Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GYS1 RNA in a cell or subject, and in certain instances reducing the amount of GYS1 protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a glycogen storage disease. Such glycogen storage diseases include Lafora disease, adult polyglucosan body disease (APBD), Andersen's disease, and Pompe disease.
C12N 15/115 - Aptamères, c.-à-d. acides nucléiques liant spécifiquement une molécule cible avec une haute affinité sans s'y hybrider
A61K 47/46 - Ingrédients de constitution indéterminée ou leurs produits de réaction, p. ex. peau, os, lait, fibre de coton, coquille d’œuf, fiel de bœuf ou extraits de plante
Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
05 - Produits pharmaceutiques, vétérinaires et hygièniques
35 - Publicité; Affaires commerciales
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical preparations and substances for the treatment
of edema, namely hereditary angioedema; pharmaceuticals for
the treatment of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders. Collecting data and information on autosomal dominant,
autosomal recessive, genetic, edema, pulmonary, respiratory,
ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders for
treatment and diagnostic purposes. Medical services; medical information services; providing
medical and healthcare information for healthcare
professionals, caregivers, patients, and providers in the
fields of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders via a website; medical consulting services in the
field of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders; healthcare services, namely, providing medical
and healthcare information in the field of autosomal
dominant, autosomal recessive, genetic, edema, pulmonary,
respiratory, ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders via an
online database; providing medical and healthcare
information concerning the screening, diagnosis, and
treatment of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders.
4.
Linkage Modified Oligomeric Compounds and Uses Thereof
The present disclosure provides oligomeric compounds (including oligomeric compounds that are antisense agents or portions thereof) comprising a modified oligonucleotide having at least one modified internucleoside linking group.
Provided are RNAi agents, methods, and pharmaceutical compositions for reducing the amount or activity of tau RNA in a cell or animal, and in certain instances reducing the amount of tau protein in a cell or animal. Such RNAi agents, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease, including a tauopathy, Alzheimer's disease, fronto-temporal dementia (FTD), FTDP-17, progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), corticobasal ganglionic degeneration (CBD), epilepsy, or Dravet's Syndrome.
Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) or Lp(a). Certain diseases, disorders or conditions related to apo(a) or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The antisense compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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.
Provided are oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of Protocadherin 19 (PCDH19) RNA in a cell or subject, and in certain instances reducing the amount of PCDH19 protein in a cell or subject. Such oligomeric agents, oligomeric compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodevelopmental disease or disorder. Such neurodevelopmental diseases or disorders include PCDH19 Epilepsy. Such symptoms or hallmarks include seizures, cognitive impairment, intellectual disabilities, autism spectrum disorder, behavioral problems, aggression, anxiety, obsessive-compulsive disorder, hyperactivity, attention deficit disorder (ADD), and attention deficit hyperactivity disorder (ADHD).
The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain embodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07H 19/20 - Radicaux purine avec le radical saccharide estérifié par des acides phosphoriques ou polyphosphoriques
C07H 21/00 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
11.
Compounds and Methods for Reducing KCNT1 Expression
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of KCNT1 RNA in a cell or subject, and in certain instances reducing the amount of KCNT1 protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurological condition. Such symptoms and hallmarks include seizures, encephalopathy, and behavioral abnormalities. Non-limiting examples of neurological conditions that benefit from these compounds, methods, and pharmaceutical compositions are epilepsy of infancy with migrating focal seizures (EIMFS), autosomal dominant nocturnal frontal lobe epilepsy (ADNFLE), West syndrome, and Ohtahara syndrome.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
35 - Publicité; Affaires commerciales
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical preparations and substances for the treatment
of edema, namely hereditary angioedema; pharmaceuticals for
the treatment of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders. Collecting data and information on autosomal dominant,
autosomal recessive, genetic, edema, pulmonary, respiratory,
ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders for
treatment and diagnostic purposes. Medical services; medical information services; providing
medical and healthcare information for healthcare
professionals, caregivers, patients, and providers in the
fields of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders via a web site; medical consulting services in the
field of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders; healthcare services, namely, providing medical
and healthcare information in the field of autosomal
dominant, autosomal recessive, genetic, edema, pulmonary,
respiratory, ophthalmic, gastrointestinal, skin, autoimmune
lymphoproliferative, and rare diseases and disorders via an
online database; providing medical and healthcare
information concerning the screening, diagnosis, and
treatment of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
35 - Publicité; Affaires commerciales
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical preparations and substances for the treatment
of edema, namely hereditary angioedema; pharmaceuticals in
the fields of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders. Collecting data and information on autosomal dominant,
autosomal recessive, genetic, edema, pulmonary, respiratory,
ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders for
treatment and diagnostic purposes. Medical services; medical information services; collecting
data and information on autosomal dominant, autosomal
recessive, genetic, edema, pulmonary, respiratory,
ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders for
treatment and diagnostic purposes; medical consulting
services in the field of autosomal dominant, autosomal
recessive, genetic, edema, pulmonary, respiratory,
ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders;
providing medical and healthcare information for healthcare
professionals, caregivers, patients, and providers in the
fields of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders via a web site; providing medical and healthcare
information concerning the screening, diagnosis, and
treatment of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceuticals for treatment of genetic diseases and
disorders; pharmaceuticals for treatment of cardiovascular,
metabolic, neurological, and inflammatory diseases and
disorders. Medical services; medical information services; providing
medical and healthcare information via a website for
healthcare professionals, caregivers, patients, and
providers in the field of genetic diseases and disorders;
medical consulting services in the field of genetic diseases
and disorders; healthcare services, namely, providing
medical and healthcare information in the field of genetic
diseases and disorders, via an online database that collects
data and information on genetic diseases and disorders for
treatment and diagnostic purposes; providing medical and
healthcare information concerning the screening, diagnosis,
and treatment of genetic diseases and disorders; providing
medical and healthcare information via a website for
healthcare professionals, caregivers, patients, and
providers in the field of cardiovascular, metabolic,
neurological and inflammatory diseases and disorders;
medical consulting services in the field of cardiovascular,
metabolic, neurological and inflammatory diseases and
disorders; healthcare services, namely, providing medical
and healthcare information in the field of cardiovascular,
metabolic, neurological and inflammatory diseases and
disorders, via an online database that collects data and
information on cardiovascular, metabolic, neurological and
inflammatory diseases and disorders for treatment and
diagnostic purposes; providing medical and healthcare
information concerning the screening, diagnosis, and
treatment of cardiovascular, metabolic, neurological and
inflammatory diseases and disorders.
15.
Compounds and Methods for Reducing LRRK2 Expression
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.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 47/46 - Ingrédients de constitution indéterminée ou leurs produits de réaction, p. ex. peau, os, lait, fibre de coton, coquille d’œuf, fiel de bœuf ou extraits de plante
Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
Provided herein are methods for decreasing Ataxin-2 mRNA expression. Such methods are useful to ameliorate symptoms of Ataxin-2 associated diseases. Such Ataxin-2 associated diseases include amyotrophic lateral sclerosis (ALS). Such symptoms include loss of motor function, reduced CMAP amplitude, denervation, and loss of motor neurons.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN1 RNA in a cell or subject, and in certain instances reducing the amount of ATXN1 in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include gait and limb ataxia, cognitive impairments, difficulty with speaking and swallowing, atrophy of the cerebellum and brainstem in magnetic resonance imaging (MRI), neurochemical abnormalities in the cerebellum and brainstem detected via magnetic resonance spectroscopy (MRS), and death within 10-15 years of symptom onset. Such neurodegenerative diseases include Spinocerebellar ataxia type 1.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.
The present embodiments provide methods, compounds, and compositions useful for inhibiting DGAT2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DGAT2.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques
C07H 21/00 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques
05 - Produits pharmaceutiques, vétérinaires et hygièniques
35 - Publicité; Affaires commerciales
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical preparations and substances for the treatment
of edema, namely hereditary angioedema; pharmaceuticals in
the fields of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders. Collecting data and information on autosomal dominant,
autosomal recessive, genetic, edema, pulmonary, respiratory,
ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders for
treatment and diagnostic purposes. Medical services; medical information services; providing
medical and healthcare information for healthcare
professionals, caregivers, patients, and providers in the
fields of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders via a website; medical consulting services in the
field of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders; healthcare services, namely, providing medical
and healthcare information in the field of autosomal
dominant, autosomal recessive, genetic, edema, pulmonary,
respiratory, ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders via an
online database; providing medical and healthcare
information concerning the screening, diagnosis, and
treatment of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
35 - Publicité; Affaires commerciales
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical preparations and substances for the treatment
of edema, namely hereditary angioedema; pharmaceuticals in
the fields of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders. Collecting data and information on autosomal dominant,
autosomal recessive, genetic, edema, pulmonary, respiratory,
ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders for
treatment and diagnostic purposes. Medical services; medical information services; providing
medical and healthcare information for healthcare
professionals, caregivers, patients, and providers in the
fields of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders via a website; medical consulting services in the
field of autosomal dominant, autosomal recessive, genetic,
edema, pulmonary, respiratory, ophthalmic, gastrointestinal,
skin, autoimmune, lymphoproliferative, and rare diseases and
disorders; healthcare services, namely, providing medical
and healthcare information in the field of autosomal
dominant, autosomal recessive, genetic, edema, pulmonary,
respiratory, ophthalmic, gastrointestinal, skin, autoimmune,
lymphoproliferative, and rare diseases and disorders via an
online database; providing medical and healthcare
information concerning the screening, diagnosis, and
treatment of autosomal dominant, autosomal recessive,
genetic, edema, pulmonary, respiratory, ophthalmic,
gastrointestinal, skin, autoimmune, lymphoproliferative, and
rare diseases and disorders.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
(1) Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, alexander disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; pharmaceutical preparations containing zilganersen. (1) Providing health information; providing healthcare information; medical information services provided via the Internet.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
(1) Pharmaceutical preparations and substances in the field of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; pharmaceutical preparations and substances in the field of glial fibrillary acidic protein-associated diseases and disorders, alexander disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; pharmaceutical preparations and substances in the field of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; pharmaceutical preparations containing zilganersen. (1) Providing health information; providing healthcare information; medical information services provided via the Internet.
The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount, activity, or expression of the target nucleic acid in a cell. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SCN2A RNA in a cell or subject, and in certain instances reducing the amount of SCN2A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a disease or disorder associated with a voltage-gated sodium channel protein, such as, for example, a Developmental and Epileptic Encephalopathy, an intellectual disability, or an autism spectrum disorder. Such symptoms and hallmarks include, but are not limited to seizures, hypotonia, sensory integration disorders, motor development delays and dysfunctions, intellectual and cognitive dysfunctions, movement and balance dysfunctions, visual dysfunctions, delayed language and speech, gastrointestinal disorders, neurodevelopmental delays, sleep problems, and sudden unexpected death in epilepsy.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceuticals for treatment of genetic diseases and
disorders; pharmaceuticals for treatment of cardiovascular,
metabolic, neurological, and inflammatory diseases and
disorders. Medical services; medical information services; providing
medical and healthcare information via a website for
healthcare professionals, caregivers, patients, and
providers in the field of genetic diseases and disorders;
medical consulting services in the field of genetic diseases
and disorders; healthcare services, namely, providing
medical and healthcare information in the field of genetic
diseases and disorders, via an online database that collects
data and information on genetic diseases and disorders for
treatment and diagnostic purposes; providing medical and
healthcare information concerning the screening, diagnosis,
and treatment of genetic diseases and disorders; providing
medical and healthcare information via a website for
healthcare professionals, caregivers, patients, and
providers in the field of cardiovascular, metabolic,
neurological and inflammatory diseases and disorders;
medical consulting services in the field of cardiovascular,
metabolic, neurological and inflammatory diseases and
disorders; healthcare services, namely, providing medical
and healthcare information in the field of cardiovascular,
metabolic, neurological and inflammatory diseases and
disorders, via an online database that collects data and
information on cardiovascular, metabolic, neurological and
inflammatory diseases and disorders for treatment and
diagnostic purposes; providing medical and healthcare
information concerning the screening, diagnosis, and
treatment of cardiovascular, metabolic, neurological and
inflammatory diseases and disorders.
The present invention provides gapped oligomeric compounds comprising from 1 to about 3 internucleoside linkages having one of formulas I to XVI. In certain embodiments, inclusion of from 1 to about 3 internucleoside linkages of one of formulas I to XVI, improves selectivity for a target RNA relative to an off target RNA. In certain embodiments, the improved selectivity also provides an improved toxicity profile. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount of activity or expression of the target nucleic acid in a cell.
36 - Services financiers, assurances et affaires immobilières
Produits et services
Financial and insurance consultation services, namely, patient advisory, support and consultancy services regarding patient finances, insurance and financing of medical treatment; providing financial and insurance information, namely, providing information and guidance in connection with health insurance benefit plans and other patient financial assistance resources; financial and insurance consultation services, namely, insurance and medical and pharmaceutical access consultation services, namely, assisting patients, their caregivers, and healthcare providers to identify the scope of coverage offered by patient medical and pharmaceutical insurance and to identify other financial assistance resources for payment of patient treatment; insurance consultation services, namely, assisting patients, their caregivers, and healthcare providers to obtain authorization and payment for medical and pharmaceutical costs; Providing pharmaceutical information regarding patient reimbursement for pharmaceuticals
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Patient advisory, support and consultancy services regarding access to medical treatment of hereditary angioedema; providing healthcare information, medical information, and pharmaceutical advice online, by telephone, and through distribution of printed healthcare materials to healthcare providers, patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to and reimbursement for pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; providing a website featuring medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the field of hereditary angioedema and edema diseases and disorders, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; medical consulting services in the field of hereditary angioedema and edema diseases and disorders, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of hereditary angioedema and edema diseases and disorders, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders, and collecting data and information on hereditary angioedema, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and collecting data and information on rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information
41 - Éducation, divertissements, activités sportives et culturelles
Produits et services
Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of hereditary angioedema; education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; online journals, namely, blogs featuring information on hereditary angioedema; online journals, namely, blogs featuring information on autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders; online journals, namely, blogs featuring information on hereditary angioedema and edema diseases and disorders; providing a website featuring blogs and non-downloadable publications in the nature of articles, blogs, brochures, and guides in the fields of hereditary angioedema, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders
34.
Compounds and Methods for Reducing APOCIII Expression
Provided are oligomeric agents, methods, and pharmaceutical compositions for reducing the amount or activity of APOC3 RNA in a cell or animal, and in certain instances reducing the amount of ApoCIII protein in a cell or animal. Such oligomeric agents, methods, and pharmaceutical compositions are useful to treat or manage hypertriglyceridemia and/or cardiovascular disease (CVD).
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
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).
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.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
38.
Methods for Separating Certain Oligonucleotide Compounds
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.
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
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.
A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
C07K 14/195 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de bactéries
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/52 - Gènes codant pour des enzymes ou des proenzymes
C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
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.
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.
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
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.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
45.
COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
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.
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 47/60 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique macromoléculaire, p. ex. une molécule oligomérique, polymérique ou dendrimérique obtenu par des réactions autres que celles faisant intervenir uniquement des liaisons non saturées carbone-carbone, p. ex. polyurées ou polyuréthanes le composé organique macromoléculaire étant un oligomère, un polymère ou un dendrimère de polyoxyalkylène, p. ex. PEG, PPG, PEO ou polyglycérol
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
47.
COMPOUNDS AND METHODS FOR MODULATING ACUTE ACTIVATION
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.
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
45 - Services juridiques; services de sécurité; services personnels pour individus
Produits et services
Organizing and conducting support groups in the field of healthcare; Providing emotional counseling and emotional support services for individuals in the field of healthcare
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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
51.
LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
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.
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).
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes
53.
METHODS AND COMPOSITIONS FOR REDUCING ANGIOTENSINOGEN
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.
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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.
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.
ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE (USA)
IONIS PHARMACEUTICALS INC. (USA)
Inventeur(s)
Hasting, Michelle L.
Centa, Jessica
Stratton, Matthew
Jafar-Nejad, Paymaan
Hunyara, John
Abrégé
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.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
05 - Produits pharmaceutiques, vétérinaires et hygièniques
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
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.
C07K 14/00 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
A61K 38/00 - Préparations médicinales contenant des peptides
C07K 9/00 - Peptides ayant jusqu'à 20 amino-acides, contenant des radicaux saccharide et comportant une séquence entièrement déterminéeLeurs dérivés
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
(1) Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory diseases and disorders. (1) Medical services; medical information services; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of genetic diseases and disorders; medical consulting services in the field of genetic diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of genetic diseases and disorders, via an online database that collects data and information on genetic diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of genetic diseases and disorders; providing medical and healthcare information via a website for healthcare professionals, caregivers, patients, and providers in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; medical consulting services in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of cardiovascular, metabolic, neurological and inflammatory diseases and disorders, via an online database that collects data and information on cardiovascular, metabolic, neurological and inflammatory diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of cardiovascular, metabolic, neurological and inflammatory diseases and disorders.
60.
COMPOUNDS AND METHODS FOR MODULATING PROGRANULIN EXPRESSION
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.
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.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
C07H 21/00 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/67 - Méthodes générales pour favoriser l'expression
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations and substances for the treatment of edema, namely, hereditary angioedema; pharmaceutical products and preparations in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations and substances for the treatment of edema, namely, hereditary angioedema; pharmaceutical products and preparations in the fields of autosomal dominant, autosomal recessive, genetic, edema, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, and rare diseases and disorders
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
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.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
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.
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.
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).
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.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical preparations and substances for the treatment of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances for the treatment of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances for the treatment of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical preparations and substances for the treatment of neurodevelopmental and neurodegenerative diseases and disorders, leukodystrophy, demyelinating diseases and disorders, and hypomyelinating diseases and disorders; Pharmaceutical preparations and substances for the treatment of glial fibrillary acidic protein-associated diseases and disorders, Alexander Disease, seizures, ataxia, motor function disorders, cognitive function disorders, dysphagia, and swallowing disorders; Pharmaceutical preparations and substances for the treatment of autosomal dominant, autosomal recessive, genetic, neurologic, parasympathetic system, neuromuscular system, skeletomuscular system, central nervous system, epileptic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, metabolic, autoimmune and lymphoproliferative diseases and disorders; Pharmaceutical preparations containing zilganersen. Providing health information; providing healthcare information; medical information services provided via the Internet
75.
COMPOUNDS AND METHODS FOR MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION
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.
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.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
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.
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
79.
COMPOUNDS AND METHODS FOR MODULATING GLYCOGEN SYNTHASE 1
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.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
80.
COMPOUNDS AND METHODS FOR REDUCING TUBB4A EXPRESSION
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.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61P 25/14 - Médicaments pour le traitement des troubles du système nerveux pour traiter les mouvements anormaux, p. ex. chorée, dyskinésie
36 - Services financiers, assurances et affaires immobilières
Produits et 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
41 - Éducation, divertissements, activités sportives et culturelles
Produits et services
Education and medical training services, namely, providing seminars, non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; online journals, namely, blogs featuring information on hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; providing a website featuring blogs and non-downloadable publications in the nature of articles, blogs, brochures, and guides in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Providing healthcare information, medical information, and pharmaceutical information online, by telephone, and through distribution of printed healthcare materials to healthcare providers, patients, physicians, medical office staff, pharmaceutical prescribers, caregivers, and pharmacies regarding patient access to and reimbursement for pharmaceuticals and safe and appropriate use and distribution of pharmaceuticals; providing healthcare information; medical information services; providing a website featuring medical information in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; medical consulting services in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders; healthcare services, namely, providing an online database comprised of medical and healthcare information in the fields of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune lymphoproliferative, asthma, and rare diseases and disorders, and collecting data and information on hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders for treatment and diagnostic purposes; providing medical and healthcare information concerning the screening, diagnosis, and treatment of hereditary angioedema, edema, autosomal dominant, autosomal recessive, genetic, pulmonary, respiratory, ophthalmic, gastrointestinal, skin, autoimmune, lymphoproliferative, asthma, and rare diseases and disorders
84.
METHODS AND COMPOSITIONS FOR INHIBITING PMP22 EXPRESSION
The present embodiments provide methods, compounds, and compositions useful for inhibiting PMP22 expression and for treating, preventing, or ameliorating a disease associated with PMP22.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61P 25/02 - Médicaments pour le traitement des troubles du système nerveux des neuropathies périphériques
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
85.
COMPOSITIONS AND METHODS TO TREAT LIVER, KIDNEY AND CELL PROLIFERATIVE DISEASES AND DISORDERS
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.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques
A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins
A61P 9/00 - Médicaments pour le traitement des troubles du système cardiovasculaire
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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.
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.
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
A61K 47/65 - Séquences de liaison, liants ou bras-espaceurs peptidiques, p. ex. séquences de liaison peptidiques vulnérable aux protéases
A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p. ex. un fragment Fc
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
C07K 16/28 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/115 - Aptamères, c.-à-d. acides nucléiques liant spécifiquement une molécule cible avec une haute affinité sans s'y hybrider
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceuticals for treatment of genetic diseases and disorders; pharmaceuticals for treatment of cardiovascular, metabolic, neurological, and inflammatory diseases and disorders
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et 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
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et 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
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
41 - Éducation, divertissements, activités sportives et culturelles
Produits et 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
41 - Éducation, divertissements, activités sportives et culturelles
Produits et 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
36 - Services financiers, assurances et affaires immobilières
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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, cardiovascular, metabolic, neurological, and inflammatory diseases and disorders, and blood disorders
36 - Services financiers, assurances et affaires immobilières
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
36 - Services financiers, assurances et affaires immobilières
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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, cardiovascular, metabolic, neurological, and inflammatory diseases and disorders, and blood disorders
36 - Services financiers, assurances et affaires immobilières
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
