The present disclosure includes cationic carrier units comprising (i) a water-soluble polymer, (ii) a positively charged carrier, and (iii) a hydrophobic moiety, wherein the cationic carrier unit does not comprise a crosslinking moiety and wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core.
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
The present disclosure includes an oligonucleotide comprising a miR-378a-3p inhibitor. The disclosure also includes pharmaceutical compositions comprising the oligonucleotide, and also methods of treating diseases or conditions (i.e., liver diseases or conditions) in a subject in need thereof comprising administering the oligonucleotide to the subject.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
The present disclosure includes the use of a miR-485 inhibitor for treating a disease or disorder associated with the increased level of miR-485-3p. In some aspects, the miR-485 inhibitor is capable of reducing the expression of a gene and/or protein associated with miR-485-3p.
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure includes a miR-485 inhibitor for treating a disease or disorder associated with the increased level of miR-485-3p. In some aspects, the miR-485 inhibitor is capable of reducing the expression of a gene and/or protein associated with miR-485-3p.
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61P 17/14 - Drugs for dermatological disorders for baldness or alopecia
5.
MICROTUBULE-ASSOCIATED PROTEIN TAU TARGETING SIRNAS AND USES THEREOF
The present disclosure relates to siRNA, which target MAPT transcript in a cell, leading to reduced expression of MAPT protein. Reduction of MAPT protein expression is beneficial for the treatment of certain medical disorders, e.g., a tauopathy.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
SK BIOPHARMACEUTICALS CO., LTD. (Republic of Korea)
Inventor
Ryu, Jin-Hyeob
Jang, Ha Na
Lim, Jongyeob
Jung, Yeon-Joo
Park, Young Jin
Kim, Insun
Lim, Yu Na
Kang, Howon
Joung, Chanmi
Choi, Eunju
Park, Ah Hyung
Yook, Chaehyun
Cho, Seokhwan
Shin, Yujin
Gil, So-Young
Abstract
The present disclosure includes the use of a miR-484 inhibitor for treating a disease or disorder associated with the increased level of miR-484. In some aspects, the miR-484 inhibitor is capable of reducing the expression of a gene and/or protein associated with miR-484.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
The present disclosure relates to siRNA, which target SNCA transcript in a cell, leading to reduced expression of SNCA protein. Reduction of SNCA protein expression is beneficial for the treatment of certain medical disorders, e.g., a neurological disorder.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61K 39/00 - Medicinal preparations containing antigens or antibodies
8.
TREATING MITOCHONDRIAL DYSFUNCTION WITH MIRNA-485 INHIBITOR
The present disclosure relates to the use of a miR-485 inhibitor to alleviate and/or prevent a mitochondrial function. In some aspects, a miR-485 inhibitor described herein can be used to regulate mitophagy, mitochondrial biogenesis, mitochondrial fission, and/or mitochondrial fusion. The present disclosure also relates to the use of a miR-485 inhibitor to treat a mitochondrial disorder.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
The present disclosure relates to antisense oligonucleotides, which target IDO1 mRNA in a cell, leading to reduced expression of IDO1 protein. Reduction of IDO1 protein expression is beneficial for the treatment of certain medical disorders, e.g., a cancer or a neuronal disease.
The present disclosure relates to small interfering RNAs, which target IDO1 mRNA in a cell, leading to reduced expression of IDO1 protein. Reduction of IDO1 protein expression is beneficial for the treatment of certain medical disorders, e.g., a cancer or a neuronal disease.
The present disclosure includes cationic carrier units comprising (i) a water-soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure relates to oligonucleotides conjugated to a probe or a quencher, and the use of such oligonucleotides to measure the level of a miRNA present in a subject. In some aspects, the miRNA is associated with a disease or disorder. Also disclosed herein are methods of treating a subject identified as having abnormal level of a miRNA.
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
C12Q 1/6818 - Hybridisation assays characterised by the detection means involving interaction of two or more labels, e.g. resonant energy transfer
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 39/00 - Medicinal preparations containing antigens or antibodies
13.
USE OF MIR-485 INHIBITORS TO TREAT AGE-ASSOCIATED DISEASES OR CONDITIONS
The present disclosure includes the use of a miRNA inhibitor for extending the length and/or protecting against a reduction in the length of a telomere in a cell. In some aspects, such miRNA inhibitors can be used in treating an age-associated disease or condition.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 25/00 - Drugs for disorders of the nervous system
14.
USE OF MIRNA-485 INHIBITORS FOR TREATING INFLAMMASOME-RELATED DISEASES OR DISORDERS
The present disclosure includes the use of a miR-485 inhibitor for treating a disease or disorder associated with abnormal (e.g., increased) inflammasome activity (e.g., cardiac disease, autoimmune disease, kidney disease, or neurological disease). In some aspects, the miR-485 inhibitor is capable of reducing the expression of a gene and/or protein associated with inflammasomes.
e.g.e.g., SARS-CoV-2) spike protein. In some aspects, the nucleotide sequence is codon-optimized. Also disclosed herein are cells, vectors, and pharmaceutical compositions comprising such nucleic acid molecules. Methods of production and methods of therapeutic use are also disclosed herein.
e.g.e.g., 5'-UTR and 3'-UTR), wherein the UTRs are heterologous to the encoded protein and capable of increasing the expression of the encoded protein, compared to a corresponding polynucleotide without the UTRs. The present disclosure is also directed to the use of such polynucleotides to treat various diseases and disorders.
C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
A61K 39/145 - Orthomyxoviridae, e.g. influenza virus
The present disclosure includes the use of a miRNA inhibitor for treating a pulmonary disease or disorder, inflammatory disease or disorder, and/or metabolic disease or disorder, such as those associated with an increased level of NLRP3 protein and/or NLRP3 gene expression. In some aspects, the miRNA inhibitor is capable of reducing NLRP3 protein and/or NLRP3 gene expression in a cell.
The present disclosure includes the use of miRNA inhibitor for treating a disease or condition associated with a decreased level of PSD95 and/or synaptophysin and/or an increased level of caspase 3 (e.g., dystonia, neuropsychiatric diseases, intellectual disability, and/or addiction). The miRNA inhibitor useful for the present disclosure can inhibit miR-485 expression and/or activity, which in turn can increase the level of PSD95 and/or synaptophysin protein of gene expression and decrease the level of caspase 3 protein or gene expression.
The present disclosure includes cationic carrier units comprising (i) a water-soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure includes cationic carrier units comprising (i) a water-soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/22 - Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moeity, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an RNA and/or DNA) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.
The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, (iii) a hydrophobic moiety, and (iv) a crosslinking moeity, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an RNA and/or DNA) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.
The present disclosure includes the use of a miRNA inhibitor for inducing hair growth, increasing the hair density, increasing the follicular density, increasing the hair shaft thickness, increasing hair length, preventing hair loss, reducing hair loss, or any combination thereof in a subject in need thereof. In some aspects, the subject has one or more disorders selected from the group consisting of alopecia greata, androgenic alopecia, alopecia areata, alopecia universalis, involutional alopecia, trichotillomania, telogen effluvium, anagen effluvium, cicatricial, alopecia, scarring alopecia, scalp thinning, hair shaft abnormalities, infectious hair disorders, genetic disorders, and hair loss due to chemotherapy, hormonal imbalance, fungal infection, medication intake, chemical hair treatment, or aging.
A61K 9/48 - Preparations in capsules, e.g. of gelatin, of chocolate
A61K 47/24 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
A61K 36/28 - Asteraceae or Compositae (Aster or Sunflower family), e.g. chamomile, feverfew, yarrow or echinacea
The present disclosure includes the use of a miRNA inhibitor for treating a symptom or condition of Huntington's disease. The miRNA inhibitor useful for the present disclosure can inhibit miR-485 expression and/or activity, which in turn can modulate the level of proteins or gene expression related to Huntington's disease.
The present disclosure provides methods and compositions to identify patients suitable for treatment with miRNA-485 inhibitors, e.g., antisense oligonucleotides. The methods disclosed herein can be used to select patients not having pathogenic single nucleotide polymorphisms (SNP) in the biomarker genes CD36, GRIA3, NRXN1, and VLDLR, as candidates for treatment with miRNA-485 inhibitors disclosed herein, and identify patients having such pathogenic SNPs as candidates for alternative therapies. The miRNA-485 inhibitors of the present disclosure can be used for treating a disease or condition associated, e.g., with a decreased level of SIRT1, PGC-1α, CD36, LRRK2, NRG1, and/or STMN2 protein or SIRT1, PGC-1α, CD36, LRRK2, NRG1, and/or STMN2 gene expression. The miRNA-485 inhibitors disclosed herein can inhibit miR-485 expression and/or activity, which in turn can increase the level of SIRT1, PGC-1α, CD36, LRRK2, NRG1, and/or STMN2 protein or gene expression.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure relates to the use of miR-485-3p expression to identify a subject that is afflicted with a cognitive disorder. In some aspects, the methods disclosed herein further comprises administering a miR-485-3p inhibitor to the subject, wherein the miR-485-3p inhibitor is capable of treating the cognitive disorder.
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure provides SETD7 modulators (e.g., polypeptides, polynucleotides, vectors, compositions, micelles, or pharmaceutical composition) which reduce or abolish SETD7 translocation to the nucleus, e.g., in response to stimuli such as increases in glucose levels. In some aspects, the SETD7 modulators mimic phosphorylated STED7, competing with STED7 and reducing or abolishing SETD7 nuclear translocation. In turn, the reduced SETD7 nuclear translocation results in a decrease in histone monomethylation. In some aspects, the SETD7 modulator is a catalytically inactive SETD7 protein. In some aspects, the SETD7 modulator is a polypeptide (e.g., a phosphomimetic polypeptide). In other aspects, the SETD7 modulator is a polynucleotide encoding a SETD7 polypeptide modulator, e.g., a phosphomimetic polypeptide or a mutant SETD7 protein. The SETD7 modulators of the present disclosure can be used to treat type diabetes or cancer.
The present disclosure relates to the use of PGC-1α expression to identify a subject that is conducive to treatment with a miR-485 inhibitor. In some aspects, the subject suffers from a disease or disorder associated with reduced PGC-1α expression. In some aspects, the PGC-1α expression is measured in the serum of the subject.
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
The present disclosure relates to the use of SIRT1 expression to identify a subject that is conducive to treatment with a miR-485 inhibitor. In some aspects, the subject suffers from a disease or disorder associated with reduced SIRT1 expression. In some aspects, the SIRT1 expression is measured in the serum of the subject.
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
The present disclosure includes the use of a miRNA inhibitor for treating a disease or condition associated with a decreased level of SIRT1, PGC-1α, CD36, LRRK2, NRG1, STMN2, VLDLR, NRXN1, GRIA4, NXPH1, PSD-95, and/or synaptophysin protein or SIRT1, PGC-1α, CD36, LRRK2, NRG1, STMN2, VLDLR, NRXN1, GRIA4, NXPH1, PSD-95, and/or synaptophysin gene expression. In some aspects, the miRNA inhibitor can be used to treat a disease or condition associated with an increased level of caspase-3 protein or gene expression. The miRNA inhibitor useful for the present disclosure can inhibit miR-485 expression and/or activity, which in turn can increase the level of SIRT1, PGC-1α, CD36, LRRK2, NRG1, STMN2, VLDLR, NRXN1, GRIA4, NXPH1, PSD-95, and/or synaptophysin protein or gene expression; and/or can decrease the level of caspase 3 protein or gene expression.
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure includes the use of a miRNA inhibitor for treating amyotrophic lateral sclerosis (ALS) associated with a decreased level of SIRT1 protein or SIRT1 gene expression, PGC-1α protein and/or PGC-1α gene expression, CD36 protein and/or CD36 gene expression, NRG1 protein and/or NRG1 gene expression, STMN2 protein and/or STMN2 gene expression, and/or NRXN1 protein and/or NRXN1 gene expression.
The present disclosure includes the use of a miRNA inhibitor for treating a tauopathy associated with a decreased level of SIRT1 protein or SIRT1 gene expression, PGC-1α protein and/or PGC-1α gene expression, and/or CD36 and/or CD36 gene expression.
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure includes cationic carrier units comprising (i) a water soluble polymer, (ii) a positively charged carrier, and (iii) an adjuvant moiety, wherein when the cationic carrier unit is mixed with an anionic payload (e.g., an antisense oligonucleotide) that electrostatically interacts with the cationic carrier unit, the resulting composition self-organizes into a micelle encapsulating the anionic payload in its core. The cationic carrier units can also comprise a tissue specific targeting moiety, which would be displayed on the surface of the micelle. The disclosure also includes micelles comprising the cationic carrier units of the disclosure, methods of manufacture of cationic carrier units and micelles, pharmaceutical compositions comprising the micelles, and also methods of treating diseases or conditions comprising administering the micelles to a subject in need thereof.
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
A61K 47/56 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
34.
COMPOSITIONS AND METHODS FOR PREPARING AN ALZHEIMER'S DISEASE ANIMAL MODEL USING MICRORNA
The present invention relates to a composition for preparing an Alzheimer's disease animal model using microRNA, a non-human Alzheimer's disease animal model, and a method for screening compounds capable of treating Alzheimer's disease using the same.
The present disclosure includes use of a vector for treating a disease or condition associated with a decreased level of a Nurr1 protein. The vector useful for the present disclosure comprises a promoter and an RNA expression region, wherein the RNA expression region is located downstream of the promoter, wherein the RNA expression region comprises a nucleotide sequence expressing an RNA comprising at least one miR-204 binding site, and wherein the RNA expression region does not encode a protein.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
The present invention relates to methods for the diagnosis of and providing information for the diagnosis of Alzheimer's disease or a brain disease using miR-485-3p and to a composition and a kit for the diagnosis of Alzheimer's disease or a brain disease. The present invention enables an objective data analysis of the diagnosis of Alzheimer's disease or a brain disease by measuring the expression level of miR-485-3p in blood, minimizes risks to a patient by measuring the concentration of amyloid beta 42 in saliva, and enables a quick and accurate diagnosis. Accordingly, the present invention is very useful for the prevention of Alzheimer's disease or a brain disease.
The present invention relates to a pharmaceutical composition for preventing or treating brain diseases using miR-485-3p and, more particularly, to a pharmaceutical composition for preventing or treating brain diseases, comprising a miR-485-3p inhibitor, and a method for screening an agent for preventing or treating brain diseases, comprising the step of measuring the expression level of miR-485-3p. According to the present invention, unlike conventional Alzheimer's disease therapeutic agents limited to alleviating symptoms by inducing a reduction in the expression of amyloid beta 42, the composition for treating brain diseases comprising a miR-485-3p inhibitor can restore the protein ELAVL2 and thus can fundamentally treat various diseases, e.g. Alzheimer's disease, autism, mental retardation, and amyotrophic lateral sclerosis, caused by a decrease in the expression level of ELAVL2. Therefore, the present invention is useful for fundamentally treating brain diseases including Alzheimer's disease.
patents
