Patents by Inventor Frank Rigo
Frank Rigo has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).
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Publication number: 20240401039Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.Type: ApplicationFiled: January 23, 2024Publication date: December 5, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Susan M. Freier, Priyam Singh, Frank Rigo, Paymaan Jafar-nejad, Holly Kordasiewicz
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Publication number: 20240360449Abstract: Provided are oligomeric compounds, methods, and pharmaceutical compositions for modulating expression of SCN1A RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a developmental or epileptic encephalopathic disease, such as, for example, Dravet Syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral dysfunctions, movement and balance dysfunctions, orthopedic conditions, motor dysfunctions, cognitive impairment, delayed language and speech, visual motor integration dysfunctions, visual perception dysfunctions, executive dysfunctions, and dysautonomia.Type: ApplicationFiled: August 26, 2022Publication date: October 31, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Paymaan Jafar-nejad, Frank Rigo
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Publication number: 20240335556Abstract: Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.Type: ApplicationFiled: October 13, 2023Publication date: October 10, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Frank Rigo, Chrissa A. Dwyer
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Publication number: 20240336915Abstract: Provided are compounds, pharmaceutical compositions, and methods of use for reducing the amount or activity of DUX4 RNA in a cell or animal, and in certain instances reducing the amount of DUX4 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a muscular dystrophy. Such symptoms and hallmarks include muscle weakness and/or muscle wasting in facio, scapula, and/or humeral muscle that can progress to the muscles of the trunk and/or lower limbs. Such muscular dystrophies include Facioscapulohumeral muscular dystrophy (FSHD).Type: ApplicationFiled: January 21, 2022Publication date: October 10, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Ruben E. Valas, Paymaan Jafar-nejad, Frank Rigo, Susan M. Freier, Huynh-Hoa Bui, Priyam Singh
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Publication number: 20240285669Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include motor delays, cognitive delays, paroxysmal deterioration, seizures, vomiting, swallowing difficulties, ataxic gait, palatal myoclonus, autonomic dysfunction, and presence of intra-astrocytic inclusions called Rosenthal fibers. Such leukodystrophies include Alexander Disease.Type: ApplicationFiled: September 7, 2023Publication date: August 29, 2024Inventors: Berit Elissa Powers, Frank Rigo
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Publication number: 20240287513Abstract: Provided are compounds, methods, and pharmaceutical compositions for modulating splicing of a pre-mRNA in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a disease or disorder.Type: ApplicationFiled: August 1, 2023Publication date: August 29, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Frank Rigo, Paymaan Jafar-Nejad
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Publication number: 20240279664Abstract: The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain embodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.Type: ApplicationFiled: June 27, 2023Publication date: August 22, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Michael Oestergaard, Punit P. Seth, Frank Rigo, Chrissa A. Dwyer
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Publication number: 20240200067Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C9ORF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).Type: ApplicationFiled: June 6, 2023Publication date: June 20, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: C. Frank Bennett, Susan M. Freier, Frank Rigo, Eric E. Swayze
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Patent number: 12013403Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.Type: GrantFiled: July 1, 2021Date of Patent: June 18, 2024Assignee: BIOGEN MA INC.Inventors: Frank Rigo, Katherine M. Bishop
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Publication number: 20240175021Abstract: Provided are methods for increasing the amount or activity of FMR1 RNA, and in certain instances of increasing the amount of FMRP protein, in an animal. Such methods are useful to prevent or ameliorate at least one symptom of a Fragile X-Spectrum disorder. Such Fragile X-Spectrum disorders include FXS, FXTAS, and FXPOI.Type: ApplicationFiled: May 9, 2023Publication date: May 30, 2024Inventors: Frank RIGO, Peter TODD, Caitlin RODRIGUEZ
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Publication number: 20240102012Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SCN2A RNA in a cell or subject, and in certain instances reducing the amount of SCN2A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a disease or disorder associated with a voltage-gated sodium channel protein, such as, for example, a Developmental and Epileptic Encephalopathy, an intellectual disability, or an autism spectrum disorder. Such symptoms and hallmarks include, but are not limited to seizures, hypotonia, sensory integration disorders, motor development delays and dysfunctions, intellectual and cognitive dysfunctions, movement and balance dysfunctions, visual dysfunctions, delayed language and speech, gastrointestinal disorders, neurodevelopmental delays, sleep problems, and sudden unexpected death in epilepsy.Type: ApplicationFiled: October 10, 2023Publication date: March 28, 2024Inventors: Paymaan Jafar-Nejad, Huynh-Hoa Bui, Susan M. Freier, Frank Rigo
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Patent number: 11926825Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.Type: GrantFiled: April 23, 2021Date of Patent: March 12, 2024Assignee: Ionis Pharmaceuticals, Inc.Inventors: Susan M. Freier, Frank Rigo
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Publication number: 20240026353Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SCN2A RNA in a cell or subject, and in certain instances reducing the amount of SCN2A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a disease or disorder associated with a voltage-gated sodium channel protein, such as, for example, a Developmental and Epileptic Encephalopathy, an intellectual disability, or an autism spectrum disorder. Such symptoms and hallmarks include, but are not limited to seizures, hypotonia, sensory integration disorders, motor development delays and dysfunctions, intellectual and cognitive dysfunctions, movement and balance dysfunctions, visual dysfunctions, delayed language and speech, gastrointestinal disorders, neurodevelopmental delays, sleep problems, and sudden unexpected death in epilepsy.Type: ApplicationFiled: August 6, 2021Publication date: January 25, 2024Inventors: Paymaan Jafar-Nejad, Huynh-Hoa Bui, Susan M. Freier, Frank Rigo
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Patent number: 11833221Abstract: Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.Type: GrantFiled: August 31, 2022Date of Patent: December 5, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventors: Frank Rigo, Chrissa A. Dwyer
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Publication number: 20230338555Abstract: Disclosed herein are compounds that comprise an oligonucleotide, a conjugate linker, and a conjugate moiety capable of interacting with a cell surface moiety, wherein the oligonucleotide and the conjugate moiety are connected via the conjugate linker. The conjugate moiety may comprise a cell-targeting moiety and a peptide extender. In general, peptide extenders have sufficient length and/or structure to reduce or prevent interaction between the oligonucleotide and the cell-targeting moiety. Such compounds are useful to treat, prevent or ameliorate a condition or disease in an individual with limited off-target effects.Type: ApplicationFiled: May 18, 2021Publication date: October 26, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventors: Thazha P. Prakash, Mehran Nikan, Michael Tanowitz, Chrissa A. Dwyer, Frank Rigo, Punit P. Seth
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Patent number: 11786546Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include motor delays, cognitive delays, paroxysmal deterioration, seizures, vomiting, swallowing difficulties, ataxic gait, palatal myoclonus, autonomic dysfunction, and presence of intra-astrocytic inclusions called Rosenthal fibers. Such leukodystrophies include Alexander Disease.Type: GrantFiled: July 24, 2020Date of Patent: October 17, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventors: Berit Elissa Powers, Frank Rigo
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Patent number: 11781135Abstract: Disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Also disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.Type: GrantFiled: March 11, 2019Date of Patent: October 10, 2023Assignees: Washington University, Biogen MA Inc.Inventors: Timothy M. Miller, Sarah Devos, C. Frank Bennett, Frank Rigo
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Patent number: 11732260Abstract: Certain embodiments disclosed herein are directed to compounds and methods for modulating APP expression. In certain embodiments, modulating the splicing of amyloid precursor protein (APP) reduces amyloid ? (A?) production.Type: GrantFiled: March 1, 2019Date of Patent: August 22, 2023Assignees: Ionis Pharmaceuticals, Inc., Rosalind Franklin University of Medicine and ScienceInventors: Frank Rigo, Michelle L. Hastings
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Patent number: 11725208Abstract: The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain embodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.Type: GrantFiled: December 14, 2018Date of Patent: August 15, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventors: Michael Oestergaard, Punit P. Seth, Frank Rigo, Chrissa A. Dwyer
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Publication number: 20230201374Abstract: Provided herein are methods, compounds, and compositions useful for targeted delivery of compounds to non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions are useful, for example, in gene therapy mediated ectopic expression of cell surface receptors and targeted delivery of compounds, such as conjugated oligonucleotides, to the non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions can be useful, for example, to treat, prevent, delay or ameliorate disease in an individual by targeted reduction of a gene of interest in the non-native cell ectopically expressing cell surface receptors.Type: ApplicationFiled: July 13, 2022Publication date: June 29, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventor: Frank Rigo