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146
resultados
Última actualización
24/04/2025 [07:30:00]
Resumen de: US2025121095A1
Provided is a recombinant adeno-associated virus (rAAV) or recombinant lentivirus, comprising an expression cassette in the genome, the expression cassette comprises a polynucleotide encoding thiamine pyrophosphokinase (TPK), which is operatively linked to a promoter. Also provided are also a pharmaceutical composition comprising the rAAV or recombinant lentivirus, and use of the rAAV, recombinant lentivirus and the pharmaceutical composition in the preparation of a medicament for treating or preventing Alzheimer's disease.
Resumen de: US2025122146A1
Disclosed herein are compounds, their pharmaceutical compositions, and their methods of use for treating a neurodegenerative disease, such as Alzheimer's disease. Lewy body dementia, or Parkinson' disease. In some embodiments, the compound is one that activates the nuclear factor (erythroid-derived 2)-like 2 (Nrf2) and/or heat-shock factor-1 (HSF-1) transcription-mediated signaling pathway: the compound is administered with at least one antibody that is directed against an aberrant misfolded protein. The compound, illustrated by camosic acid in one example, is unexpectedly effective in reducing the type of neuroinflammation resulting from antibody-protein complexes encountered in antibody therapies of the disease. The compounds also are useful in a method of treating neuroinflammation in a subject who suffers from a neurodegenerative disease and/or has been administered at least one antibody that is directed against an aberrant misfolded protein.
Resumen de: US2025120957A1
This invention relates to the treatment of a neuropsychiatric disorder, such as schizophrenia or Parkinson's disease, by administration (for example, transdermally) of tilivapram, zatolmilast, roflumilast, or a pharmaceutically acceptable salt thereof.
Resumen de: US2025121020A1
A composition including Yukgunjatang as effective component is effective for improving memory and preventing, alleviating, or treating cognitive disorder. Yukgunjatang, which is prepared by boiling a mixture of Gingseng Radix, Atractylodes rhizoma alba, Hoelen, Glycyrrhizae Radix, Aurantii Nobilis Pericarpium, Pinelliae Rhizoma, Zingiberis Rhizoma, and Zizyphi Fructus in water, exhibits superior neuroprotective effect compared to individual extracts of Gingseng Radix, Atractylodes rhizoma alba, Hoelen, Glycyrrhizae Radix, Aurantii Nobilis Pericarpium, Pinelliae Rhizoma, Zingiberis Rhizoma, and Zizyphi Fructus, and, in an animal model of cognitive decline induced by scopolamine, administration of Yukgunjatang shows the effect of improving memory and cognitive function. Thus, the composition can be advantageously used as a food product, a medicinal product, or the like for preventing or treating brain diseases including Alzheimer's disease, Parkinson's disease, and mild cognitive impairment.
Resumen de: US2025121013A1
Disclosed herein are compositions and methods for preventing, ameliorating, or treating Parkinson's disease and/or reducing the severity of one or more risk factors, signs, or symptoms associated with Parkinson's disease. In particular, the technology of the present disclosure relates to methods for administering an effective amount of a composition comprising one or more strains of an operational group Bacillus amyloliquefaciens bacteria, identified as ART24 and ART12, to a subject suffering from or at risk for Parkinson's disease.
Resumen de: WO2025080080A1
According to pharmaceutical composition for preventing or treating Parkinson's disease, containing a pyruvate dehydrogenase kinase (PDK) inhibitor as an active ingredient, a pharmaceutical preparation for preventing or treating Parkinson's disease, containing same, and a method for predicting a Parkinson's disease therapeutic effect of a candidate drug, of the present invention, it can be expected that a candidate drug treating Parkinson's disease and having an optimal therapeutic effect can be selected.
Resumen de: WO2025080252A1
The present disclosure provides methods of treating Alzheimer's disease and other disorders using (4R,5R)-5-(2-chlorophenyl)-4-(5-(phenylethynyl)pyridin-3-yl)oxazolidin-2-one (Compound 1).
Resumen de: WO2025080753A1
The present invention relates to bifunctional compounds, which find utility to degrade and (inhibit) one or more of the following kinases: DYRK1A, DYRK1B, DYRK2, DYRK3, DYRK4, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, HIPKs, and/or CMGC kinases leading to inhibition of WNT signaling. In particular, the present invention is directed to compounds, which contain on one end an E3 ubiquitin ligase binding moiety which binds to an E3 ubiquitin ligase and on the other end a moiety which binds one or more of the following kinases: DYRK1A, DYRK1B, DYRK2, DYRK3, DYRK4, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, HIPKs, and/or CMGC kinases leading to inhibition of WNT signaling, such that the one or more kinases is placed in proximity to the ubiquitin ligase to effect degradation (and inhibition) of the one or more kinases. The bifunctional compounds serve as therapeutics for the treatment of Alzheimer's disease, down syndrome, diabetes, an autoimmune disease, an inflammatory disorder (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer, acute myeloid leukemia, myelodysplastic syndrome), a viral infection (e.g., SARS-CoV-2 infection (e.g., COVID-19)), and other diseases.
Resumen de: WO2025077839A1
The use of α-ketoglutaric acid in the preparation of a drug for preventing and treating demyelination-related diseases. The α-ketoglutaric acid or a derivative thereof is used for preventing and/or treating myelin sheath defects in demyelination-related diseases, such as demyelinating diseases, amyotrophic lateral sclerosis, Huntington's disease, hypomyelinating leukodystrophy, Alzheimer's disease, Parkinson's syndrome and diabetes-related visual impairment, and can promote myelin sheath generation, regeneration or repair, and improve the immune microenvironment in the lesion area.
Resumen de: WO2025078660A1
An isolated protein comprising (i) a first protein moiety selected from the group of proteins comprising an amino acid sequence having at least 70% identity to residues 113-231 of Bri2 from human (SEQ ID NO: 2); and proteins comprising an amino acid sequence having at least 70% identity to any one of the BRICHOS domains of Bri2 from human (SEQ ID NO: 5), chimpanzee (SEQ ID NO: 6), bovine (SEQ ID NO: 7), pig (SEQ ID NO: 8), mouse (SEQ ID NO: 9) and rat (SEQ ID NO: 10); and and optionally (ii) a second protein or polypeptide moiety, preferably containing at least 50 amino acid residues, wherein the second protein or polypeptide moiety is selected from the group consisting of protein drugs, polypeptide drugs, antibodies and/or neurotrophic factors; wherein the second protein or polypeptide moiety is effective for treatment of Parkinson's Disease; for use as a medicament, in particular for treatment of Parkinson's Disease.
Resumen de: WO2025080415A1
This invention relates to the treatment of a neuropsychiatric disorder, such as schizophrenia or Parkinson's disease, by administration (for example, transdermally) of tilivapram, zatolmilast, roflumilast, or a pharmaceutically acceptable salt thereof.
Resumen de: EP4537846A2
Composition and method for treatment of Alzheimer's disease that includes ANAVEX2-73. Method of treatment of Alzheimer's disease using pharmaceutical compositions comprising ANAVEX2-73 according to an intermittent dosage regimen.
Resumen de: WO2023245008A1
Provide herein are methods of treating human patients with familial Alzheimer's disease that result in delayed in symptom onset and/or slowed cognitive decline by administering a humanized monoclonal anti-amyloid beta (Aβ) antibody.
Resumen de: EP4537842A1
The present invention addresses the problem of providing an agent for preventing or treating amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Huntington's disease (HD), spinocerebellar ataxia (SCA), aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging, as well as a more stable antibody that exhibits an effect of preventing or treating these diseases, Alzheimer's disease (AD), or frontotemporal lobar degeneration (FTLD). A human monoclonal antibody that specifically binds to human HMGB1, wherein the human monoclonal antibody (anti-human HMGB1 antibody) comprises a heavy chain CDR1, heavy chain CDR2, and heavy chain CDR3 each consisting of a specific amino acid sequence and a light chain CDR1, light chain CDR2, and light chain CDR3 each consisting of a specific amino acid sequence, is used as an agent for preventing or treating ALS, PD, HD, SCA, aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging. An antibody in which the light chain complementarity determining region (CDR) 3 of the anti-human HMGB1 antibody has been modified is used.
Resumen de: EP4537845A1
The present invention pertains to a method for controlling a membrane potential-dependent ion channel (VGSC or the like) through a type I taste receptor present in a nerve cell or the like. In the present invention, it has been found that an A β peptide, or a sweet amino acid or an umami substance specifically binds to a type I taste receptor on the surface of a nerve cell to exert an agonist-like or antagonist-like action, thereby amplifying or suppressing a VGSC active current.Moreover, with the binding of an Aβ peptide or the like to a type I taste receptor, the amplification of a VGSC active current occurs, the overactivity of nerve cells causing epileptiform attack occurs, and a large number of substances, which can effectively suppress the amplification of the VGSC active current, among ligand substances that specifically bind to the type I taste receptor, can be found.The present invention provides: a type I taste receptor-specific ligand substance that can control the amplification or suppression of a VGSC active current; and a pharmaceutical composition for preventing or treating various neurodegenerative diseases, such as Alzheimer's disease (AD), due to the amplification of a VGSC active current caused by the binding of an Aβ peptide or the like to a type I taste receptor. Moreover, a method for using, as a target receptor, a type I taste receptor present in a nerve cell or the like to screen a ligand substance for controlling a VGSC or the like in the cell is a
Resumen de: US2025114397A1
The present invention provides a method of treating frontotemporal dementia, or a childhood genetic neurodegenerative disease such as Ataxia Telangiectasia (A-T), or neurodegenerative diseases such as Parkinson's disease or neuropsychiatric diseases comprising administering to a subject in need thereof an effective amount of chlorite composition, such as sodium chlorite. The present invention thereby provides a method of modulating the immune system in a subject in need thereof. Described herein are methods of administration and treatment.
Resumen de: US2025115911A1
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SNCA gene, particularly in a CNS tissue, as well as methods of inhibiting expression of a SNCA gene and methods of treating subjects having a SNCA-associated neurodegenerative disease or disorder, e.g., Parkinson's Disease (PD), multiple system atrophy (MSA), Lewy body dementia (LBD), among other synucleinopathies, using such dsRNAi agents and compositions.
Resumen de: US2025115604A1
Compounds of formula (I)wherein A, R, W, Q, n and m have the meaning according to the claims can be employed, inter alia, for the treatment of tauopathies and Alzheimer's disease.
Resumen de: WO2025075789A1
Human tau protein phosphorylated at the amino acid, serine 413 (pS413 tau), can serve as a biomarker for tauopathies such as Alzheimer's disease. Detection and quantitation of pS413 tau in a biological sample such as cerebrospinal fluid can be useful in developing therapeutics for certain tauopathies. However, pS413 tau is present in biological samples at very low levels. Thus, the invention provides a highly sensitive assay for the detection and quantitation of pS413 tau in a biological sample comprising a series of steps as described herein.
Resumen de: WO2025076156A1
This invention provides a method for determining whether a human subject is afflicted with Alzheimer's disease ("AD") or non-Alzheimer's dementia ("non-ADD") when the subject is suspected of being afflicted with AD or non-ADD, comprising the steps of (a) synchronizing a population of suitable cells derived from the subject, wherein the suitable cells are cultured skin cell fibroblasts or cultured B lymphocytes; and (b) in the resulting synchronized cell population, measuring the expression levels of two or more genes selected from the group consisting of FAM149B, NHLH1, SHISA5, URB2, and WASF2, whereby (i) the subject is afflicted with AD if the expression levels measured in step (b) are consistent with those genes' expression levels in corresponding synchronized cells derived from AD patients, and (ii) the subject is afflicted with non-ADD if the expression levels measured in step (b) are consistent with those genes' expression levels in corresponding synchronized cells derived from non-ADD patients. This invention also provides related diagnostic and therapeutic methods, including diagnostic methods based on NDS subject gene expression levels.
Resumen de: WO2025076219A1
The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease (HD).
Resumen de: WO2025076236A1
The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease (HD).
Resumen de: WO2025076497A1
A method for treating or preventing a tau pathology such as Alzheimer's disease in a subject in need thereof is described. The method includes administering a therapeutically effective amount of an amyloid-β receptor inhibitor in combination with a quercetin analog to the subject. Anti-tau pathology compositions including a therapeutically effective amount of an AβR inhibitor in combination with a quercetin analog are also described.
Resumen de: AU2023330511A1
The present disclosure relates to an sgRNA and its application in the preparation of a product for the treatment of Huntington's disease. The present disclosure was designed and screened to obtain an sgRNA targeting exon 1 of the human HTT gene as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The CRISPR/Cas9 system mediated HTT gene knockout strategy based on this sgRNA and its high homologue sgRNA can efficiently knock out the human Huntingtin gene and achieve gene therapy for Huntington's disease.
Nº publicación: AU2023353995A1 10/04/2025
Solicitante:
ATALANTA THERAPEUTICS INC
ATALANTA THERAPEUTICS, INC
Resumen de: AU2023353995A1
The present disclosure provides single- or double-stranded interfering RNA molecules (e.g., siRNA) that target a membrane-spanning 4-domains AGA (MS4A6A) gene. The interfering RNA molecules may contain specific patterns of nucleoside modifications and internucleoside linkage modifications, as pharmaceutical compositions including the same. The siRNA molecules may be branched siRNA molecules, such as di-branched, tri-branched, or tetra-branched siRNA molecules. The disclosed siRNA molecules may further feature a 5' phosphorus stabilizing moiety and/or a hydrophobic moiety. Additionally, the disclosure provides methods for delivering the siRNA molecule of the disclosure to the central nervous system of a subject, such as a subject identified as having a neuroinflammatory disease (e.g., Alzheimer's disease).
BOPI
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