Patents Assigned to Ionis Pharmaceuticals, Inc.
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Publication number: 20240141343Abstract: 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: June 12, 2023Publication date: May 2, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Punit P. Seth, Michael Oestergaard, Eric E. Swayze
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Publication number: 20240132885Abstract: Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.Type: ApplicationFiled: December 2, 2022Publication date: April 25, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Brett P. Monia, Susan M. Freier, Andrew M. Siwkowski, Shuling Guo
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Publication number: 20240124513Abstract: Provided are oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of methyl CpG binding protein 2 (MECP2) RNA in a cell or animal, and in certain instances reducing the amount of MECP2 protein in a cell or animal Such oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodevelopmental disease or disorder. Such neurodevelopmental diseases or disorders include MECP2 duplication syndrome. Such symptoms or hallmarks include autism, intellectual disability, motor dysfunction, hypotonia, global developmental delays, gastrointestinal symptoms, anxiety, epilepsy, recurrent respiratory tract infections, epileptic encephalopathy, and early death.Type: ApplicationFiled: September 22, 2023Publication date: April 18, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventor: Susan M. Freier
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Patent number: 11959080Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting PMP22 expression and for treating, preventing, or ameliorating a disease associated with PMP22.Type: GrantFiled: October 4, 2021Date of Patent: April 16, 2024Assignee: Ionis Pharmaceuticals, Inc.Inventors: Gene Hung, Holly Kordasiewicz, Hien Thuy Zhao, Eric E. Swayze
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Publication number: 20240092822Abstract: The present disclosure provides a process of separating designated oligomeric compounds from sample solutions comprising at least one contaminant In certain embodiments, the designated oligomeric compounds comprise a modified oligonucleotide and a conjugate group comprising at least one carbohydrate cluster. In certain embodiments, the present disclosure provides HPLC conditions that increase the separation of a designated oligomeric compound from at least one contaminant compared to standard HPLC conditions.Type: ApplicationFiled: March 2, 2022Publication date: March 21, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Andrew A. Rodriguez, Christopher Michael Gabriel
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Publication number: 20240093201Abstract: Provided herein are compositions and methods for non-parenteral delivery of conjugated oligomeric compounds. In certain embodiments, compositions and methods are provided for oral delivery of conjugated oligomeric compounds. In certain embodiments, the oligomeric compounds are conjugated to one or more N-acetylgalactosamines or N-acetylgalactosamine analogues.Type: ApplicationFiled: December 2, 2022Publication date: March 21, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Stanley T. Crooke
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Publication number: 20240082291Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 mRNA in a cell or animal, and in certain instances reducing the amount of Ataxin-3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to prevent or 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 SCA3.Type: ApplicationFiled: February 13, 2023Publication date: March 14, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Susan M. Freier, Holly Kordasiewicz
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Patent number: 11926830Abstract: 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: September 2, 2021Date of Patent: March 12, 2024Assignee: Ionis Pharmaceuticals, Inc.Inventors: Tamar R. Grossman, Michael L McCaleb, Andrew T. Watt, Susan M. Freier
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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: 20240067962Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN1 RNA in a cell or subject, and in certain instances reducing the amount of ATXN1 in a cell or subject. 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 gait and limb ataxia, cognitive impairments, difficulty with speaking and swallowing, atrophy of the cerebellum and brainstem in magnetic resonance imaging (MRI), neurochemical abnormalities in the cerebellum and brainstem detected via magnetic resonance spectroscopy (MRS), and death within 10-15 years of symptom onset. Such neurodegenerative diseases include Spinocerebellar ataxia type 1.Type: ApplicationFiled: October 19, 2022Publication date: February 29, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventor: Susan M. Freier
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Publication number: 20240067964Abstract: Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) or Lp(a). Certain diseases, disorders or conditions related to apo(a) or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The antisense compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.Type: ApplicationFiled: February 28, 2023Publication date: February 29, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Rosanne M. Crooke, Mark J. Graham, Susan M. Freier, Marc Lim, Andrew Dibble
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Publication number: 20240058370Abstract: Provided herein are gapmer oligomeric compounds for reduction of target RNA in vivo comprising different nucleotide modifications within one or both wing regions. Also provided are methods of using such oligomeric compounds, including use in animals. In certain embodiments, such compound have desirable potency and toxicity characteristics.Type: ApplicationFiled: January 5, 2023Publication date: February 22, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventor: Eric E. Swayze
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Publication number: 20240026364Abstract: Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as polycythemia, hemochromatosis or ?-thalassemia can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to TMPRSS6.Type: ApplicationFiled: July 25, 2023Publication date: January 25, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Shuling Guo, Mariam Aghajan, Eric E. Swayze
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Patent number: 11873495Abstract: Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 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 Parkinson's disease.Type: GrantFiled: April 4, 2022Date of Patent: January 16, 2024Assignee: Ionis Pharmaceuticals, Inc.Inventors: Tracy A. Cole, Susan M. Freier
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Publication number: 20240002852Abstract: Provided are compounds and pharmaceutical compositions for reducing the amount or activity of Charged Multivesicular Body Protein 7 (CHMP7) RNA in a cell or subject, and in certain instances reducing the amount of CHMP7 protein in a cell or subject. Such compounds and pharmaceutical compositions are useful to ameliorate diseases or conditions associated with aberrant activation of Endosomal Sorting Complexes Required for Transport-III proteins.Type: ApplicationFiled: October 1, 2021Publication date: January 4, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventor: Huynh-Hoa Bui
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Publication number: 20240002851Abstract: The present disclosure provides oligomeric compounds (including oligomeric compounds that are antisense agents or portions thereof) comprising a modified oligonucleotide having at least one modified internucleoside linking group.Type: ApplicationFiled: January 25, 2023Publication date: January 4, 2024Applicant: Ionis Pharmaceuticals, Inc.Inventors: Brooke A. Anderson, Xue-Hai Liang, William John Drury, III, Michael Oestergaard, Michael T. Migawa, Punit P. Seth
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Patent number: 11859180Abstract: The present disclosure provides compositions comprising an antisense oligonucleotide and one or more excipients that modulates viscosity, turbidity or both viscosity and turbidity. In certain embodiments, compositions comprising an antisense oligonucleotide and one or more excipients having low viscosity are provided. In certain embodiments, compositions comprising an antisense oligonucleotide and one or more excipients having low turbidity are provided. In certain embodiments, pharmaceutical compositions comprising an antisense oligonucleotide and one or more excipients having low viscosity and turbidity are provided.Type: GrantFiled: June 9, 2020Date of Patent: January 2, 2024Assignee: Ionis Pharmaceuticals, Inc.Inventors: Andrew Dibble, Lloyd Tillman, Bret Coldren, Marc Lim
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Patent number: 11851655Abstract: Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.Type: GrantFiled: October 1, 2020Date of Patent: December 26, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventors: Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Mark J. Graham
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Patent number: 11840689Abstract: The present disclosure provides methods of treating or preventing fatty liver disease and/or lowering cholesterol and LDL cholesterol levels in a subject. The present disclosure further provides methods of lowering expression of Pleckstrin and Sec7 Domain Containing 3 (PSD3) in a subject.Type: GrantFiled: September 21, 2021Date of Patent: December 12, 2023Assignees: ASTRAZENECA AB, IONIS PHARMACEUTICALS, INC.Inventors: Daniel Linden, Richard Lee, Stefano Romeo, Huynh-Hoa Bui
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Patent number: 11840686Abstract: Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.Type: GrantFiled: July 2, 2021Date of Patent: December 12, 2023Assignee: Ionis Pharmaceuticals, Inc.Inventors: Susan M. Freier, Huynh-Hoa Bui