125 patents
Page 5 of 7
Utility
Methods for Modulating Rna Splicing
11 Mar 21
In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein.
Nikolai Naryshkin, Amal Dakka
Filed: 13 Apr 20
Utility
2-SUBSTITUTED Amino-naphth (1,2-D) IMIDAZOL-5-ONE Compounds of Pharmaceutically Acceptable Salts Thereof
4 Mar 21
Hiroyuki KITANO, Kazuto Mori
Filed: 10 Aug 20
Utility
Methods for Dosing an Orally Active 1,2,4-OXADIAZOLE for Nonsense Mutation Suppression Therapy
11 Feb 21
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.
Samit HIRAWAT, Langdon MILLER
Filed: 23 Oct 20
Utility
Compounds for Treating Huntington's Disease
28 Jan 21
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
Filed: 27 Mar 19
Utility
Polymorphic and Amorphous Forms of (R)-2-HYDROXY-2-METHYL-4-(2,4,5-TRIMETHYL-3,6-DIOXOCYCLOHEXA-1,4-DIENYL)BUTANAMIDE
21 Jan 21
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.
Paul MOLLARD, Peter GIANNOUSIS, Shazad SUCHIT, Mahmoud MIRMEHRABI
Filed: 1 Jul 20
Utility
Compounds for Treating Huntington's Disease
14 Jan 21
Nadiya Sydorenko, Suresh Babu, Anuradha Bhattacharyya, Young-Choon Moon, Jana Narasimhan, Jigar S. Patel
Filed: 26 Mar 19
Utility
Methods for treating Huntington's disease
4 Jan 21
Suresh Babu, Anuradha Bhattacharyya, Seongwoo Hwang, Minakshi Jani, Young-choon Moon, Nadiya Sydorenko
Filed: 10 Dec 19
Utility
Compounds for treating spinal muscular atrophy
4 Jan 21
Hasane Ratni, Luke Green, Marla L. Weetall, Nikolai A. Naryshkin
Filed: 12 Apr 18
Utility
Methods for treating Huntington's disease
28 Dec 20
Suresh Babu, Anuradha Bhattacharyya, Seongwoo Hwang, Minakshi Jani, Young-choon Moon, Nadiya Sydorenko
Filed: 10 Dec 16
Utility
Methods for Enriching Alpha-tocotrienol from Mixed Tocol Compositions
2 Dec 20
Provided herein are methods for making an alpha-tocotrienol enriched tocol mixture, such as from a plant or plant-derived material.
Peter GIANNOUSIS, Paul MOLLARD, Noah M. BENJAMIN, Jeffrey D. BUTLER, Olivier DAPREMONT, James B. FALABELLA
Filed: 16 Aug 20
Utility
Compounds for treating spinal muscular atrophy
30 Nov 20
Hongyan Qi, Soongyu Choi, Amal Dakka, Gary Mitchell Karp, Jana Narasimhan, Nikolai Naryshkin, Anthony A. Turpoff, Marla L. Weetall, Ellen Welch, Matthew G. Woll, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang, Xin Zhao, Luke Green, Emmanuel Pinard, Hasane Ratni
Filed: 14 Aug 19
Utility
Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
30 Nov 20
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.
Samit Hirawat, Langdon Miller
Filed: 3 Dec 18
Utility
Methods for Modifying Rna Splicing
25 Nov 20
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.
Anuradha BHATTACHARYYA, Amal DAKKA, Kerstin EFFENBERGER, Vijayalakshmi GABBETA, Minakshi B. JANI, Wencheng LI, Nikolai NARYSHKIN, Christopher TROTTA, Kari WIEDINGER
Filed: 12 Jun 18
Utility
Treatment of Leigh Syndrome and Leigh-like Syndrome with Tocotrienol Quinones
18 Nov 20
The present invention relates to methods of treating Leigh Syndrome and Leigh-like Syndrome with tocotrienol quinones, including alpha-tocotrienol quinone, in order to alleviate symptoms of the disease.
Orion D. JANKOWSKI, Kieron E. WESSON, Katsuyuki MURASE
Filed: 3 Aug 20
Utility
Crystalline Forms of 3-[5-(2-FLUOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]-BENZOIC Acid for the Treatment of Disease
21 Oct 20
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.
Neil G. Almstead, Peter Seongwoo Hwang, Young-Choon Moon
Filed: 6 Jul 20
Utility
Fluoroalkyl, Fluoroalkoxy, Phenoxy, Heteroaryloxy, Alkoxy, and Amine 1,4-BENZOQUINONE Derivatives for Treatment of Oxidative Stress Disorders
21 Oct 20
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.
Andrew W. HINMAN, Steven J. RICHARDS
Filed: 5 Jul 20
Utility
Method of Inhibiting or Reducing a Coronaviridae Virus Infection
7 Oct 20
Compounds that inhibit viral replication or the production of viral RNA or DNA or viral protein and compositions comprising such Compounds are described.
Jason Graci, Zhengxian Gu
Filed: 22 Jun 20
Utility
Polymorphic and amorphous forms of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide
24 Aug 20
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.
Paul Mollard, Peter Giannousis, Shazad Suchit, Mahmoud Mirmehrabi
Filed: 13 Mar 19
Utility
Compositions for an Orally Active 1,2,4-OXADIAZOLE for the Treatment of Disease
19 Aug 20
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.
Samit Hirawat, Langdon Miller
Filed: 4 May 20
Utility
Hydrogen Isotope-enriched Analogues of 1,2,4-OXADIAZOLE Benzoic Acid Compounds, Compositions and Uses Thereof
19 Aug 20
Novel hydrogen isotope-enriched 1,2,4-oxadiazole benzoic acid compounds, methods of their use and pharmaceutical compositions thereof are disclosed.
Seongwoo HWANG, Ellen WELCH
Filed: 14 Nov 16