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Ionis Pharmaceuticals Inc patents


Recent patent applications related to Ionis Pharmaceuticals Inc. Ionis Pharmaceuticals Inc is listed as an Agent/Assignee. Note: Ionis Pharmaceuticals Inc may have other listings under different names/spellings. We're not affiliated with Ionis Pharmaceuticals Inc, we're just tracking patents.

ARCHIVE: New 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 | Company Directory "I" | Ionis Pharmaceuticals Inc-related inventors


 new patent  Compounds and methods for the modulation of proteins

In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the... Ionis Pharmaceuticals Inc

 new patent  Antisense compounds and uses thereof

The present disclosure provides oligomeric compounds. The present disclosure provides metabolically stable linkers that do not rapidly degrade in vivo. In certain embodiments, the present disclosure provides metabolically stable linkers for use in attaching a conjugate group to an oligonucleotide.... Ionis Pharmaceuticals Inc

Compositions and methods

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.... Ionis Pharmaceuticals Inc

Compounds and methods for improved cellular uptake of antisense compounds

The present invention provides method of increasing the efficacy and potency of antisense compounds. In certain embodiments, the invention provides methods for improved cellular uptake.... Ionis Pharmaceuticals Inc

Compositions comprising alternating 2'-modified nucleosides for use in gene modulation

The present invention provides compositions comprising at least one oligomeric compound comprising an alternating motif and further include a region that is complementary to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments... Ionis Pharmaceuticals Inc

Modulation of hepatitis b virus (hbv) expression

Disclosed herein are antisense compounds and methods for decreasing HBV mRNA, DNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate HBV-related diseases, disorders or conditions.... Ionis Pharmaceuticals Inc

Modulation of ube3a-ats expression

Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for. Several embodiments provided herein relate to the discovery that antisense compounds targeting UBE3A-ATS induce paternal expression of UBE3A. Several embodiments are drawn... Ionis Pharmaceuticals Inc

Conjugated antisense compounds and their use

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the gomeric compounds are conjugated to N-Acetylgalactosamine or to N-Acetylgalactosamine analogues.... Ionis Pharmaceuticals Inc

Compositions for modulating c9orf72 expression

Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal. Such methods are useful to treat, prevent, ameliorate, or slow progression of neurodegenerative diseases in an individual in need thereof.... Ionis Pharmaceuticals Inc

Methods for treating hypercholesterolemia

Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an... Ionis Pharmaceuticals Inc

Compounds and methods for the modulation of comp

In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. In certain embodiments, the target nucleic acid is a nucleic acid that encodes cartilage oligomeric matrix protein. In certain embodiments, the target nucleic acid is a nucleic acid expressed... Ionis Pharmaceuticals Inc

Antisense oligonucleotides directed against connective tissue growth factor and uses thereof

This invention provides compounds which comprise modified oligonucleotides capable of inhibitory expression of connective tissue factor and composition containing same as well as methods of treating hyperprolific disorders and fibrotic diseases, and of reducing scarring resulting from wound healing using such compounds.... Ionis Pharmaceuticals Inc

Method for solution phase detritylation of oligomeric compounds

Provided herein are methods for the synthesis of oligomeric compounds wherein removal of the 5′-terminal trityl group is performed at reduced temperature and lower pH relative to standard methods. In certain embodiments, the present methods provide detritylated oligomeric compounds having a reduced percentage of depurination relative to the same detritylated... Ionis Pharmaceuticals Inc

Selective antisense compounds and uses thereof

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.... Ionis Pharmaceuticals Inc

Methods of predicting toxicity

Described herein are compounds useful for the treatment and investigation of diseases, methods for the prediction of in vivo toxicity of compounds useful for the treatment and investigation of diseases, and methods of discovering and identifying compounds useful for the treatment and investigation of diseases that have reduced in vivo... Ionis Pharmaceuticals Inc

Modulation of agpat5 expression

Provided herein are methods, compounds, and compositions for reducing expression of an AGPAT5 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids, insulin resistance and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate... Ionis Pharmaceuticals Inc

Modulation of prekallikrein (pkk) expression

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.... Ionis Pharmaceuticals Inc

Antisense compounds and uses thereof

The present disclosure provides methods for sensitizing a cell for modulation by 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... Ionis Pharmaceuticals Inc

Modulation of apolipoprotein c-iii expression

Compounds, compositions and methods are provided for modulating the expression of apolipoprotein C-III. The compositions comprise oligonucleotides, targeted to nucleic acid encoding apolipoprotein C-III. Methods of using these compounds for modulation of apolipoprotein C-III expression and for diagnosis and treatment of disease associated with expression of apolipoprotein C-III are provided... Ionis Pharmaceuticals Inc

Antisense compounds and uses thereof

The present invention provides compounds comprising oligonucleotides complementary to a CLN3 transcript. Certain such compounds are useful for hybridizing to a CLN3 transcript, including but not limited to a CLN3 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CLN3 transcript. In certain... Ionis Pharmaceuticals Inc

Modulation of apolipoprotein ciii (apociii) expression

Provided herein are methods, compounds, and compositions for reducing expression of ApoCIII mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for increasing HDL levels and/or improving the ratio of TG to HDL and reducing plasma lipids and plasma glucose in an animal. Such methods,... Ionis Pharmaceuticals Inc

Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations

Provided herein are methods, compounds, and compositions for reducing expression of ApoCIII mRNA and protein in a patient with Fredrickson Type I dyslipidemia, FCS, LPLD. Also provided herein are methods, compounds, and compositions for treating, preventing, delaying, or ameliorating Fredrickson Type I dyslipidemia, FCS, LPLD, in a patient. Further provided... Ionis Pharmaceuticals Inc

Modulation of enhancer rna mediated gene expression

Disclosed herein are methods and compounds for inhibiting gene expression by inhibiting enhancer RNAs (eRNAs). Such methods and compounds are useful for reducing expression of certain genes, many of which are associated with a variety of diseases and disorders.... Ionis Pharmaceuticals Inc

Antisense oligonucleotide compositions

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... Ionis Pharmaceuticals Inc

Compositions and methods for modulating tau expression

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... Ionis Pharmaceuticals Inc

Antisense compounds targeting apolipoprotein e receptor 2

The present invention provides compounds comprising oligonucleotides complementary to an LRP8 transcript. Certain such compounds are useful for hybridizing to an LRP8 transcript, including but not limited, to an LRP8 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRP8 transcript. In certain... Ionis Pharmaceuticals Inc

Antisense compounds

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.... Ionis Pharmaceuticals Inc

Methods and compositions for modulating apolipoprotein (a) expression

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... Ionis Pharmaceuticals Inc

Nucleoside derivatives as inhibitors of rna-dependent rna viral polyermase

The present invention provides nucleoside compounds and certain derivatives thereof which are inhibitors of RNA-dependent RNA viral polymerase. These compounds are inhibitors of RNA-dependent RNA viral replication and are useful for the treatment of RNA-dependent RNA viral infection. They are particularly useful as inhibitors of hepatitis C virus (HCV) NS5B... Ionis Pharmaceuticals Inc

Compositions and methods for modulating pkk expression

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.... Ionis Pharmaceuticals Inc

Methods for modulating ataxin 2 expression

Disclosed herein are methods for decreasing Ataxin 2 mRNA and protein expression. Such methods are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism. Provided are methods for reducing... Ionis Pharmaceuticals Inc

Compositions for modulating ataxin 2 expression

Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.... Ionis Pharmaceuticals Inc

Compositions and methods for modulating growth hormone receptor expression

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating a disease associated with excess growth hormone using antisense compounds oligonucleotides targeted to growth hormone receptor (GHR).... Ionis Pharmaceuticals Inc

Compositions and methods for modulating complement factor b expression

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.... Ionis Pharmaceuticals Inc

Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom

The present invention provides novel bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom. Incorporation of one or more of the bicyclic carbocyclic nucleosides into an oligomeric compound is expected to enhance one or more properties of the oligomeric compound. In certain embodiments, the oligomeric compounds provided herein hybridize to a... Ionis Pharmaceuticals Inc

05/25/17 / #20170145413

Cancer treatment

In certain embodiments, methods, compounds, and compositions for treating B-cell lymphoma or hepatocellular carcinoma by inhibiting expression of ST AT3 mRNA or protein in an animal are provided herein. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate B-celllymphoma or hepatocellular carcinoma. The STAT (signal transducers and... Ionis Pharmaceuticals Inc

05/25/17 / #20170145424

Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds

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.... Ionis Pharmaceuticals Inc

05/11/17 / #20170130224

Linkage modified gapped oligomeric compounds and uses thereof

The present invention provides gapped oligomeric compounds. More particularly the gapped oligomeric compounds provided herein comprise at least one modified internucleoside linkage in the gap region. Such gapped oligomeric compounds have one or more improved properties such as selectivity, potency, improved toxicity profile and or improved proinflammatory profile. Certain such... Ionis Pharmaceuticals Inc

05/04/17 / #20170121710

Methods and compositions for modlating apolipoprotein (a) expression

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... Ionis Pharmaceuticals Inc

03/16/17 / #20170073688

Modulation of tmprss6 expression

Disclosed herein are antisense compounds and methods 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 hemochromatosis or β-thalassemia can be ameliorated or prevented with the administration of antisense compounds targeted to TMPRSS6.... Ionis Pharmaceuticals Inc

03/16/17 / #20170073689

Modulators of growth hormone receptor

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating a disease associated with excess growth hormone using antisense compounds oligonucleotides targeted to growth hormone receptor (GHR).... Ionis Pharmaceuticals Inc

02/16/17 / #20170043025

Method for synthesis of reactive conjugate clusters

Provided herein are improved methods for the synthesis of reactive conjugate clusters and intermediates used in such methods. In particular, improvements are provided that enhance the synthesis of reactive conjugate clusters by reducing the number of synthetic steps required. The reactive conjugate clusters prepared using the improved methods don't include... Ionis Pharmaceuticals Inc

02/16/17 / #20170044526

Linkage modified oligomeric compounds

The present invention provides oligomeric compounds comprising at least one neutral methoxypropyl phosphonate modified internucleoside linkage. Such oligomeric compounds have one or more improved properties such as selectivity, potency, improved toxicity profile and or improved proinflammatory profile. Such oligomeric compounds have enhanced stability to exposure to base during synthesis. Certain... Ionis Pharmaceuticals Inc

02/16/17 / #20170044539

Oligomeric compounds comprising alpha-beta-constrained nucleic acid

The present disclosure provides oligomeric compounds comprising at least one α-β-constrained nucleic acid as provided herein. More particularly, the α-β-constrained nucleic acid provided herein comprise an optionally modified nucleoside with a phosphorus containing constrained internucleoside linkage such as for example a cyclic phosphate internucleoside linkage. The α-β-constrained nucleic acid provided... Ionis Pharmaceuticals Inc

02/09/17 / #20170035798

Modulation of factor 11 expression

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... Ionis Pharmaceuticals Inc

02/09/17 / #20170037409

Modulation of angiopoietin-like 3 expression

Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one... Ionis Pharmaceuticals Inc

02/09/17 / #20170037410

Compositions for modulating sod-1 expression

Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS).... Ionis Pharmaceuticals Inc

01/19/17 / #20170015995

Compositions and methods for modulation of smn2 splicing in a subject

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.... Ionis Pharmaceuticals Inc

01/05/17 / #20170002359

Modulation of prekallikrein (pkk) expression

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.... Ionis Pharmaceuticals Inc








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