Oligonucleotides and compositions including the same are disclosed for inhibiting or reducing patatin-like phospholipase domain-containing protein 3 (PNPLA3) gene expression. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders and/or conditions associated with PNPLA3 expression.
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
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
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
Ligand-conjugated oligonucleotide are provided herein that inhibit or reduce expression of target genes. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with an RNAi trigger induced decrease in target gene expression.
Oligonucleotides are provided herein that inhibit apolipoprotein(a) (LPA) expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with LPA expression.
Oligonucleotides are provided herein that inhibit TMPRSS6 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with hepcidin deficiency or suppression.
The present disclosure relates to oligonucleotides for selective reduction of levels of target RNA expression and/or proteins expressed in glial cells, and related methods of delivering such oligonucleotides into glial cells.
Oligonucleotide conjugates are provided herein that inhibit or reduce expression of target genes in the neurons of the central nervous system. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with expression of a neuronal target gene in the CNS.
The subject matter disclosed herein is directed to modulating STAT3 gene expression using siRNA compositions and methods directed to affecting key cell populations supporting the growth and metastasis of cancer to affect the beneficial treatment, remission or removal of the underlying tumor in a patient.
The present invention provides methods for the treatment of non-alcoholic steatohepatitis (NASH) including advanced fibrotic and/or cirrhotic stages of NASH. The methods include administration of a compound comprising a double stranded RNAi (dsRNAi) oligonucleotide, or a pharmaceutically acceptable salt thereof, to the patient. The dsRNAi oligonucleotide optionally contains an antisense strand and a sense strand, wherein the antisense strand and the sense strand form a duplex region, and wherein the antisense strand comprises a region of complementarity to a KHK mRNA target sequence.
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
dsRNAi oligonucleotides can be used in methods for the treatment of NASH, preferably the advanced fibrotic and/or cirrhotic stages thereof, using dosage regimens comprising a loading phase followed by a maintenance phase.
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
The subject matter disclosed herein is directed to modulating gene expression using siRNA compositions and methods directed to affecting key cell populations supporting the growth and metastasis of cancer to affect the beneficial treatment, remission or removal of the underlying tumor in a patient.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
Oligonucleotide conjugates are provided herein that inhibit or reduce expression of target genes in the CNS. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with target gene expression in the CNS.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
13.
COMPOSITIONS AND METHODS FOR INHIBITING PLP1 EXPRESSION
Oligonucleotides are provided herein that inhibit PLP1 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with PLP1 expression.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 47/26 - Hydrates de carbone, p. ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharidesLeurs dérivés, p. ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
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
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
Double-stranded oligonucleotides are provided herein that provide increased inhibition or reduction of expression of target genes in extra-hepatic tissue compared to hepatocytes. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with an RNAi trigger induced decrease in target gene expression.
Oligonucleotides (e.g., RNAi oligonucleotides) are provided herein that inhibit SNCA gene expression, including oligonucleotides conjugated to a targeting ligand (e.g., GalNAC moiety or lipid moiety). Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders, and/or conditions associated with SNCA gene expression.
This disclosure relates to chemically modified oligonucleotides, compositions, and methods useful for reducing ALDH2 expression, to treat alcoholism. Disclosed oligonucleotide for the reduction of ALDH2 expression is modified for enhanced pharmacological properties.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/145 - Amines, p. ex. amantadine ayant des atomes de soufre, p. ex. thiurames (N-C(S)-S-C(S)-N ou N-C(S)-S-S-C(S)-N)Sulfinylamines (-N=SO)Sulfonylamines (-N=SO2)
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
17.
COMPOSITIONS AND METHODS FOR INHIBITING MAPT EXPRESSION
Oligonucleotides are provided herein that inhibit MAPT gene expression, including oligonucleotides conjugated to a targeting ligand (e.g., lipid moiety). Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders, and/or conditions associated with MAPT gene expression.
Oligonucleotides are provided herein that inhibit TMPRSS6 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with hepcidin deficiency or suppression. 5
Provided herein are methods and compositions for treating cancer, including cancer that is not responsive to immunotherapy. In one aspect, the methods of treatment comprise administering to the subject a therapeutically effective amount of a β-catenin inhibitor and a therapeutically effective amount of an immunotherapeutic agent. Another aspect is directed to pharmaceutical compositions comprising a β-catenin inhibitor for use in treating cancer, wherein the composition is administered in combination with an immunotherapeutic agent. Yet another aspect is directed to a method of potentiating the therapeutic effect of immunotherapy against a cancer using a β-catenin inhibitor, such as a β-catenin nucleic acid inhibitor molecule.
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
A61K 47/69 - 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 le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
20.
COMPOSITIONS AND METHODS FOR MODULATING SCAP ACTIVITY
Oligonucleotides and compositions including the same are disclosed that modulate (e.g., inhibit, limit or reduce) sterol regulatory element-binding protein (SREBP) cleavage-activating protein (SCAP) activity. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders, and/or conditions associated with SCAP activity such as nonalcoholic fatty liver disease (NAFLD), (NASH), dyslipidemia, atherosclerotic cardiovascular disease (ASCVD), and/or other SCAP-associated conditions, diseases, and/or disorders.
This disclosure relates to oligonucleotides, compositions and methods useful for reducing GYS2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of GYS2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of GYS2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat glycogen storage diseases (e.g., GSDIa, GSDIII, GSDIV, GSDVI, and GSDIX) and related conditions.
The disclosure provides methods for synthesis of peracetylgalactosamine-1-pentanoic acid, also called peracetylated D-galactosamine C5 linker or GalNAc C5 linker, using a vegetal source as a starting material, such as vegetal-sourced D-glucosamine or D-glucosamine hydrochloride.Also provided are methods for purifying the peracetylgalactosamine-1-pentanoic acid, or GalNAc C5 linker, thus produced so that the end product comprises fewer impurities.
C07H 1/00 - Procédés de préparation des dérivés du sucre
C07H 13/08 - Composés contenant des radicaux saccharide estérifiés soit par l'acide carbonique ou ses dérivés, soit par des acides organiques, p. ex. acides phosphoniques par des acides carboxyliques comportant les radicaux carboxyle estérifiants liés directement à des carbocycles
26.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF METABOLIC SYNDROME
Disclosed are methods for treating, preventing and alleviating obesity, fatty liver syndrome, diabetes, liver fibrosis, NASH or other more metabolic syndrome conditions or complications comprising administering an effective amount of oligonucleotides designed to prevent, limit or modulate the expression of mRNA molecules, preferably ACC and/or 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
27.
COMPOSITIONS AND METHODS FOR INHIBITING MITOCHONDRIA AMIDOXIME REDUCING COMPONENT 1 (MARC1) EXPRESSION
Oligonucleotides are provided herein that inhibit MARC1 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with MARC1 expression.
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
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
29.
COMBINATION OF STAT3 TARGETING OLIGONUCLEOTIDES AND PD-L1 INHIBITORS
The subject matter disclosed herein is directed to modulating gene expression using siRNA compositions and methods directed to affecting key cell populations supporting the growth and metastasis of cancer to affect the beneficial treatment, remission or removal of the underlying tumor in a patient.
DECARBOXYLATIVE ACETOXYLATION USING MN(II) OR MN(III) REAGENT FOR SYNTHESIS OF 4'-ACETOXY- NUCLEOSIDE AND USE THEREOF FOR SYNTHESIS OF CORRESPONDING 4'-(DIMETHOXYPHOSPHORYL)METHOXY- NUCLEOTIDE
The present invention relates to the preparation of nucleic acids, nucleosides, nucleotides, and analogues thereof useful as potent and stable RNA interference agents. The present invention provides improved yields and avoids the use of lead tetraacetate for decarboxylative acetylation which decreases the overall cost of production by removing the need for lead remediation and offers a significant reduction in the environmental costs in production.
C07C 67/05 - Préparation d'esters d'acides carboxyliques par réaction d'acides carboxyliques ou d'anhydrides symétriques sur des liaisons carbone-carbone non saturées avec oxydation
31.
COMPOSITIONS AND METHODS FOR INHIBITING ANGPTL3 EXPRESSION
Oligonucleotides are provided herein that inhibit angiopoietin-like protein 3 (ANGPTL3) expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with ANGPTL3 expression.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
The disclosure provides oligonucleotide-ligand conjugates to facilitate the systemic delivery of oligonucleotides designed to prevent, limit or modulate the expression of mRNA molecules. The conjugates comprise nucleotides which are linked to lipid conjugate moieties or adamantyl groups.
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
33.
COMPOSITIONS AND METHODS FOR MODULATING APOC3 EXPRESSION
Oligonucleotides and compositions including the same are disclosed for inhibiting or reducing apolipoprotein C-III (APOC3) gene expression. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders and/or conditions associated with APOC3 expression.
This disclosure relates to oligonucleotides, compositions and methods useful for reducing PCSK9 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of PCSK9 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of PCSK9 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat hypercholesterolemia, atherosclerosis, and/or one or more symptoms or complications thereof.
Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.
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
36.
PROCESS FOR THE BACKBONE DEPROTECTION OF OLIGONUCLEOTIDES CONTAINING A TERMINAL ALKYL PHOSPHONATE GROUP
The invention comprises a process for the production of a linear P-linked oligonucleotide bearing an alkyl phosphonate group at the 5'-terminus of the formula (I) wherein R11-41-4-alkyl group, R21-41-4-alkoxy, X is sulfur or oxygen, the term Nucleobase stands for an optionally modified adenine, cytosine, guanine or uracil and the term oligo strand stands for the remaining P-linked oligo nucleotide strand, and wherein the oligo strand comprises at least one uracil nucleobase. The process is performed under conditions that the level of alkyl transfer impurities and the level of cyanoethyl (CNET) impurities in the linear P-linked oligonucleotide of formula I, is substantially reduced.
C07H 1/00 - Procédés de préparation des dérivés du sucre
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
37.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF A BETA-CATENIN-ASSOCIATED DISEASE OR DISORDER
Disclosed herein are methods for the treatment of cancer, comprising administering to a subject a β-catenin nucleic acid inhibitor molecule and a therapeutically effective amount of an MEK inhibitor or a c-Myc nucleic acid inhibitor molecule. Also disclosed herein is a pharmaceutical composition comprising a therapeutically effective amount of a β-catenin nucleic acid inhibitor molecule; a therapeutically effective amount of an MEK inhibitor or a c-Myc nucleic acid inhibitor molecule and at least one pharmaceutical carrier.
Oligonucleotides and compositions including the same are disclosed for inhibiting or reducing apolipoprotein C-III (APOC3) gene expression. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders and/or conditions associated with APOC3 expression.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
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
39.
COMPOSITIONS AND METHODS FOR MODULATING APOC3 EXPRESSION
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
40.
LIPID CONJUGATION FOR TARGETING OLIGODENDROCYTES OF THE CENTRAL NERVOUS SYSTEM
Oligonucleotide conjugates are provided herein that inhibit or reduce expression of target genes in the oligodendrocytes of the central nervous system. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with expression of an oligodendrocyte target gene in the CNS.
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
A61K 31/70 - Hydrates de carboneSucresLeurs dérivés
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
Lipid-conjugated oligonucleotide are provided herein that inhibit or reduce expression of target genes. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with an RNAi trigger induced decrease in target gene expression.
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
42.
LIPID CONJUGATION FOR TARGETING ASTROCYTES OF THE CENTRAL NERVOUS SYSTEM
Oligonucleotide conjugates are provided herein that inhibit or reduce expression of target genes in the astrocytes of the central nervous system. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with expression of an astrocyte target gene in the CNS.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
43.
4'-O-METHYLENE PHOSPHONATE NUCLEIC ACIDS AND ANALOGUES THEREOF
The present invention relates to nucleic acids and analogues thereof useful as potent and stable RNA interference agents.
The present invention relates to nucleic acids and analogues thereof useful as potent and stable RNA interference agents.
This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer substrate siRNA (DsiRNA) agents possessing asymmetric end structures.
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
45.
COMPOSITIONS AND METHODS FOR INHIBITING MELANOCORTIN 2 RECEPTOR AND CYTOCHROME P450 11B1 EXPRESSION
Disclosed are oligonucleotides for inhibiting or reducing expression of MC2R or CYP11B1, and lipid conjugated oligonucleotides for targeting MC2R or CYP11B1 in the adrenal gland. Also disclosed are methods for treating, preventing, and alleviating diseases associated with MC2R and/or CYP11B1 expression, such as Cushing's Disease, Cushing's Syndrome, Familial Glucocorticoid Deficiency, hereditary adrenocortical unresponsiveness to ACTH, Congenital Adrenal Hypoplasia, familial Addison's Disease, or other cortisol synthesis or signaling pathway associated conditions or complications, comprising administering an effective amount of the oligonucleotides, alone or in combination to a patient.
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
A61P 5/08 - Médicaments pour le traitement des troubles du système endocrinien des hormones de l'hypophyse antérieure, p. ex. TSH, ACTH, FSH, LH, PRL, GH pour réduire, bloquer ou contrarier l'activité des hormones de l'hypophyse antérieure
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
46.
Compositions comprising reversibly modified oligonucleotides and uses thereof
Disclosed herein are glutathione-sensitive oligonucleotides and methods of using the same. Any oligonucleotide of interest may be modified with a glutathione-sensitive moiety, including oligonucleotides used for in vivo delivery, such as nucleic acid inhibitor molecules. Typically, the glutathione-sensitive moiety is used to reversibly modify the 2′-carbon of a sugar moiety in one or more nucleotides in the oligonucleotide, although other carbon positions may also be modified with the glutathione-sensitive moiety. Also disclosed are glutathione-sensitive nucleotide and nucleoside monomers, including glutathione-sensitive nucleoside phosphoramidites that can be used, for example, in standard oligonucleotide synthesis methods. In addition, glutathione-sensitive nucleotide and nucleoside monomers without a phosphoramidite can be used therapeutically, for example, as anti-viral agents.
A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
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 15/11 - 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
47.
4′-phosphate analogs and oligonucleotides comprising the same
Disclosed herein are oligonucleotides, such as nucleic acid inhibitor molecules, having a 4′-phosphate analog and methods of using the same, for example, to modulate the expression of a target gene in a cell. The phosphate analogs are bound to the 4′-carbon of the sugar moiety (e.g., a ribose or deoxyribose or analog thereof) of the 5′-terminal nucleotide of an oligonucleotide. Typically, the phosphate analog is an oxymethylphosphonate, where the oxygen atom of the oxymethyl group is bound to the 4′-carbon of the sugar moiety or analog 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 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
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
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
C07H 19/10 - Radicaux pyrimidine avec le radical saccharide estérifié par des acides phosphoriques ou polyphosphoriques
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
48.
COMPOSITIONS AND METHODS FOR INHIBITING ΑLPHA-1 ANTITRYPSIN EXPRESSION
This invention relates to compounds, compositions, and methods useful for reducing transthyretin (TTR) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
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 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
50.
Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (MARC1) expression
Oligonucleotides are provided herein that inhibit MARC1 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with MARC1 expression.
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
This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
This invention relates to compounds, compositions, and methods useful for reducing lactate dehydrogenase target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Oligonucleotides are provided herein that inhibit MARC1 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with MARC1 expression.
Oligonucleotides and compositions including the same are disclosed for inhibiting or reducing patatin-like phospholipase domain-containing protein 3 (PNPLA3) gene expression. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders and/or conditions associated with PNPLA3 expression.
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
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
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
57.
LIPID CONJUGATION FOR TARGETING NEURONS OF THE CENTRAL NERVOUS SYSTEM
Oligonucleotide conjugates are provided herein that inhibit or reduce expression of target genes in the neurons of the central nervous system. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with expression of a neuronal target gene in the CNS.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C07H 19/00 - Composés contenant un hétérocycle partageant un hétéro-atome du cycle avec un radical saccharideNucléosidesMononucléotidesLeurs anhydro-dérivés
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
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 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
58.
COMPOSITIONS AND METHODS FOR INHIBITING NUCLEAR RECEPTOR SUBFAMILY 1 GROUP H MEMBER 3 (NR1H3) EXPRESSION
Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.
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
59.
Compositions and methods for inhibiting nuclear receptor subfamily 1 group H member 3 (NR1H3) expression
Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.
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
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
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
61.
COMPOSITIONS AND METHODS FOR MODULATING PNPLA3 EXPRESSION
Oligonucleotides and compositions including the same are disclosed for inhibiting or reducing patatin-like phospholipase domain-containing protein 3 (PNPLA3) gene expression. Methods of making and using the oligonucleotides also are disclosed, particularly uses relating to treating diseases, disorders and/or conditions associated with PNPLA3 expression.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
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
62.
COMPOSITIONS AND METHODS FOR MODULATING PNPLA3 EXPRESSION
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
63.
Extended dicer substrate agents and methods for the specific inhibition of gene expression
The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.
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
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
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
64.
Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
The present invention relates to methods for synthesizing compounds useful as potent and stable RNA interference agents, derivatives thereof, and intermediates thereto.
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 19/167 - Radicaux purine avec un ribosyle comme radical saccharide
C07H 19/067 - Radicaux pyrimidine avec un ribosyle comme radical saccharide
C07H 1/00 - Procédés de préparation des dérivés du sucre
Formulations comprising anionic agents such as nucleic acids within a lipid-containing particle methods of formulating a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for preparing a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for therapeutic delivery of an anionic agent to a patient in need thereof, where the anionic agent is formulated in a lipid-containing particle as described herein.
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 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
A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
The subject matter disclosed herein is directed to modulating gene expression using siRNA compositions and methods directed to affecting key cell populations supporting the growth and metastasis of cancer to affect the beneficial treatment, remission or removal of the underlying tumor in a patient.
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 31/711 - Acides désoxyribonucléiques naturels, c.-à-d. contenant uniquement des 2'-désoxyriboses liés à l'adénine, la guanine, la cytosine ou la thymine et ayant des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
The subject matter disclosed herein is directed to modulating gene expression using siRNA compositions and methods directed to affecting key cell populations supporting the growth and metastasis of cancer to affect the beneficial treatment, remission or removal of the underlying tumor in a patient.
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61K 31/711 - Acides désoxyribonucléiques naturels, c.-à-d. contenant uniquement des 2'-désoxyriboses liés à l'adénine, la guanine, la cytosine ou la thymine et ayant des liaisons 3'-5' phosphodiester
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
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
C12N 15/12 - Gènes codant pour des protéines animales
69.
Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Oligonucleotide conjugates are provided herein that inhibit or reduce expression of target genes in the CNS. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with target gene expression in the CNS.
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
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
71.
Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Provided herein are double-stranded nucleic acid inhibitor molecules having a shortened sense strand with a stem loop structure and an antisense strand. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.
This disclosure relates to the use of RNA oligonucleotides, compositions and methods useful for reducing ALDH2 or other target gene expression, in the central nervous system. In some embodiments, the oligonucleotide is used in methods of treating neurological diseases. Stable oligonucleotide derivatives that have enhanced activity in the central nervous system are provided.
The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.
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
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
75.
CHEMICAL MODIFICATIONS FOR INHIBITING EXPRESSION OF ALDH2
This disclosure relates to chemically modified oligonucleotides, compositions, and methods useful for reducing ALDH2 expression, to treat alcoholism. Disclosed oligonucleotide for the reduction of ALDH2 expression is modified for enhanced pharmacological properties.
Provided herein are methods of treating a KRAS-associated cancer in a subject, comprising administering to the subject a therapeutically-effective amount of a KRAS nucleic acid inhibitor molecule and a therapeutically-effective amount of an MEK inhibitor or an immunotherapeutic agent. Also disclosed herein is a method of potentiating a therapeutic effect of an immunotherapeutic agent against a KRAS-associated cancer, comprising administering to a subject having the KRAS-associated cancer a KRAS nucleic acid inhibitor molecule in an amount sufficient to potentiate the therapeutic effect of the immunotherapeutic agent against the cancer.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/519 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime condensées en ortho ou en péri avec des hétérocycles
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
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
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
This disclosure relates to chemically modified oligonucleotides, compositions, and methods useful for reducing ALDH2 expression, to treat alcoholism. Disclosed oligonucleotide for the reduction of ALDH2 expression is modified for enhanced pharmacological properties.
This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
This invention relates to compounds, compositions, and methods useful for reducing α-1 antitrypsin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
The present disclosure relates to oligonucleotides for selective reduction of levels of target RNA expression and/or proteins expressed in glial cells, and related methods of delivering such oligonucleotides into glial cells.
The present disclosure relates to oligonucleotides for selective reduction of levels of target RNA expression and/or proteins expressed in glial cells, and related methods of delivering such oligonucleotides into glial cells.
This disclosure relates to oligonucleotides, compositions and methods useful for reducing HMGB1 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of HMGB1 expression may be either double-stranded or single-stranded and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of HMGB1 expression may also be designed to include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat liver fibrosis and related conditions.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
83.
DOUBLE-STRANDED NUCLEIC ACID INHIBITOR MOLECULES CONTAINING A TRILOOP
Provided herein are double-stranded nucleic acid inhibitor molecules having a sense strand with a stem loop structure and an antisense strand, where the loop portion of the stem loop structure is a triloop. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
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
The disclosure provides oligonucleotide-ligand conjugates to facilitate the systemic delivery of oligonucleotides designed to prevent, limit or modulate the expression of mRNA molecules. The conjugates comprise nucleotides which are linked to lipid conjugate moieties or adamantyl groups.
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
The disclosure provides oligonucleotide-ligand conjugates to facilitate the systemic delivery of oligonucleotides designed to prevent, limit or modulate the expression of mRNA molecules. The conjugates comprise nucleotides which are linked to lipid conjugate moieties or adamantyl groups.
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
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
Disclosed are methods for treating, preventing and alleviating obesity, fatty liver syndrome, diabetes, liver fibrosis, NASH or other more metabolic syndrome conditions or complications comprising administering an effective amount of oligonucleotides designed to prevent, limit or modulate the expression of mRNA molecules, preferably ACC and/or DGAT2.
Oligonucleotides are provided herein that inhibit apolipoprotein(a) (LPA) expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with LPA expression.
Oligonucleotides are provided herein that inhibit PLP1 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with PLP1 expression.
The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.
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
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
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
94.
Extended dicer substrate agents and methods for the specific inhibition of gene expression
The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.
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
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
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
95.
Compositions and methods for inhibiting ALDH2 expression
This disclosure relates to oligonucleotides, compositions and methods useful for reducing ALDH2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of ALDH2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of ALDH2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat alcoholism and related conditions.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Oligonucleotide compounds for use in targeted pharmaceutical
therapies for the treatment of rare diseases, genetic
disorders, cardiovascular diseases, and viral infections;
oligonucleotide delivery agents for use in targeted
pharmaceutical therapies for the treatment of rare diseases,
genetic disorders, cardiovascular diseases, and viral
infections; oligonucleotide compounds for use in the
targeted delivery of RNAi therapy; oligonucleotide delivery
agents consisting of compounds that facilitate the delivery
of a wide range of pharmaceutical and therapeutic agents
intravenously or subcutaneously for use in targeted
pharmaceutical therapies for a wide range of medical
purposes; drug delivery agents consisting of oligonucleotide
compounds that facilitate the delivery of a wide range of
pharmaceutical based therapies. Research and development of oligonucleotide compounds for
use in targeted pharmaceutical therapies for the treatment
of rare diseases, genetic disorders, cardiovascular
diseases, and viral infections; research and development of
pharmaceutical preparations for the treatment of disorders
outside the liver; research and development of
oligonucleotide compounds for targeted gene silencing;
research and development of oligonucleotide drug delivery
platforms for use in the treatment of rare diseases, genetic
disorders, cardiovascular diseases, and viral infections.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Biological preparations for use in targeted pharmaceutical
therapies for the treatment of rare diseases, genetic
disorders, chronic liver diseases, cardiovascular diseases
and viral infections; biological compound in the form of a
molecular complex that acts as a platform for the delivery
of RNA-based drugs to biological cells; drug delivery agents
consisting of oligonucleotide compounds that facilitate the
delivery of a wide range of pharmaceutical based therapies;
oligonucleotide compounds for use in the targeted delivery
of RNAi therapy; oligonucleotide delivery agents consisting
of compounds that facilitate the delivery of a wide range of
pharmaceutical and therapeutic agents intravenously or
subcutaneously for use in targeted pharmaceutical therapies
for a wide range of medical purposes. Custom manufacture of oligonucleotides. Research and development of pharmaceutical products;
research and development of oligonucleotide drug delivery
platforms for use in the treatment of rare diseases, genetic
disorders, chronic liver diseases, cardiovascular diseases,
and viral infections.
98.
COMPOSITIONS AND METHODS FOR INHIBITING ANGPTL3 EXPRESSION
Oligonucleotides are provided herein that inhibit angiopoietin-like protein 3 (ANGPTL3) expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with ANGPTL3 expression.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Biologic preparations for use in targeted pharmaceutical
therapies for the treatment of rare diseases, genetic
disorders, chronic liver diseases, cardiovascular diseases
and viral infections; biological compound in the form of a
molecular complex that acts as a platform for the delivery
of RNA-based drugs to biological cells; drug delivery agents
consisting of oligonucleotide compounds that facilitate the
delivery of a wide range of pharmaceutical based therapies;
oligonucleotide compounds for use in the targeted delivery
of RNAi therapy; oligonucleotide delivery agents consisting
of compounds that facilitate the delivery of a wide range of
pharmaceutical and therapeutic agents intravenously or
subcutaneously for use in targeted pharmaceutical therapies
for a wide range of medical purposes. Custom manufacture of oligonucleotides. Research and development of pharmaceutical products;
research and development of oligonucleotide drug delivery
platforms for use in the treatment of rare diseases, genetic
disorders, chronic liver diseases, cardiovascular diseases,
and viral infections.
100.
Extended dicer substrate agents and methods for the specific inhibition of gene expression
The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.
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 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
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
