Patents Assigned to Ionis Pharmaceuticals, Inc.
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Patent number: 11833168Abstract: Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).Type: GrantFiled: June 14, 2019Date of Patent: December 5, 2023Assignees: Ionis Pharmaceuticals, Inc., Ludwig Institute For Cancer ResearchInventors: Huynh-Hoa Bui, Don W. Cleveland, Ze'ev Melamed
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Publication number: 20230374519Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PMP22 RNA in a cell or animal, and in certain instances reducing the amount of PMP22 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 demyelination, progressive axonal damage and/or loss, weakness and wasting of foot and lower leg muscles, foot deformities, and weakness and atrophy in the hands. Such neurodegenerative diseases include Charcot-Marie-Tooth disease.Type: ApplicationFiled: June 18, 2021Publication date: November 23, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventors: Eric E. Swayze, Holly Kordasiewicz, Punit P. Seth, Hien Thuy Zhao, Michael T. Migawa, Ruben E. Valas, Thazha P. Prakash
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Publication number: 20230357779Abstract: Provided are oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of IFNAR1 RNA in a cell or animal, and in certain instances reducing the amount of IFNAR1 protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat diseases and conditions associated with neuroinflammation, including Aicardi-Goutières Syndrome, stroke, neuropsychiatric systemic lupus erythematosus, neuroinflammation following traumatic brain injury, neuro-autoimmune disorders, Alzheimer's disease, post-operative delirium and cognitive decline, cranial radiation-induced cognitive decline, viral infection-induced cognitive decline, neuromyelitis optica, and ataxia telangiectasia.Type: ApplicationFiled: July 26, 2023Publication date: November 9, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventor: Fredrik Carl Kamme
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Publication number: 20230357770Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of APOE RNA in a cell or animal, and in certain instances reducing the amount of APOE 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 cognitive impairment, progressive memory loss, behavioral abnormality, dementia, difficulty performing daily activities, amyloid plaques, neurofibrillary tangles, and neuroinflammation.Type: ApplicationFiled: September 23, 2021Publication date: November 9, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventors: Tracy A. Cole, Swagatam Mukhopadhyay, Huynh-Hoa Bui, Priyam Singh, Holly Kordasiewicz, Susan M. Freier, Hien Thuy Zhao
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Publication number: 20230357776Abstract: The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.Type: ApplicationFiled: May 19, 2023Publication date: November 9, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Tamar R. Grossman, Michael L. McCaleb, Andrew T. Watt, Susan M. Freier
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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: 11781143Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting PNPLA3 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PNPLA3.Type: GrantFiled: July 22, 2020Date of Patent: October 10, 2023Assignee: IONIS PHARMACEUTICALS, INC.Inventors: Susan M. Freier, Huynh-Hoa Bui
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Publication number: 20230310483Abstract: Methods, compounds, and compositions useful for inhibiting HSD17B13 expression are provided. Such compounds, compositions, and methods are useful for treating, preventing, or ameliorating a disease associated with HSD 17B 13.Type: ApplicationFiled: June 14, 2023Publication date: October 5, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventors: Susan M. Freier, Susan F. Murray
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Patent number: 11761000Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's Disease (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.Type: GrantFiled: June 21, 2021Date of Patent: September 19, 2023Assignee: IONIS PHARMACEUTICALS, INC.Inventor: Susan M. Freier
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Patent number: 11753644Abstract: Provided are oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of IFNAR1 RNA in a cell or animal, and in certain instances reducing the amount of IFNAR1 protein in a cell or animal Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat diseases and conditions associated with neuroinflammation, including Aicardi-Goutières Syndrome, stroke, neuropsychiatric systemic lupus erythematosus, neuroinflammation following traumatic brain injury, neuro-autoimmune disorders, Alzheimer's disease, post-operative delirium and cognitive decline, cranial radiation-induced cognitive decline, viral infection-induced cognitive decline, neuromyelitis optica, and ataxia telangiectasia.Type: GrantFiled: June 17, 2022Date of Patent: September 12, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventor: Fredrik Carl Kamme
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Publication number: 20230279396Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.Type: ApplicationFiled: December 16, 2022Publication date: September 7, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventors: Susan M. Freier, Eric E. Swayze
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Publication number: 20230263822Abstract: Disclosed herein are antisense compounds and methods for decreasing Factor 11 and treating or preventing thromboembolic complications in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to Factor 11 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Antisense compounds targeting Factor 11 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.Type: ApplicationFiled: January 5, 2023Publication date: August 24, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventors: Susan M. Freier, Chenguang Zhao, Brett P. Monia, Hong Zhang, Jeffrey R. Crosby, Andrew M. Siwkowski
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Patent number: 11732265Abstract: The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.Type: GrantFiled: May 7, 2021Date of Patent: August 22, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Tamar R. Grossman, Michael L. McCaleb, Andrew T. Watt, Susan M. Freier
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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: 11732261Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.Type: GrantFiled: September 3, 2020Date of Patent: August 22, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventors: Punit P. Seth, Michael Oestergaard, Eric E. Swayze
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Patent number: 11732263Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PLP1 RNA in a cell or subject, and in certain instances reducing the amount of proteolipid protein 1 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 hypotonia, nystagmus, optic atrophy, respiratory distress, motor delays, cognitive dysfunction, speech dysfunction, spasticity, ataxia, seizures, choreiform movements, and death. Such leukodystrophies include Pelizaeus-Merzbacher disease.Type: GrantFiled: June 29, 2021Date of Patent: August 22, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventor: Berit Elissa Powers
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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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Patent number: 11713462Abstract: Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a glycogen storage disease or disorder in an individual in need.Type: GrantFiled: December 23, 2021Date of Patent: August 1, 2023Assignees: Ionis Pharmaceuticals, Inc., The Hospital for Sick ChildrenInventors: Tamar R. Grossman, Susan M. Freier, Berge Minassian, Saija Ahonen
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Publication number: 20230235323Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 RNA in a cell or animal, and in certain embodiments reducing the amount of ATXN3 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 motor dysfunction, aggregation formation, and neuron death. Such neurodegenerative diseases include spinocerebellar ataxia type 3 (SCA3).Type: ApplicationFiled: June 29, 2022Publication date: July 27, 2023Applicant: Ionis Pharmaceuticals, Inc.Inventor: Susan M. Freier