M. Benjamin ,  Jeffrey D. Butler ,  Olivier Dapremont ,  James B. Falabella

IPC: C07D311/72

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.

公开(公告)号：US10968166B2

公开(公告)日：2021-04-06

申请号：US16228657

申请日：2018-12-20

Applicant: PTC Therapeutics, Inc.

Inventor： Orion D. Jankowski ,  Kieron E. Wesson ,  Paul Mollard ,  William D. Shrader

IPC: C07C235/80 ,  C07C235/32 ,  C07C235/78 ,  C07C317/28 ,  C07D207/27 ,  C07D211/46 ,  C07D213/40 ,  C07D233/61 ,  C07D295/13 ,  C07D295/192 ,  C07D265/30 ,  C07D311/66 ,  C07D207/26 ,  C07D213/50 ,  C07D295/116 ,  C07D295/185 ,  A61P21/00 ,  A61P25/00 ,  A61P25/16 ,  A61P27/00

Abstract: 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-quinoly)-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.