Resumen de: WO2026006503A1
NLRP3 PET radiotracers are provided, as are methods of using the NLRP3 PET radiotracers to image and/or diagnose diseases and conditions associated with inflammation, such as multiple sclerosis (MS), Alzheimer's disease (AD), traumatic brain injury (TBI), Parkinson's disease (PD), acute myocardial infarction (AMI), heart failure, gout, rheumatoid arthritis, COVID- 19, diabetes, macular degeneration, and autoimmune/autoinflammatory diseases.
Resumen de: WO2026002845A1
The invention relates to an inhibitor of Rho GTPase Cdc42 for use in the prevention and/or treatment of Parkinson's disease of the human or animal body. In a mouse model in which the mice were genetically modified to accumulate α-synuclein and develop Parkinson's disease, it has been shown that the administration of a Cdc42 inhibitor in the diseased mice reduces the accumulation of α-synuclein, improves the symptoms of Parkinson's disease, increases the average life expectancy, and prolongs the total life span. Only minimal to no side effects occurred in the diseased mice.
Resumen de: WO2026002277A1
The present invention provides a nucleic acid molecule used for regulating the level or amount of Tau mRNA. Specifically, the present invention provides the delivery of a primary microRNA to form a precursor after in vivo processing, and a microRNA used for the in vivo inhibition of the expression of Tau mRNA. The present invention also provides a delivery system for the nucleic acid molecule, comprising a vector, an exosome and a cell, and a pharmaceutical composition containing same. The present invention also provides an application of the nucleic acid molecule and the delivery system in neurodegenerative disease treatment and drug preparation, in particular a method and a drug targeting Alzheimer's disease.
Resumen de: WO2026006477A1
Among other tilings, the present disclosure provides various technologies including chirally controlled oligonucleotide compositions and technologies for manufacturing and using such oligonucleotide compositions. In some embodiments, the present disclosure provides technologies useful for allele-specific knockdown of mutant Huntingtin transcripts. In some embodiments, the present disclosure provides technologies usefill for reducing the expression, level, amount, and/or activity of mutant Huntingtin transcripts or products thereof. In some embodiments, the present disclosure provides methods for treating Huntington's disease.
Resumen de: EP4671757A2
Provided herein is the use of a compound of Formula I:or a pharmaceutically acceptable salt thereof, for treating a disease characterized by amyloid and amyloid-like aggregates, e.g., Alzheimer's disease.
Resumen de: WO2025263948A1
The present invention relates to a composition comprising HAPLN1 as an active ingredient for preventing or treating senile degenerative brain diseases. Specifically, recombinant human HAPLN1 protein (rhHAPLN1) lowers the protein level of p16 in cultured human astrocytes to inhibit cellular senescence caused by the accumulation of beta amyloid peptides, and further inhibits phosphorylation (p-p38 MAPK) of p38 MAPK protein, thereby also having the possibility of inhibiting inflammatory responses associated with the onset of Alzheimer's disease and Parkinson's disease. In addition, the recombinant human HAPLN1 protein (rhHAPLN1) exhibits significant memory and learning improvement effects in in vivo experiments performed using a mouse acute Alzheimer's disease model, and thus can be expected to exhibit preventive and therapeutic effects against Alzheimer's disease that may occur with aging and the like. In addition, the inhibitory effect of the rhHAPLN1 protein on cellular senescence and inflammatory responses of astrocytes can provide a very important clue for establishing prevention and treatment strategies not only for aging itself but also for brain functions, motor behaviors, memory, seizures, dementia, brain tumors, and the like.
Resumen de: WO2025264967A1
The present disclosure relates to methods of preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons. The present disclosure also relates to methods of treating, preventing, or delaying the progression of, a synucleinopathy (e.g., Parkinson's disease). Also disclosed are related in vitro, ex vivo, and in vivo methods of identifying agents and/or compositions useful for preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons and agents and/or compositions useful for treating, preventing, or delaying the progression of, a synucleinopathy. The agents and/or compositions of the present disclosure decrease the level and/or activity of a cis-regulatory element that propagates the misfolding and aggregation of proteins encoded by synucleinopathy-associated genes in neurons and/or glial cells.
Resumen de: WO2025265035A1
Provided are crystalline forms of 3-(5-(4-((3,3-dimethyl-4-((1-(6-(5-(1- methylcyclopropoxy)-1H-indazol-3-yl)pyrimidin-4-yl)piperidin-4-yl)methyl)piperazin-1- yl)methyl)piperidin-1-yl)-4-fluoro-1-oxoisoindolin-2-yl)piperidine-2, 6-dione, pharmaceutical compositions comprising the crystalline form, and their use in treating conditions associated with LRRK2, such as Parkinson's disease, dementia, Progressive Supranuclear Palsy, Huntington's disease, or neuroinflammation.
Resumen de: WO2025261396A1
Provided is a nucleic acid molecule used for regulating the level or amount of APP mRNA. Specifically, provided is delivery of primary microRNA for formation of pre- and microRNA following in-vivo processing and for use in the in-vivo inhibition of APP mRNA expression. Provided is a delivery system for the nucleic acid molecule, the delivery system comprising a carrier, an exosome, and a cell, and a pharmaceutical composition containing same. Provided is the use of the nucleic acid molecule and the delivery system in amyloidosis treatment and drug preparation, in particular a method and a drug for Alzheimer's disease.
Resumen de: WO2025264861A1
Compositions and methods for disrupting pathological aggregation and/or mis-localization of TDP-43 in the brain/CNS have been developed. Compositions including engineered helical polypeptides that bind TDP-43's amyloidogenic core but resist β-sheet conversion are provided. In some forms, the engineered polypeptides include peptide degradation motifs (PDM) to enhance proteolytic degradation of aggregates, and/or targeting motifs to direct the peptides to the brain/CNS. Recombinant constructs including nucleic acids expressing or encoding the polypeptides are also provided. Methods of using the engineered peptides to treat or prevent one or more diseases or disorders associated with pathological aggregation and/or mis-localization of TDP-43 in the brain/CNS are also provided. In some forms, the methods treat or prevent ALS or FTD in a subject in need thereof.
Resumen de: WO2025264845A1
The present disclosure relates to the novel use of ANAVEX3-71 and related compounds in medical treatments, such as Parkinson's Disease, Frontotemporal Dementia, Schizophrenia, and Alzheimer's Disease.
Resumen de: WO2025264823A1
This disclosure relates to the use of Purine Nucleoside Phosphorylase (PNP) inhibitors such as ulodesine and its salts, in the treatment and/or prevention of diseases associated with nicotinamide adenine dinucleotide (NAD+) depletion, including diseases of mitochondrial dysfunction (including neurodegeneration and peripheral neuropathies), the preservation of cognitive function and in muscular disorders such as sarcopenia; and in metabolic syndrome and associated conditions. In particular the disclosure provides the use of PNP inhibitors such as ulodesine in the treatment of neurodegenerative conditions such as Parkinson's disease and amyotrophic lateral sclerosis.
Resumen de: WO2025264449A1
The present invention describes methods of treating Huntington's disease in a subject in need thereof. The method comprising administrating to the subject in need thereof a therapeutically effective amount of 2-3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H-1,2,3triazolo4,5-cpyridazin-6-yl-5-(2H-1,2,3-triazol-2-yl)phenol or a pharmaceutically acceptable salt thereof.
Resumen de: US2025388592A1
The application relates to compounds of formula (B1A), (B1B), (B1C) or a salt, a solvate, a hydrate, a polymorph, a tautomer, a racemate or a stereoisomer thereof, to pharmaceutical compositions comprising such a compound, and to the compounds for use as a medicine, in particular for use in the treatment of neurodegenerative disorders such as Alzheimer's disease.
Resumen de: US2025388697A1
Composition and methods of diagnosing, monitoring, and treating subjects with a motor neuron pathology, such as motor neuron disorders (including but not limited to amyotrophic lateral sclerosis (ALS)) and neuropathies.
Resumen de: EP4667461A2
Compounds and pharmaceutical compositions are useful for the treatment of Gaucher disease and Parkinson's disease. Methods of treating Gaucher disease or Parkinson's disease include administration of one or more compounds or pharmaceutically acceptable salts or prodrugs thereof to a subject diagnosed with, or at risk of developing Gaucher disease or Parkinson's disease.
Resumen de: US2025382301A1
This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (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 and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.
Resumen de: WO2025259709A1
The present disclosure provides methods of screening for, identifying and using a Gi-GPCR agonist for a CNS disorder. The CNS disorder can be any disorder in which astrocyte morphology and/or astrocyte tissue support are altered or compromised (e.g., OCD, Alzheimer's disease, or Huntington's disease). Provided herein are methods of screening for and identifying Gi-GPCR agonist ex vivo based on assessment of astrocyte morphology and/or Gi-GPCR activation (e.g., wherein the Gi-GPCR is GPR3711, S1PR1, EDNRB, GRM3, or AD0RA2A). Also provided herein are methods of identifying a therapeutic agent for the treatment of a CNS disorder in vivo at least in part based on its effect on astrocyte morphology and/or Gi-GPCR activation. Also provided herein are methods for the treatment or prevention of a CNS disorder comprising administering to a subject a Gi-GPCR (e.g., GPR3711, S1PR1, EDNRB, GRM3, or AD0RA2A) agonist.
Resumen de: US2025382274A1
Compounds and pharmaceutical compositions are useful for the treatment of Gaucher disease and Parkinson's disease. Methods of treating Gaucher disease or Parkinson's disease include administration of one or more compounds or pharmaceutically acceptable salts or prodrugs thereof to a subject diagnosed with, or at risk of developing Gaucher disease or Parkinson's disease.
Resumen de: US2025381230A1
Disclosed are compositions and methods for treating Alzheimer's disease or for use in treating Alzheimer's disease. Also disclosed are compositions and methods for treating or for use in treating a neurodegenerative disorder characterized by the presence of tau protein aggregates. Furthermore, disclosed are compositions and methods for reducing tau protein aggregates or for use in reducing tau protein aggregates.
Resumen de: US2025381197A1
The present invention relates to the treatment of an ALS patient having disease onset of at least 24 months prior to initiation of treatment with fasudil. Fasudil is administered at a dose of 60-240 mg/day according to specific treatment regimens. This results in an anticipated 25-50% reduction in the average decline over at least three months as measured using the revised ALS Functional Rating Scale.
Resumen de: US2025381305A1
Provided is a method for treating a Parkinson Disease, comprising a TRIM72 protein modulator. Further provided is a composition comprising the TRIM72 protein modulator and use thereof.
Resumen de: AU2024286486A1
Disclosed herein are methods of 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 based on the risk of an ARIA event or brain hemorrhage. Also disclosed herein are methods of treating subjects having or suspected of having AD comprising subcutaneously administering an anti-Aβ protofibril antibody.
Resumen de: AU2024285799A1
The present invention relates to a pharmaceutical composition for the prevention or treatment of periodontal disease, or atherosclerosis or Alzheimer's disease caused by periodontal disease. More specifically, the present invention relates to a pharmaceutical composition for the prevention or treatment of periodontal disease, or atherosclerosis or Alzheimer's disease caused by periodontal disease, the composition comprising a peptide with the amino acid sequence of SEQ ID NO: 1, which is effective in inhibiting osteoclast formation, suppressing the formation of Porphyromonas gingivalis colonies, and inhibiting gingipain expression, while being safe for the body and having fewer side effects, including adverse reactions.
Nº publicación: WO2025258415A1 18/12/2025
Solicitante:
SANBIO CO LTD [JP]
KEIO UNIV [JP]
SANBIO CO. LTD,
KEIO UNIVERSITY
Resumen de: WO2025258415A1
Disclosed are compositions and methods for treating Alzheimer's disease or for use in treating Alzheimer's disease. Also disclosed are compositions and methods for treating or for use in treating a neurodegenerative disorder characterized by the presence of tau protein aggregates. Furthermore, disclosed are compositions and methods for reducing tau protein aggregates or for use in reducing tau protein aggregates.