Resumen de: AU2023347329A1
This disclosure relates to, inter alia, certain compounds, compositions, and pharmaceutical compositions thereof, that modulate the activity of the transporter protein vesicular monoamine transporter-2 (VMAT2) and are directed to methods useful in the treatment of transporter protein vesicular monoamine transporter-2 mediated disorders, such as, neurological or psychiatric disease or disorders, including but not limited to, hyperkinetic movement disorders (e.g., tardive dyskinesia, Tourette's syndrome, Huntington's disease, tics, ataxia, chorea (such as, chorea associated with Huntington's disease), dystonia, hemifacial spasm, myoclonus, restless leg syndrome, and tremors). The disclosure further relates to synthetic methods and intermediates useful in the preparation of the compounds.
Resumen de: AU2023406056A1
Disclosed herein are methods of diagnosing, selecting, monitoring, and treating subjects with Alzheimer's disease (AD) or suspected of having AD or another disorder associated with amyloid accumulation in the brain using a tau PET level.
Resumen de: WO2023250249A1
As described herein, inhibitors of High mobility group box protein 1 (HMGB1) can significantly reduce HMGB1 nucleo-cytoplasmic translocation, gliosis, neurodegeneration, Tau pathologies, and myelin deficits, especially in subjects having an AP0E4 allele. Methods are therefore described herein that include administering one or more inhibitors of High mobility group box protein 1 (HMGB1) to a subject having at least one genomic AP0E4 allele.
Resumen de: EP4545071A1
A sustained-release microsphere containing a drug, pamoic acid, and a biocompatible polymer, a pharmaceutical composition containing the sustained-release microsphere for prevention, improvement, or treatment of hepatitis B, prostate cancer, breast cancer, endometriosis, uterine fibroid, precocious puberty, acromegaly, gastro·entero·pancreatic endocrine tumor, Alzheimer's disease, or cognitive disorder, and a preparing method of the sustained-release microsphere are provided.
Resumen de: AU2023360721A1
The present relates to polynucleotide constructs encoding an ApoE3 related protein optionally containing one or more intron. Potential uses of the different constructs include gene therapy targeting one or more disease or disorder, for example, diseases or disorders related to cholesterol levels, atherosclerosis, coronary heart disease, dementia, cerebral amyloid angiopathy, or Alzheimer's disease.
Resumen de: WO2025084727A1
The present invention relates to a composition for delaying or treating Huntington's disease, comprising a Lin28A inhibitor as an active ingredient. The present invention relates to various inhibitors capable of having the effect of preventing or treating Huntington's disease by inhibiting the expression of Lin28A, which is highly secreted specifically in Huntington's disease, and a screening method therefor.
Resumen de: WO2025082069A1
The present invention belongs to the technical field of medicinal chemistry, specifically relates to a compound capable of binding to a CRBN protein and a composition thereof, and also relates to a protein degradation agent based on a CRBN protein and the use thereof. Specifically disclosed is a compound capable of binding to a CRBN protein, wherein the compound is composed of two sub-structure units A and B, the two sub-structures A and B are connected by means of a chemical single bond between Y of the sub-structure A and one of C2 (carbon at position 2), C4 (carbon at position 4), C5 (carbon at position 5), C6 (carbon at position 6) and C7 (carbon at position 7) of the sub-structure B, and the compound is as shown in formula I. The compound can be used as a structural unit of an E3 ubiquitin ligase ligand in PROTAC technology, thereby providing more therapeutic means for cancers or diseases such as prostate cancer, breast cancer, non-small cell lung cancer, Alzheimer's disease, neuroblastoma, hepatocellular carcinoma, colorectal cancer, pancreatic cancer, malignant rhabdomyosarcoma, and oral squamous cell carcinoma.
Resumen de: WO2025085624A1
The present disclosure provides synthetic polypeptides that mimic the Switch 2 domain of a Rab protein. The synthetic polypeptides described are capable of inhibiting, modulating, or decreasing the interaction between a Rab protein and an RILPL protein. Methods of treating neurological diseases, disorders, or conditions, such as Parkinson's disease, are also provided.
Resumen de: WO2025085704A1
This disclosure relates to vectors, compositions, pharmaceutical compositions, and kits that provide for brain cell-specific expression of reprogramming genes such as the Yamanaka factors Oct4, Sox2, Klf4 and c-Myc (OSKM). Also provided are methods and uses comprising the same for treating Alzheimer' s disease and progeria through brain cell-specific expression of reprogramming genes such as OSKM.
Resumen de: WO2025083211A1
The inventors hypothesized that inhibition of complement activation could reduce the inflammatory period observed even before clinical signs of Alzheimer's disease and thus slow down the onset and progression of AD. In order to validate the hypothesis, the inventors injected Factor H (FH: the main inhibitor of complement activation) into the brain of APP/PS1 AD- mice model at early or late stage of this pathology. The results showed effects of FH brain injection on the AD-onset as well as progression by reducing pro-inflammatory IL6, TNF-α, Il1β, MAC and Aβ levels associated with an increase of VGLUT1 and Psd95 neurotransmitters levels in hippocampal region leading to improvement of cognitive functions even at late stage of the pathology. The results thus prompt the inventors to consider that FH would be suitable for the treatment of dementia, and more particularly for the curative treatment of dementia.
Resumen de: US2025127775A1
The present invention discloses a method to recover and restore dendritic and synaptic neuron connections that have been degraded or destroyed by neurodegenerative diseases. In the present invention tryptamines are used to induce neuro plasticity and restore both dendritic density and synaptic connections of neurons in the brain. In the preferred embodiment LSD given in micro doses can induce dendritic and synaptic genesis in neuronal networks and improve the quality of life of people with neurodegenerative diseases such as Alzheimer's, Huntington's, Multiple Sclerosis, Parkinson's and Frontotemporal dementia.
Resumen de: US2025127817A1
An application of Bone Marrow Mesenchyml Stem Cell Exosomes (BMSC-Exos) in treating Parkinson's disease (PD) is provided, wherein the BMSC-Exos are generated by stimulating BMSCs with a culture solution and extracted from the culture solution after passage; and the culture solution of the BMSCs is an α-MEM culture solution containing FBS and PS. The BMSC-Exos can greatly improve a motor function of a model mouse with PD, protect dopaminergic neurons of the model mouse with PD, improve an olfactory function of the model mouse with PD, and also inhibit the activation of olfactory astrocytes of the model mouse with PD.
Resumen de: US2025129158A1
The invention provides compositions and methods for suppressing autoimmune components of neurodegenerative diseases and thereby providing therapeutic effects to patients suffering from such diseases. Compositions and methods include immunosuppressive moieties such as regulatory T cells (Tregs) and proteins expressed by Tregs coupled to a chimeric antigen receptor or protein that specifically binds one or more glial cell markers. Therapeutically effective doses of said compounds for treating neurodegenerative diseases including progressive supranuclear palsy (PSP), Parkinson's disease (PD), Alzheimer's, Huntington's disease, amyotrophic lateral sclerosis (ALS), chronic traumatic encephalopathy (CTE), and prion diseases are disclosed.
Resumen de: AU2023351193A1
Provided herein are methods and compositions that block Integrin Subunit beta 8 (ITGB8, also known as integrin αvβ8) to treat neurodegenerative diseases associated with microglial impairment including Alzheimer's disease (AD) and amyotrophic lateral sclerosis (ALS).
Resumen de: AU2023358527A1
Described herein are methods and compositions for treating Alzheimer's Disease (AD), as well as compositions comprising a reelin-derived peptide and methods of use thereof.
Resumen de: US2025127867A1
The application describes a phosphorylated tau targeted active immunotherapy to treat preclinical Alzheimer's Disease.
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: 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: 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: 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: 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: 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: 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.
Nº publicación: WO2025077839A1 17/04/2025
Solicitante:
WEST CHINA SECOND UNIV HOSPITAL SICHUAN UNIV [CN]
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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.