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1049
resultados
Última actualización
13/01/2026 [07:05:00]
Resumen de: AU2024283557A1
Disclosed herein are UNC13A oligonucleotides with one or more spacers or without a spacer. In various embodiments, UNC13A oligonucleotides with spacer(s) reduce mis-spliced UNC13A transcripts and increase full length UNC13A transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).
Resumen de: AU2024305538A1
The present disclosure provides methods of differentiating pluripotent stem cells, including induced pluripotent stem cells, into lineage-specific floor plate midbrain progenitor cells, determined dopaminergic neuronal progenitor cells, committed dopaminergic neuronal progenitor cells and/or dopaminergic neuronal cells. Also provided are compositions uses thereof, such as for treating neurodegenerative diseases and conditions, including Parkinson's disease, and articles of manufacture and kits for use thereof.
Resumen de: WO2026010936A1
Provided herein are compositions, including epitope megapools, and methods for detecting the presence of: a neurodegenerative disorder-associated or an immune response relevant to a neurodegenerative disorder including T cells responsive to one or more Neurodegenerative disease-associated peptides or proteins, fusion protein, a pool of two or more peptides, a polynucleotide encoding the same comprising, consisting of, or consisting essentially of: one or more amino acid sequences of a target set forth SEQ ID NOS: 1 to 123, Table 1, or Table 2. The invention further provides vaccines, diagnostics, therapies, and kits, comprising such proteins or peptides.
Resumen de: WO2026010789A1
This disclosure provides compounds of Formula I and the pharmaceutically acceptable salts thereof. The variables, e.g., R1-R6, L, and W are defined herein. The compounds of the disclosure are highly selective D2 receptor antagonists, useful for treating schizophrenia, depression, bipolar disorder, post-operative nausea or vomiting, Tourette's syndrome, tardive dyskinesia, Huntington's chorea, and gastroesophageal reflux disease. The disclosure also provides pharmaceutical compositions comprising a compound or salt of Formula I.
Resumen de: WO2026006909A1
The present application relates to treatment of Alzheimer's disease. More specifically, the present application relates to a method for treating, palliating or preventing progression of Alzheimer's disease in a subject in need thereof, or improving cognition, suicidality, behavioral disturbance, clinical global impression, and/or functional ability in a subject with Alzheimer's disease, or improving global cognition, the method comprising administering to the subject at least about 50 mg of Montelukast daily, wherein the Montelukast is formulated as an oral dosage film for oral administration.
Resumen de: EP4674433A2
Provided herewith is a pharmaceutical composition comprising, separately or together, a pulsatile release component comprising levodopa and a DOPA decarboxylase inhibitor for the management of OFF-time episodes in patients with Parkinson's disease.
Resumen de: MX2025014083A
Herein is reported a bispecific antibody specifically binding to human Abeta protein and human transferrin receptor (bispecific anti-Abeta/TfR antibody) as well as the use of such bispecific antibodies as a medicament in the treatment of Alzheimer's Disease, including where the bispecific antibody is administered intravenously at a dose of 0.2 mg/kg to 7.2 mg/kg once every four weeks.
Resumen de: MX2024002080A
The present invention is directed to a method for treating Parkinson's disease. The method comprises administering to a subject in need thereof dapansutrile, in an effective amount. Dapansutrile minimizes the clinical features of PD such as locomotor impairments through the modulation of the inflammatory response, reduction in α-synuclein levels, and protection of dopaminergic neurons. A preferred route of administration is oral administration.
Resumen de: MX2025009971A
Provided herein is an anti-CD2 antibody or antigen binding fragment thereof for treating and/or preventing ALS in a subject in need thereof.
Resumen de: MX2025013653A
The present disclosure pertains to compositions and methods for the treatment and/or prevention of one or more of obesity, diabetes, metabolic syndrome, Alzheimer's disease, Chronic Fatigue Syndrome (CFS), aging, fibromyalgia, dyslipidemia, hypercholesterolemia, dyslipidemia, Parkinson's disease, migraines, Traumatic Brain Injury (TBI), Attention Deficit Disorder (ADD)/ Attention Deficit Hyperactivity Disorder (ADHD), Cancer, Cardiovascular Disease (CVD)ZCoronary Artery Disease (CAD), Chronic Pain, neuralgia, depression, amyotrophic lateral sclerosis (ALS), and epilepsy, Insufficient Cellular Energy (ICE) and mitochondrial dysfunction. The present disclosure also pertains to methods for increasing mental and/or physical performance levels and/or decreasing exertion during exercise in a subject by the administration of C5 ketones.
Resumen de: MX2025009645A
Provided herein are compounds, phannaceutical compositions, and methods of use for reducing the amount or activity of SNCA mRNA in a cell or subject, and in certain instances reducing the amount of alpha-synuclein protein in a cell or subject. Such compounds, pharmaceutical compositions, and methods of use are useful to ameliorate at least one symptom or hallmark of a synucleinopathy. Such synucleinopathies include Parkinson's disease, dementia with Lewy bodies (DLB), diffuse Lewy body disease, Parkinson's disease dementia (PDD), pure autonomic failure, multiple system atrophy (MSA), neuronopathic Gaucher's disease, and Alzheimer's disease.
Resumen de: GB2642355A
The present disclosure relates to crystalline polymorphs of the compound of Formula (I), namely N-(4-bromo-2,5-difluorophenyl)-6-chloro-1H-pyrrolo2,3-bpyridine-3-sulfonamide: Crystalline forms designated F1, F2, and F3 are provided. Each form is characterized by a distinct X-ray powder diffraction (XRPD) pattern. Form F1 exhibits characteristic 2θ peaks at approximately 7.3, 7.8, 8.1, 12.9, 16.8, 19.8, 25.5, 25.9, 26.2, and 32.3° (± 0.2°). Form F2 exhibits characteristic peaks at approximately 8.1, 14.8, 16.6, 18.8, 19.5, 21.1, 22.6, 26.0, 26.7, and 28.8° (± 0.2°). Form F3 exhibits characteristic peaks at approximately 11.8, 15.2, 15.4, 17.9, 23.8, 24.4, 26.6, 28.3, 28.8, and 30.5° (± 0.2°). Pharmaceutical compositions comprising one of these crystalline forms and one or more pharmaceutically acceptable excipients are also provided. The disclosed crystalline forms and compositions are useful in the treatment or prophylaxis of GPR17-associated diseases, including multiple sclerosis, neuromyelitis optica, and other demyelinating disorders, as well as neurodegenerative conditions such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis. Methods for manufacturing pharmaceutical formulations comprising these crystalline forms are also described.
Resumen de: WO2024182311A2
Disclosed are novel strategies for the treatment of patients with Parkinson's disease and other primary and secondary Parkinsonian disorders by enhancing cell engraftment. Cell viability, engraftment, proliferation, migration, or differentiation of administered DA neuronal cells is enhanced by treating the patient with an antilipemic agent and/or a CSF-1R antagonist before, during and/or after transplantation of DA neuronal cells.
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: 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: 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: 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: 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: 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: 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: 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: 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.
Nº publicación: WO2025264861A1 26/12/2025
Solicitante:
YALE UNIV [US]
YALE UNIVERSITY
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.
BOPI
Sede Electrónica
