48 patents
Utility
Use of (S)-3-AMINO-4-(DIFLUOROMETHYLENYL)CYCLOPENT-1-ENE-1-CARBOXYLIC Acid In the Treatment of Cancer
11 Jan 24
Methods and compositions including (S)-3-amino-4-(difluoromethylenyl)cyclopent-1-ene-1-carboxylic acid or a pharmaceutically acceptable salt thereof are provided for use in treating cancer and inhibiting cancer proliferation and metastasis.
Aaron Goldman
Filed: 6 Jul 23
Utility
Use of (1S,3S)-3-AMINO-4-(DIFLUOROMETHYLIDENE) CYCLOPENTANE-1-CARBOXYLIC Acid and (S)-3-AMINO-4-(DIFLUOROMETHYLENYL)CYCLOPENT-1-ENE-1-CARBOXYLIC Acid In the Treatment of Tinnitus, Acute Sensorineural Hearing Loss, Meniere’s Disease, Tourette’s Syndrome, Attention Deficit Hyperactivity Disorder and Addiction
21 Dec 23
Methods of treating tinnitus, acute sensorineural hearing loss, Meniere's disease, Tourette's syndrome, ADHD or addiction with (1S,3S)-3-amino-4-(difluoromethylidene) cyclopentane-1-carboxylic acid or a pharmaceutically acceptable salt thereof are provided.
Matthew During
Filed: 25 Aug 23
Utility
shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
19 Oct 23
Provided herein are compositions and methods for activating expression from the paternally-inherited allele of UBE3A in Angelman syndrome using viral vector delivery of short hairpin RNAs.
Stormy CHAMBERLAIN, Noelle Germain, Peter Perrino
Filed: 7 Mar 23
Utility
shRNA TARGETING UBE3A-ATS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
19 Oct 23
Provided herein are compositions and methods for activating expression from the paternally-inherited allele of UBE3A in Angelman syndrome using viral vector delivery of short hairpin RNAs.
Stormy CHAMBERLAIN, Noelle Germain, Peter Perrino
Filed: 7 Mar 23
Utility
Methods and Compositions for Treatment of Epileptic Disorders
21 Sep 23
Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.
Matthew During
Filed: 6 Apr 23
Utility
Methods and Compositions for Treatment of Epileptic Disorders
21 Sep 23
Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.
Matthew During
Filed: 30 May 23
Utility
Use of Gaboxadol for the Treatment of NON-24 Hour Sleep-wake Disorder
14 Sep 23
Treatment of non 24 sleep wake disorder using gaboxadol or a pharmaceutically acceptable salt thereof is provided.
Matthew During
Filed: 19 May 23
Utility
Use of (S)-3-AMINO-4-(DIFLUOROMETHYLENYL) CYCLOPENT-1-ENE-1-CARBOXYLIC Acid and Related Compounds, (1S,3S)-3-AMINO-4-(DIFLUOROMETHYLIDENE) CYCLOPENTANE-1-CARBOXYLIC Acid and Vigabatrin In the Treatment of Developmental Disorders
17 Aug 23
Methods and compositions for use treating Fragile X syndrome, Angelman syndrome, Fragile X-associated tremor/ataxia syndrome, Autism Spectrum Disorder, Asperger's syndrome, Pervasive developmental disorder not otherwise characterized, Childhood Disintegrative Disorder, Williams syndrome, or Jacobsen syndrome with a compound of Formula 1 as described herein, (S)-3-amino-4-(difluoromethylenyl) cyclopent-1-ene-1-carboxylic acid, (1S,3S)-3-amino-4-(difluoromethylidene) cyclopentane-1-carboxylic acid, or vagabatrin, or pharmaceutically acceptable salts of any of the foregoing, are provided.
Matthew During, Brett Abrahams
Filed: 19 Apr 23
Utility
Methods of Treating Seizure Disorders and Prader-willi Syndrome
18 May 23
Methods of treating a seizure disorder with (1S,3S)-3-amino (difluoromethylidene)cyclopentane-1-carboxylic acid, or (S)-3-amino (difluoromethylenyl)cyclopent-1-ene-1-carboxylic acid (KT-II-115), or a pharmaceutically acceptable salt of any of the preceding, are provided.
Matthew DURING
Filed: 17 May 21
Utility
Methods of Treating Developmental Disorders with Gaboxadol
9 Mar 23
Methods of treating developmental disorders such as Angelman syndrome, Fragile X syndrome, Fragile X-associated tremor/ataxia syndrome (FXTAS), Autistic Spectrum Disorder, Autism, Asperger's syndrome, pervasive developmental disorder, Childhood Disintegrative Disorder, Rett syndrome, Lanau-Kleffner Syndrome, Prader-Willi Syndrome, Tardive Dyskinesia, and/or Williams Syndrome with gaboxadol or a pharmaceutically acceptable salt thereof are provided.
Matthew DURING
Filed: 4 Nov 22
Utility
Methods of Increasing Tonic Inhibition and Treating Secondary Insomnia
16 Feb 23
Methods of increasing tonic inhibition in a subject in need thereof, for example a subject with Fragile X syndrome or Angelman syndrome are disclosed.
Matthew During
Filed: 8 Aug 22
Utility
Methods and Compositions for Treatment of Epileptic Disorders
17 Nov 22
Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.
Matthew During
Filed: 13 Jul 22
Utility
Methods and Compositions for Treatment of Epileptic Disorders
13 Oct 22
Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.
Matthew During
Filed: 17 Jun 22
Utility
Use of Gaboxadol In the Treatment of Substance Use Disorders
4 Aug 22
Methods of using gaboxadol or a pharmaceutically acceptable salt thereof and compositions of gaboxadol or a pharmaceutically acceptable salt thereof for use in treating a substance use disorder, preventing a substance use craving, diminishing a substance use craving, and/or facilitating substance use cessation are provided.
Matthew During
Filed: 19 Apr 22
Utility
Use of Gaboxadol, Ganaxolone and Allopregnanolone to Treat Movement Disorders
14 Apr 22
Use of gaboxadol, allopregnanolone, or ganaxolone, or a pharmaceutical salt of any of the foregoing, for the treatment of cervical dystonia, multiple system atrophy, myoclonus, progressive supranuclear palsy, restless legs syndrome, Wilson's disease, ataxia, chorea, or dystonia in a subject in need thereof.
Matthew DURING
Filed: 21 Dec 21
Utility
Pharmaceutical Formulations Containing Gaboxadol for Therapeutic Treatment
10 Feb 22
Pharmaceutical formulations containing gaboxadol or a pharmaceutically acceptable salt thereof and methods of treating essential tremors, Tourette syndrome or Fragile X syndrome are provided.
Matthew DURING
Filed: 21 Oct 21
Utility
Methods of Increasing Tonic Inhibition and Treating Secondary Insomnia
20 Jan 22
Methods of increasing tonic inhibition in a subject in need thereof, for example a subject with Fragile X syndrome or Angelman syndrome are disclosed.
Matthew During
Filed: 30 Sep 21
Utility
Treatment of Depressive Disorders
9 Dec 21
Formulations for treating depressive disorders and methods for treating depressive disorders using gaboxadol or a pharmaceutically acceptable salt thereof are provided.
Matthew DURING
Filed: 18 Aug 21
Utility
Recombinant Adeno-associated Vectors
2 Dec 21
Adeno-associated virus (AAV) vectors and uses thereof are provided.
Matthew DURING
Filed: 12 Aug 21
Utility
Methods of Treating Attention Deficit Hyperactivity Disorder
16 Sep 21
Methods and compositions for treating Attention-deficit/hyperactivity disorder are provided which include administering to a patient in need thereof captodiamine or a pharmaceutically acceptable salt thereof.
Matthew During
Filed: 2 Jun 21