Abstract

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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

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.

IPC Classes  ?

• C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
• A61K 31/7048 - Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin

Abstract

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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.

IPC Classes  ?

• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

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 embodiments, such compounds are used to treat one or more symptoms associated with Batten Disease.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides

Abstract

Provided are oligomeric agents, pharmaceutical compositions, and methods for reducing the amount or activity of APOE RNA in a cell or subject, and in certain instances reducing the amount of ApoE protein in a cell or subject. Such oligomeric agents, pharmaceutical compositions, and methods are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include cognitive impairment, progressive memory loss, a decline in language skills, behavioral abnormality, dementia, difficulty performing daily activities, aphasia, agnosia, apraxia, loss of motor function, amyloid plaque, neurofibrillary tangle, and/or neuroinflammation.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
• C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides

Abstract

Provided are oligomeric agents, methods, and pharmaceutical compositions for reducing the amount or activity of a target RNA in a cell or a subject, and in certain instances reducing the amount of a protein encoded by such RNA in a cell or a subject. Such oligomeric agents, methods, and pharmaceutical compositions are useful in the treatment of one or more diseases or conditions.

IPC Classes  ?

• C12N 15/13 - Immunoglobulins
• A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals for the treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. Collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes. Medical services; medical information services; providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via a website; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via an online database; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Abstract

Provided are oligomeric agents, pharmaceutical compositions, and methods for reducing the amount or activity of APOE RNA in a cell or subject, and in certain instances reducing the amount of ApoE protein in a cell or subject. Such oligomeric agents, pharmaceutical compositions, and methods are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include cognitive impairment, progressive memory loss, a decline in language skills, behavioral abnormality, dementia, difficulty performing daily activities, aphasia, agnosia, apraxia, loss of motor function, amyloid plaque, neurofibrillary tangle, and/or neuroinflammation.

IPC Classes  ?

• C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters

Abstract

Provided are oligomeric agents, methods, and pharmaceutical compositions for reducing the amount or activity of a target RNA in a cell or animal, and in certain instances reducing the amount of a target protein in a cell or animal.

IPC Classes  ?

• A61K 31/70 - CarbohydratesSugarsDerivatives thereof
• A61K 51/04 - Organic compounds
• C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
• A61K 51/02 - Preparations containing radioactive substances for use in therapy or testing in vivo characterised by the carrier

Goods & Services

Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet.

Goods & Services

Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet.

Goods & Services

Pharmaceutical preparations and substances for the treatment of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances for the treatment of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances for the treatment of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet

Goods & Services

Pharmaceutical preparations and substances for the treatment of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances for the treatment of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances for the treatment of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet

Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GYS1 RNA in a cell or subject, and in certain instances reducing the amount of GYS1 protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a glycogen storage disease. Such glycogen storage diseases include Lafora disease, adult polyglucosan body disease (APBD), Andersen's disease, and Pompe disease.

IPC Classes  ?

• C12N 15/115 - Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith
• A61K 47/46 - Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts

Abstract

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

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
• C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Provided are oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PLN RNA in a cell or animal, and in certain instances reducing the amount of phospholamban protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat cardiomyopathy, heart failure, or arrhythmia.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 9/04 - Inotropic agents, i.e. stimulants of cardiac contractionDrugs for heart failure

Abstract

Provided are RNAi agents, methods, and pharmaceutical compositions for reducing the amount or activity of tau RNA in a cell or animal, and in certain instances reducing the amount of tau protein in a cell or animal. Such RNAi agents, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease, including a tauopathy, Alzheimer's disease, fronto-temporal dementia (FTD), FTDP-17, progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), corticobasal ganglionic degeneration (CBD), epilepsy, or Dravet's Syndrome.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

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.

IPC Classes  ?

• A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Provided are oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of Protocadherin 19 (PCDH19) RNA in a cell or subject, and in certain instances reducing the amount of PCDH19 protein in a cell or subject. 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 PCDH19 Epilepsy. Such symptoms or hallmarks include seizures, cognitive impairment, intellectual disabilities, autism spectrum disorder, behavioral problems, aggression, anxiety, obsessive-compulsive disorder, hyperactivity, attention deficit disorder (ADD), and attention deficit hyperactivity disorder (ADHD).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 9/00 - Medicinal preparations characterised by special physical form

Abstract

Provided herein are methods of administering gapmer oligomeric compounds with GalNAc conjugate groups to a human.

IPC Classes  ?

• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 9/00 - Medicinal preparations characterised by special physical form
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• A61P 3/06 - Antihyperlipidemics
• C07H 19/20 - Purine radicals with the saccharide radical being esterified by phosphoric or polyphosphoric acids
• C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
• C07H 21/02 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
• C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of KCNT1 RNA in a cell or subject, and in certain instances reducing the amount of KCNT1 protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurological condition. Such symptoms and hallmarks include seizures, encephalopathy, and behavioral abnormalities. Non-limiting examples of neurological conditions that benefit from these compounds, methods, and pharmaceutical compositions are epilepsy of infancy with migrating focal seizures (EIMFS), autosomal dominant nocturnal frontal lobe epilepsy (ADNFLE), West syndrome, and Ohtahara syndrome.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals for the treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. Collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes. Medical services; medical information services; providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via a web site; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, and rare diseases and disorders via an online database; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. Collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes. Medical services; medical information services; collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via a web site; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Abstract

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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61K 47/02 - Inorganic compounds
• A61K 47/46 - Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts
• A61P 25/16 - Anti-Parkinson drugs

Abstract

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 individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy

Abstract

Provided herein are methods for decreasing Ataxin-2 mRNA expression. Such methods are useful to ameliorate symptoms of Ataxin-2 associated diseases. Such Ataxin-2 associated diseases include amyotrophic lateral sclerosis (ALS). Such symptoms include loss of motor function, reduced CMAP amplitude, denervation, and loss of motor neurons.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
• C12N 5/0793 - Neurons

Abstract

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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 35/30 - NervesBrainEyesCorneal cellsCerebrospinal fluidNeuronal stem cellsNeuronal precursor cellsGlial cellsOligodendrocytesSchwann cellsAstrogliaAstrocytesChoroid plexusSpinal cord tissue
• A61K 47/02 - Inorganic compounds
• A61P 25/00 - Drugs for disorders of the nervous system

Goods & Services

Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, alexander disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet.

Goods & Services

Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, alexander disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet.

Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

The present embodiments provide methods, compounds, and compositions useful for inhibiting DGAT2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DGAT2.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
• C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. Collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes. Medical services; medical information services; providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via a website; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via an online database; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. Collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes. Medical services; medical information services; providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via a website; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via an online database; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Goods & Services

(1) Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, alexander disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; pharmaceutical preparations containing zilganersen. (1) Providing health information; providing healthcare information; medical information services provided via the Internet.

Goods & Services

(1) Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, alexander disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; pharmaceutical preparations containing zilganersen. (1) Providing health information; providing healthcare information; medical information services provided via the Internet.

Abstract

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. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C07H 21/02 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical

Abstract

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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 25/00 - Drugs for disorders of the nervous system

Goods & Services

Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory diseases and disorders. Medical services; medical information services; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of genetic diseases and disorders; medical consulting services in the field of genetic diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of genetic diseases and disorders, via an online database that collects data and information on genetic diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of genetic diseases and disorders; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; medical consulting services in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, via an online database that collects data and information on cardiovascular, metabolic, neurological and inflammatory diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of cardiovascular, metabolic, neurological and inflammatory diseases and disorders.

Abstract

The present invention provides gapped oligomeric compounds comprising from 1 to about 3 internucleoside linkages having one of formulas I to XVI. In certain embodiments, inclusion of from 1 to about 3 internucleoside linkages of one of formulas I to XVI, improves selectivity for a target RNA relative to an off target RNA. In certain embodiments, the improved selectivity also provides an improved toxicity profile. 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 of activity or expression of the target nucleic acid in a cell.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca

Goods & Services

Financial and insurance consultation services, namely, patient advisory, support and consultancy services regarding patient finances, insurance and financing of medical treatment; providing financial and insurance information, namely, providing information and guidance in connection with health insurance benefit plans and other patient financial assistance resources; financial and insurance consultation services, namely, insurance and medical and pharmaceutical access consultation services, namely, assisting patients, their caregivers, and healthcare providers to identify the scope of coverage offered by patient medical and pharmaceutical insurance and to identify other financial assistance resources for payment of patient treatment; insurance consultation services, namely, assisting patients, their caregivers, and healthcare providers to obtain authorization and payment for medical and pharmaceutical costs; Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals

Goods & Services

Patient advisory, support and consultancy services regarding access to medical treatment of hereditary angioedema; providing healthcare information, medical information, and pharmaceutical advice online, by telephone, and through distribution of printed healthcare materials to healthcare providers, patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to and reimbursement for pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the field of hereditary angioedema and edema diseases and disorders, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; medical consulting services in the field of hereditary angioedema and edema diseases and disorders, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of hereditary angioedema and edema diseases and disorders, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders, and collecting data and information on hereditary angioedema, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and collecting data and information on rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information

Goods & Services

Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of hereditary angioedema; education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; online journals, namely, blogs featuring information on hereditary angioedema; online journals, namely, blogs featuring information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; online journals, namely, blogs featuring information on hereditary angioedema and edema diseases and disorders; providing a website featuring blogs and non-downloadable publications in the nature of articles, blogs, brochures, and guides in the fields of hereditary angioedema, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders

Abstract

Provided are oligomeric agents, methods, and pharmaceutical compositions for reducing the amount or activity of APOC3 RNA in a cell or animal, and in certain instances reducing the amount of ApoCIII protein in a cell or animal. Such oligomeric agents, methods, and pharmaceutical compositions are useful to treat or manage hypertriglyceridemia and/or cardiovascular disease (CVD).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 9/10 - Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis

Abstract

Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Provided are oligomeric duplexes, oligomeric compounds and antisense agents, methods, and pharmaceutical compositions for reducing the amount or activity of LPA RNA in a cell or animal, and in certain instances reducing the amount of Lp(a) in a subject. Such oligomeric duplexes, oligomeric compounds and agents, methods, and pharmaceutical compositions are useful to treat or manage inflammatory, cardiovascular and/or metabolic diseases, disorders, or conditions.

IPC Classes  ?

• C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides

Goods & Services

(1) Financial and insurance consultation services, namely, patient advisory, support and consultancy services regarding patient finances, insurance and access to medical treatment; providing financial and insurance information, namely, providing information and guidance in connection with health insurance benefit plans and other patient financial assistance resources; financial and insurance consultation services, namely, insurance and medical and pharmaceutical access consultation services, namely, assisting patients, their caregivers, and healthcare providers to identify the scope of coverage offered by patient medical and pharmaceutical insurance and to identify other financial assistance resources for payment of patient treatment; insurance consultation services, namely, assisting patients, their caregivers, and healthcare providers to obtain authorization and payment for medical and pharmaceutical costs (2) Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; online journals, namely, blogs featuring information on hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; providing a website featuring blogs and non-downloadable publications in the nature of articles, blogs, brochures, and guides in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders (3) Providing healthcare information, medical information, and pharmaceutical information online, by telephone, and through distribution of printed healthcare materials to healthcare providers, patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to and reimbursement for pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; providing healthcare information; medical information services; providing a website featuring information in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; medical consulting services in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, asthma, and rare diseases and disorders, and collecting data and information on hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders

Abstract

Provided are methods for reducing the amount of a contaminant in a sample containing an oligomeric compound. The method may comprise chromatography using a mobile phase comprising a strong salt and a chaotrope. Also provided are oligomeric compounds prepared by the method.

IPC Classes  ?

• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical

Abstract

Provided are guided nucleic acid binding agents, systems comprising guided nucleic acid binding agents, guides, and methods of use thereof. Such guided nucleic acid binding agents and systems are useful for gene editing.

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• A61K 38/46 - Hydrolases (3)
• A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
• C07K 14/195 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria
• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/52 - Genes encoding for enzymes or proenzymes
• C12N 15/86 - Viral vectors
• C12N 5/10 - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
• A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells

Abstract

Provided are antisense agents, RNAi agents, pharmaceutical compositions, and methods of use 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. In certain embodiments, also provided herein are oligomeric compounds and oligomeric duplexes for reducing the amount or activity of proteolipid protein 1 (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, delay in motor function development, cognitive dysfunction, speech dysfunction, spasticity, ataxia, seizures, choreiform movements, and death. Such leukodystrophies include Pelizaeus-Merzbacher disease.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 1/00 - Drugs for disorders of the alimentary tract or the digestive system

Goods & Services

(1) Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals for the treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. (1) Collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes. (2) Medical services; medical information services; providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via a website; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via an online database; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Abstract

Provided are oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of IFNAR1 RNA in a cell or an animal, and in certain instances, reducing the amount of IFNAR1 protein in a cell or animal. Such oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions are useful to treat diseases and conditions associated with neuroinflammation, including Aicardi-Goutieres 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.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection

Goods & Services

(1) Financial and insurance consultation services, namely, patient advisory, support and consultancy services regarding patient finances, insurance and access to medical treatment; providing financial and insurance information, namely, providing information and guidance in connection with health insurance benefit plans and other patient financial assistance resources; financial and insurance consultation services, namely, insurance and medical and pharmaceutical access consultation services, namely, assisting patients, their caregivers, and healthcare providers to identify the scope of coverage offered by patient medical and pharmaceutical insurance and to identify other financial assistance resources for payment of patient treatment; insurance consultation services, namely, assisting patients, their caregivers, and healthcare providers to obtain authorization and payment for medical and pharmaceutical costs (2) Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of genetic diseases and disorders; online journals, namely, blogs featuring information on genetic diseases and disorders; education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of edema, cardiovascular, metabolic, neurological, inflammatory, respiratory, pulmonary, renal, ophthalmologic, and rare diseases and disorders; online journals, namely, blogs featuring information on diseases and disorders of the cardiovascular, metabolic, neurological and inflammatory diseases and disorders; providing a website featuring blogs and non-downloadable publications in the nature of articles, blogs, brochures, and guides in the fields of triglycerides, triglyceride management, familial chylomicronemia syndrome, severe hypertriglyceridemia, and pancreatitis (3) Providing healthcare information, medical information, and pharmaceutical information online, by telephone, and through distribution of printed healthcare materials to healthcare providers, patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to and reimbursement for pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; providing healthcare information; medical information services; providing a website featuring information regarding triglycerides, triglyceride management, familial chylomicronemia syndrome, severe hypertriglyceridemia, and pancreatitis; providing medical quizzes and assessments via a website that are designed to provide custom tailored outputs about recommended resources and treatments associated with a defined set of symptoms and concerns; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the field of genetic diseases and disorders; medical consulting services in the field of genetic diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of genetic diseases and disorders, and collecting data and information on genetic diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of genetic diseases and disorders; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; medical consulting services in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, and collecting data and information on cardiovascular, metabolic, neurological and inflammatory diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of cardiovascular, metabolic, neurological and inflammatory diseases and disorders

Goods & Services

(1) Financial and insurance consultation services, namely, patient advisory, support and consultancy services regarding patient finances, insurance and access to medical treatment; providing financial and insurance information, namely, providing information and guidance in connection with health insurance benefit plans and other patient financial assistance resources; financial and insurance consultation services, namely, insurance and medical and pharmaceutical access consultation services, namely, assisting patients, their caregivers, and healthcare providers to identify the scope of coverage offered by patient medical and pharmaceutical insurance and to identify other financial assistance resources for payment of patient treatment; insurance consultation services, namely, assisting patients, their caregivers, and healthcare providers to obtain authorization and payment for medical and pharmaceutical costs (2) Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, and providers in the field of genetic diseases and disorders; online journals, namely, blogs featuring information on genetic diseases and disorders; education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, and providers in the field of edema, cardiovascular, metabolic, neurological, inflammatory, respiratory, pulmonary, renal, ophthalmologic, and rare diseases and disorders; online journals, namely, blogs featuring information on diseases and disorders of the cardiovascular, metabolic, neurological and inflammatory diseases and disorders; providing a website featuring blogs and non-downloadable publications in the nature of articles, blogs, brochures, and guides in the fields of triglycerides, triglyceride management, familial chylomicronemia syndrome, severe hypertriglyceridemia, and pancreatitis (3) Providing healthcare information, medical information, and pharmaceutical information online, by telephone, and through distribution of printed healthcare materials to healthcare providers, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to and reimbursement for pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; providing healthcare information; medical information services; providing a website featuring information regarding triglycerides, triglyceride management, familial chylomicronemia syndrome, severe hypertriglyceridemia, and pancreatitis; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, and providers in the field of genetic diseases and disorders; medical consulting services in the field of genetic diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of genetic diseases and disorders, and collecting data and information on genetic diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of genetic diseases and disorders; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, and providers in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; medical consulting services in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, and collecting data and information on cardiovascular, metabolic, neurological and inflammatory diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of cardiovascular, metabolic, neurological and inflammatory diseases and disorders

Abstract

Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 9/00 - Medicinal preparations characterised by special physical form
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters

Abstract

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.

IPC Classes  ?

• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

The present embodiments provide methods of modulating gene expression by recruitment of endogenous transcription factors.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61K 47/60 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

Provided herein are pharmaceutical compositions comprising an oligomeric compound and a pharmaceutically acceptable diluent wherein the pharmaceutical composition is formulated to ameliorate an acute neurotoxicity (e.g., an acute activation) in a subject in need thereof. Also provided herein are pharmaceutical compositions comprising an oligomeric compound and a pharmaceutically acceptable diluent wherein the pharmaceutical composition is formulated to prevent an acute neurotoxicity (e.g., an acute activation) in a subject in need thereof.

IPC Classes  ?

• A61K 47/02 - Inorganic compounds
• A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy

Goods & Services

Organizing and conducting support groups in the field of healthcare; Providing emotional counseling and emotional support services for individuals in the field of healthcare

Goods & Services

Mobile healthcare services; Health care services, namely, providing disease and chronic condition management

Goods & Services

Providing on-line non-downloadable software for use in organizing, and conducting individual support groups, virtual peer matching, and providing individuals with access to healthcare and support groups

Abstract

The present disclosure provides oligomeric compounds (including oligomeric compounds that are antisense agents or portions thereof) comprising a modified oligonucleotide having at least one chemical modification.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose

Abstract

Provided are methods of administering ION582 for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in the cell or subject. In certain embodiments, the subject has Angelman syndrome (AS).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61P 43/00 - Drugs for specific purposes, not provided for in groups

Abstract

Provided herein are methods of reducing the amount of AGT RNA and/or AGT protein in a subject having or at risk for heart failure, such as heart failure with reduced ejection fraction (HFrEF), heart failure with mid-range ejection fraction (HFmrEF), or heart failure with preserved ejection fraction (HFpEF), and, in particular instances, a subject who is, or is at risk of being, intolerant to treatment with a renin-angiotensin-aldosterone system (RAAS) inhibitor. Also provided herein are methods for treating heart failure (such as HFrEF, HFmrEF or HFpEF). Methods provided herein include administering to a subject an oligomeric agent containing a modified oligonucleotide containing a nucleobase sequence complementary to a sequence in an AGT nucleic acid, such as ION904. Further provided herein are pharmaceutical compositions containing ION904.

IPC Classes  ?

• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• C07H 21/02 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 9/12 - Antihypertensives

Abstract

Disclosed herein are antisense compounds and methods for decreasing alpha-synuclein mRNA and protein expression. Also disclosed herein are methods for treating, preventing, and ameliorating neurodegenerative diseases in an individual in need thereof.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

The present embodiments provide methods, compounds, and compositions useful for inhibiting MALAT1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with MALAT1.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Provided are compounds, methods, and pharmaceutical compositions for modulating the expression of CLN3 RNA in a cell or animal, and in certain instances modulating the expression of CLN3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are capable of useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

Goods & Services

(1) Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory diseases and disorders. (1) Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of genetic diseases and disorders, online journals, namely, blogs featuring information on genetic diseases and disorders; education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, online journals, namely, blogs featuring information on diseases and disorders of the cardiovascular, metabolic, neurological and inflammatory diseases and disorders. (2) Pharmaceutical research and development in the field of genetic diseases and disorders, providing medical and scientific research and medical and scientific research information in the field of genetic diseases and disorders, research in the field of genetic diseases and disorders; pharmaceutical research and development in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, providing medical and scientific research and medical and scientific research information in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, research in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders. (3) Medical services; medical information services; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of genetic diseases and disorders; medical consulting services in the field of genetic diseases and disorders; healthcare services, namely, providing medical and healthcare information via an online database in the field of genetic diseases and disorders; providing medical and healthcare information concerning the screening, diagnosis, and treatment of genetic diseases and disorders; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; medical consulting services in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; healthcare services, namely, providing medical and healthcare information via an online database in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; providing medical and healthcare information concerning the screening, diagnosis, and treatment of cardiovascular, metabolic, neurological and inflammatory diseases and disorders.

Abstract

Provided are complexes and methods for targeting cells of interest for delivery of a cargo such as an oligomeric compound or an oligomeric duplex. In particular, the complex may comprise a transferrin receptor binding moiety and a half-life extension moiety.

IPC Classes  ?

• C07K 14/00 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof
• A61K 9/00 - Medicinal preparations characterised by special physical form
• A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
• A61K 38/00 - Medicinal preparations containing peptides
• C07K 9/00 - Peptides having up to 20 amino acids, containing saccharide radicals and having a fully defined sequenceDerivatives thereof
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy

Goods & Services

(1) Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory diseases and disorders. (1) Medical services; medical information services; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of genetic diseases and disorders; medical consulting services in the field of genetic diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of genetic diseases and disorders, via an online database that collects data and information on genetic diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of genetic diseases and disorders; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; medical consulting services in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, via an online database that collects data and information on cardiovascular, metabolic, neurological and inflammatory diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of cardiovascular, metabolic, neurological and inflammatory diseases and disorders.

Abstract

Provided are oligomeric compounds, methods, and pharmaceutical compositions for modulating expression of progranulin RNA, or modulating expression of progranulin protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurological disease or disorder. Such neurological diseases or disorders include those associated with insufficient expression of progranulin, including frontotemporal dementia, frontotemporal lobar degeneration, Alzheimer's disease, amyotrophic lateral sclerosis and neuronal ceroid lipofuscinosis. Such symptoms or hallmarks include deterioration in behavior and personality, language impairment, disturbances or alterations in muscle or motor functions, memory loss, cognitive dysfunction, tremor, seizures, or dizziness.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

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 uORF inhibitor is an antisense compound.

IPC Classes  ?

• C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/67 - General methods for enhancing the expression
• C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides

Goods & Services

Medical services; medical information services; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, and rare diseases and disorders, and collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely, hereditary angioedema; pharmaceutical products and preparations in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely, hereditary angioedema; pharmaceutical products and preparations in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders

Goods & Services

Medical services; medical information services; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, and rare diseases and disorders, and collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders

Abstract

Provided are oligomeric duplexes, oligomeric compounds and antisense agents, methods, and pharmaceutical compositions for reducing the amount or activity of LPA RNA in a cell or animal, and in certain instances reducing the amount of Lp(a) in a subject. Such oligomeric duplexes, oligomeric compounds and agents, methods, and pharmaceutical compositions are useful to treat or manage inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions.

IPC Classes  ?

• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy

Abstract

Provided are oligomeric duplexes, oligomeric compounds and antisense agents, methods, and pharmaceutical compositions for reducing the amount or activity of LPA RNA in a cell or animal, and in certain instances reducing the amount of Lp(a) in a subject. Such oligomeric duplexes, oligomeric compounds and agents, methods, and pharmaceutical compositions are useful to treat or manage inflammatory, cardiovascular and/or metabolic diseases, disorders, or conditions.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides

Abstract

Provided are oligomeric agents, methods, and pharmaceutical compositions for reducing the amount or activity of LPA RNA in a cell or animal, and in certain instances reducing the amount of Lp(a) in a subject. Such oligomeric agents, methods, and pharmaceutical compositions are useful to treat or manage inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions.

IPC Classes  ?

• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61P 9/00 - Drugs for disorders of the cardiovascular system
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Provided are oligomeric agents, methods, and pharmaceutical compositions for reducing the amount or activity of APoC3RNA in a cell or animal, and in certain instances reducing the amount of ApoCIII protein in a cell or animal. Such oligomeric agents, methods, and pharmaceutical compositions are useful to treat or manage hypertriglyceridemia and/or cardiovascular disease (CVD).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides

Abstract

Provided are guided nucleic acid binding agents, systems comprising guided nucleic acid binding agents, and methods of use thereof. Such guided nucleic acid binding agents and systems are useful for gene editing and epigenetic modulation of gene expression.

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• A61K 38/46 - Hydrolases (3)
• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C12N 15/90 - Stable introduction of foreign DNA into chromosome

Goods & Services

Pharmaceutical preparations and substances for the treatment of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances for the treatment of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances for the treatment of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet

Goods & Services

Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet.

Goods & Services

Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet.

Goods & Services

Pharmaceutical preparations and substances for the treatment of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances for the treatment of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances for the treatment of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet

Abstract

Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Provided are oligomeric agents, methods, and pharmaceutical compositions for reducing the amount or activity of ATN1 RNA in a cell or a subject, and in certain instances reducing the amount of atrophin-1 protein in a cell or a subject. Such oligomeric agents, methods, and pharmaceutical compositions are useful to treat a polyglutamine (polyQ) disease or disorder associated with ATN1.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61P 25/08 - AntiepilepticsAnticonvulsants
• A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

Abstract

The present embodiments provide methods, compounds, and compositions useful for inhibiting APOL1 expression, which may be useful for treating, preventing, or ameliorating a disease associated with APOL1.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 13/02 - Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers

Goods & Services

Medical services; medical information services; providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders via website; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, and rare diseases and disorders, and collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GYSI RNA in a cell or subject, and in certain instances reducing the amount of GYSI protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a glycogen storage disease. Such glycogen storage diseases include Lafora disease, adult polyglucosan body disease (APBD), Andersen's disease, and Pompe disease.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of TUBB4A RNA in a cell or subject, and in certain instances reducing the amount of TUBB4A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a disease or disorder associated with TUBB4A. Such symptoms and hallmarks include hypomyelination, demyelination, dysphonia, dystonia, ataxia, spasticity, atrophy of cerebellar and/or basal ganglia, poor vision, rigidity, microcephaly, focal cortical dysplasia, seizures, and early childhood death.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61P 25/14 - Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia

Goods & Services

Financial and insurance consultation services, namely, patient advisory, support and consultancy services regarding patient finances, insurance and access to medical treatment; providing financial and insurance information, namely, providing information and guidance in connection with health insurance benefit plans and other patient financial assistance resources; financial and insurance consultation services, namely, insurance and medical and pharmaceutical access consultation services, namely, assisting patients, their caregivers, and healthcare providers to identify the scope of coverage offered by patient medical and pharmaceutical insurance and to identify other financial assistance resources for payment of patient treatment; insurance consultation services, namely, assisting patients, their caregivers, and healthcare providers to obtain authorization and payment for medical and pharmaceutical costs

Goods & Services

Education and medical training services, namely, providing seminars, non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; online journals, namely, blogs featuring information on hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; providing a website featuring blogs and non-downloadable publications in the nature of articles, blogs, brochures, and guides in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders

Goods & Services

Providing healthcare information, medical information, and pharmaceutical information online, by telephone, and through distribution of printed healthcare materials to healthcare providers, patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to and reimbursement for pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; providing healthcare information; medical information services; providing a website featuring medical information in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; medical consulting services in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; healthcare services, namely, providing an online database comprised of medical and healthcare information in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, asthma, and rare diseases and disorders, and collecting data and information on hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders

Abstract

The present embodiments provide methods, compounds, and compositions useful for inhibiting PMP22 expression and for treating, preventing, or ameliorating a disease associated with PMP22.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 9/00 - Medicinal preparations characterised by special physical form
• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61P 25/02 - Drugs for disorders of the nervous system for peripheral neuropathies
• A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

Abstract

Disclosed is a method of treating a subject who has a UBD disease or disorder such as a liver disease or disorder. In one aspect, the method includes a step of administering an effective dose of an antisense or inhibitory nucleic acid to a subject in need thereof.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
• A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
• A61P 9/00 - Drugs for disorders of the cardiovascular system

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. Medical services; medical information services; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, and rare diseases and disorders, and collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.

Goods & Services

Pharmaceutical preparations and substances for the treatment of edema, namely hereditary angioedema; pharmaceuticals in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders. Medical services; medical information services; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; medical consulting services in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, and rare diseases and disorders, and collecting data and information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders.