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

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

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; telehealth and support group videoconferencing, scheduling services, referral services, healthcare support services

Goods & Services

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

Goods & Services

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

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

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

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

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; pharmaceuticals 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 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 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 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 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

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 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

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.

Abstract

Embodiments provided herein are directed to oligomeric agents comprising a CD29-binding moiety and a modified oligonucleotide for modulating the expression of a nucleic acid target in cells expressing CD29. In certain embodiments, an oligomeric agent comprises an oligonucleotide linked to a CD29-binding moiety. In certain embodiments, an oligomeric agent comprises an oligonucleotide, a conjugate linker, and a CD29-binding moiety. In certain embodiments, contacting a CD29-expressing cel! with an oligomeric agent provided herein modulates expression of a nucleic acid target in the cell. In certain embodiments, an oligomeric agent comprising a CD29-binding moiety selectively or preferentially targets a cell expressing CD29 compared to a cell not expressing CD29. In certain embodiments, a compound comprising a CD29-binding moiety selectively or preferentially targets a cell expressing CD29 compared to a compound not comprising a CD29-binding moiety. In certain embodiments, the CD29-binding conjugate moiety is an antibody or a fragment thereof.

IPC Classes  ?

• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
• 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 47/65 - Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
• A61K 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
• A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
• C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/115 - Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith

Goods & Services

Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory 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 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

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 preparations for the 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 for the treatment 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

Goods & Services

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

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 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

Goods & Services

Patient advisory, support and consultancy services regarding access to medical treatment in the nature of physician referrals 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 Patient advisory, support and consultancy services regarding access to medical treatment of genetic diseases and disorders; and treatment of cardiovascular, metabolic, neurological, and inflammatory diseases and disorders; and blood disorders

Goods & Services

Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals 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 healthcare information; medical information services; providing a website featuring medical information regarding triglycerides, triglyceride management, familial chylomicronemia syndrome, severe hypertriglyceridemia, and pancreatitis; medical testing services, namely, 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

Patient advisory, support and consultancy services regarding access to medical treatment in the nature of physician referrals 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 Patient advisory, support and consultancy services regarding access to medical treatment of genetic diseases and disorders; and treatment of cardiovascular, metabolic, neurological, and inflammatory diseases and disorders; and blood disorders

Goods & Services

Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals Providing healthcare information, medical information, and pharmaceutical advice 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 medical information regarding triglycerides, triglyceride management, familial chylomicronemia syndrome, severe hypertriglyceridemia, and pancreatitis; medical testing services, namely, 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

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 and their caregivers 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 and their caregivers to obtain authorization and payment for medical and pharmaceutical costs

Goods & Services

Providing healthcare information, medical information, and pharmaceutical information online, by telephone, and through distribution of printed healthcare materials to 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

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. Pharmaceutical research and development 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 scientific research and medical and scientific research information in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; research in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders for medical and scientific research purposes.

Goods & Services

Education and training services, namely, providing seminars, online 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, providing seminars, online 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

Abstract

Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein C-III (ApoCIII). In certain embodiments, the ApoCIII targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting ApoCIII for use in decreasing ApoCIII to treat, prevent, or ameliorate diseases, disorders or conditions related to ApoCIII. Certain diseases, disorders or conditions related to ApoCIII include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

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

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

Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

IPC Classes  ?

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

Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.

IPC Classes  ?

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

Abstract

Provided herein are oligomeric compounds comprising a bicycle ligand and a modified oligonucleotide. This compound may comprise a bicycle ligand as the cell-targeting moiety, and may also comprise a conjugate linker to connect the bicycle ligand and modified oligonucleotide. This compound may be used in conjunction with a pharmaceutically acceptable salt.

IPC Classes  ?

• 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 9/00 - Medicinal preparations characterised by special physical form
• A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Goods & Services

Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory 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 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. 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. 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.

Goods & Services

Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory 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 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. 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. 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 compounds, methods, and pharmaceutical compositions for reducing the amount or activity of HTT RNA in a cell or subject, and in certain instances reducing the amount of HTT protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to prevent, treat, or ameliorate at least one symptom or hallmark of a repeat expansion disease. Such repeat expansion diseases include myotonic dystrophy, amyotrophic lateral sclerosis, frontotemporal dementia, Huntington's disease, polyglutamine disorders, Fragile X syndrome, and spinocerebellar ataxia. Such symptoms or hallmarks include brain atrophy, muscle atrophy, nerve degeneration, uncontrolled movement, seizure, tremor, anxiety, and depression.

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

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 RNAi agents comprising a stereo-non-standard nucleoside in the seed region thereof.

IPC Classes  ?

• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61K 47/50 - 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
• 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
• A61K 47/66 - 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 a protein, peptide or polyamino acid the modifying agent being a pre-targeting system involving a peptide or protein for targeting specific cells
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• A61P 43/00 - Drugs for specific purposes, not provided for in groups
• C07H 19/06 - Pyrimidine radicals
• 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 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
• C12N 15/09 - Recombinant DNA-technology
• 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

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 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

Goods & Services

Patient advisory, support and consultancy services regarding access to medical treatment in the nature of physician referrals Patient advisory, support and consultancy services regarding access to medical treatment in the nature of physician referrals 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 and their caregivers 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 and their caregivers to obtain authorization and payment for medical and pharmaceutical costs 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 and their caregivers 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 and their caregivers to obtain authorization and payment for medical and pharmaceutical costs Patient advisory, support and consultancy services regarding access to medical treatment in the nature of physician referrals Patient advisory, support and consultancy services regarding access to medical treatment in the nature of physician referrals 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 and their caregivers 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 and their caregivers to obtain authorization and payment for medical and pharmaceutical costs 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 and their caregivers 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 and their caregivers to obtain authorization and payment for medical and pharmaceutical costs

Goods & Services

Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals Providing healthcare information, medical information, and pharmaceutical advice online, by telephone, and through distribution of printed healthcare materials to patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; Patient advisory, support and consultancy services regarding access to medical treatment of autoimmune, autosomal dominant, autosomal recessive, cardiovascular, edema, gastrointestinal, genetic, inflammatory, lymphoproliferative, metabolic, neurological, ophthalmic, pulmonary, rare, renal, respiratory, and skin diseases and disorders Providing healthcare information, medical information, and pharmaceutical advice online, by telephone, and through distribution of printed healthcare materials to patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; Patient advisory, support and consultancy services regarding access to medical treatment of autoimmune, autosomal dominant, autosomal recessive, cardiovascular, edema, gastrointestinal, genetic, inflammatory, lymphoproliferative, metabolic, neurological, ophthalmic, pulmonary, rare, renal, respiratory, and skin diseases and disorders Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals Providing healthcare information, medical information, and pharmaceutical advice online, by telephone, and through distribution of printed healthcare materials to patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; Patient advisory, support and consultancy services regarding access to medical treatment of autoimmune, autosomal dominant, autosomal recessive, cardiovascular, edema, gastrointestinal, genetic, inflammatory, lymphoproliferative, metabolic, neurological, ophthalmic, pulmonary, rare, renal, respiratory, and skin diseases and disorders Providing healthcare information, medical information, and pharmaceutical advice online, by telephone, and through distribution of printed healthcare materials to patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; Patient advisory, support and consultancy services regarding access to medical treatment of autoimmune, autosomal dominant, autosomal recessive, cardiovascular, edema, gastrointestinal, genetic, inflammatory, lymphoproliferative, metabolic, neurological, ophthalmic, pulmonary, rare, renal, respiratory, and skin diseases and disorders

Abstract

Provided are oligomeric compounds, methods, and pharmaceutical compositions for modulating expression of SCN1A RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a developmental or epileptic encephalopathic disease, such as, for example, Dravet Syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral dysfunctions, movement and balance dysfunctions, orthopedic conditions, motor dysfunctions, cognitive impairment, delayed language and speech, visual motor integration dysfunctions, visual perception dysfunctions, executive dysfunctions, and dysautonomia.

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

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 intemucleoside linkage. In some embodiments, the disclosure provides oligomeric compounds and modified oligonucleotides comprising a central region in which purine-containing central region nucleosides are adjacent to methanesulfonyl phosphoramidate intemucleoside linkages.

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

Abstract

Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

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
• 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
• 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

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 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

Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory 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 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 Medical services; medical information services; 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

Abstract

Provided are compounds, pharmaceutical compositions, and methods of use for reducing the amount or activity of DUX4 RNA in a cell or animal, and in certain instances reducing the amount of DUX4 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a muscular dystrophy. Such symptoms and hallmarks include muscle weakness and/or muscle wasting in facio, scapula, and/or humeral muscle that can progress to the muscles of the trunk and/or lower limbs. Such muscular dystrophies include Facioscapulohumeral muscular dystrophy (FSHD).

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 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 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
• 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
• 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
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• A61P 9/00 - Drugs for disorders of the cardiovascular system

Abstract

Provided herein are compounds, pharmaceutical compositions, and methods of use for selectively reducing the amount or activity of HTT RNA comprising SNP rs7685686 in a cell or subject, and in certain instances reducing the amount of mutant HTT protein in a cell or subject. Such compounds, pharmaceutical compositions, and methods of use are useful to ameliorate at least one symptom or hallmark of Huntington's disease.

IPC Classes  ?

• 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
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

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

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 31/404 - Indoles, e.g. pindolol
• A61K 31/47 - QuinolinesIsoquinolines
• A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61M 11/00 - Sprayers or atomisers specially adapted for therapeutic purposes
• A61P 11/00 - Drugs for disorders of the respiratory system

Abstract

Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).

IPC Classes  ?

• 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

Abstract

Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

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 38/36 - Blood coagulation or fibrinolysis factors
• 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

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

IPC Classes  ?

• C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12Q 1/6811 - Selection methods for production or design of target specific oligonucleotides or binding molecules
• C12Q 1/6897 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters

Abstract

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

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 reducing the amount or activity of APP RNA in a cell or a subject, and in certain instances reducing the amount of APP protein in a cell or a subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease or disorder associated with APP. Such symptoms and hallmarks include cognitive impairment, including a decline in memory and language skills, behavioral and psychological symptoms such as apathy and lack of motivation, gait disturbances, seizures, progressive dementia, and abnormal amyloid deposits.

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 reducing the amount or activity of ATXN3 RNA in a cell or animal, and in certain embodiments reducing the amount of ATXN3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation formation, and neuron death. Such neurodegenerative diseases include spinocerebellar ataxia type 3(SCA3).

IPC Classes  ?

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

Abstract

Provided are oligomeric compounds and pharmaceutical compositions for increasing the amount or activity of UNC13A RNA in a cell or animal, and/or decreasing the amount of UNC13A RNA that includes a cryptic exon in a cell or animal, and in certain instances increasing the amount of UNC13A protein in a cell or animal. Such oligomeric compounds and pharmaceutical compositions are useful to treat neurodegenerative diseases such as amyotrophic lateral sclerosis and frontotemporal dementia.

IPC Classes  ?

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