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153
resultados
Última actualización
13/02/2026 [07:27:00]
Resumen de: AU2024330245A1
Provided are a gene therapy vector for treating Parkinson's disease and a use thereof. Specifically, provided is an adeno-associated virus (AAV) vector for treating Parkinson's disease, which can simultaneously express functional tyrosine hydroxylase (TH), GTP-cyclohydrolase 1 (GCH1) and aromatic amino acid decarboxylase (AADC) to promote dopamine synthesis. Also provided are an AAV virus particle containing the AAV vector, a composition containing the AAV vector or the AAV virus particle, and uses of the AAV vector, the AAV virus particle and the composition in the preparation of drugs for preventing or treating Parkinson's disease.
Resumen de: AU2024343037A1
Provided is the new compositions and methods useful for the treatment, prevention, and potential cure of a genetic disease, such as familial Alzheimer's Disease, by disrupting the genomic sequence harboring one or more SNPs that are of high prevalence in a population but no relevance to the particular disease except for their genomic locations being in the same gene exon as a disease-relevant allele and upstream from such disease-relevant allele present in the genome of a treatment recipient.
Resumen de: AU2024315761A1
Among other things, 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 useful 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: AU2024299217A1
The present invention relates to the use of bis(phenylmethylene)cycloalkanones and heterocyclic analogues thereof in human and veterinary medicine, for the treatment of diseases caused by the presence or elevated levels of metastable proteins in the cell, imbalances in protein homeostasis and proteotoxic stress, in general proteinopathy, in particular their use in the treatment of neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Alzheimer's disease (AD), Kennedy's disease (KD), Huntington's disease (HD), Creutzfeldt-Jakob disease (CJD), spinocerebellar ataxia (SCA), dentatorubral- pallidoluysian atrophy, transthyretin familial amyloid polyneuropathy, systemic amyloidosis, organ- confined amyloidosis, cystic fibrosis, and diabetes. Furthermore, the invention provides novel bis(phenylmethylene)cycloalkanones.
Resumen de: EP4691472A1
For dementia, particularly Alzheimer's disease, there is currently no effective agent for ameliorating or treating it. The object of the present invention is to find a drug for ameliorating or treating dementia thereby providing a novel agent for ameliorating or treating dementia. The present invention provides an agent for ameliorating or treating dementia, which comprises cyclic phosphatidic acid, carbacyclic phosphatidic acid, thiacyclic phosphatidic acid, or carba-lysophosphatidic acid, or a salt thereof.
Resumen de: WO2026030596A1
The present disclosure provides recombinantly manufactured fusion proteins comprising a HERV-K (HML-2) Env protein fragment or an analog thereof linked to a human Fc fragment. Embodiments include the administration of the fusion proteins to patients having a disease or a disorder with the intention of mitigating and/or reducing the duration of symptoms associated with the condition or disease (for example but not limited to muscular weakness, paralysis and respiratory failure), and/or preventing symptoms associated with the condition or disease, for example, by preventing motor neuron degeneration and cell death in ALS patients associated with the condition or disease. Accordingly, "treatment" generally means both therapeutic treatment and prophylactic or preventative measures. Improvement after treatment may be manifested as a decrease or elimination of such symptoms, e.g., by a decrease or elimination of symptoms associated with ALS, and/or by a decrease in the duration of such symptoms.
Resumen de: WO2026030249A1
Described herein is an active Alzheimer's Disease (AD) immunotherapy based on a nanoparticle vaccine comprising a plurality of Aβ peptides and/or a plurality of tau peptides. These peptides may correspond to both soluble and aggregated targets and are displayed on the surface of immunogenic liposomes in an orientation that maintains reactivity with epitope-specific monoclonal antibodies. Also provided are methods of making and using same.
Resumen de: US20260034109A1
Provided herein is a method for treating a human subject afflicted with ALS by administering to the subject a therapeutically effective amount of pridopidine or pharmaceutically acceptable salt thereof.
Resumen de: US20260035342A1
There are provided isotope-enriched compounds of Formula (I) and pharmaceutically acceptable salts or esters thereof, as well as pharmaceutical compositions thereof and methods of use thereof for prevention and treatment and amyloid-β related diseases, such as Alzheimer's disease.
Resumen de: US20260034085A1
The invention is a method for treating patients with Parkinson's disease by orally administering a controlled release levodopa formulation and the method provides an improvement of a patient's total post-dose “Off” time, total post dose “On” time and total post dose “Good On” time compared to post-dose of treatment regimens with oral immediate release levodopa tablets.
Resumen de: US20260034088A1
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: US20260034233A1
Provided herein are antibodies that target Galectin-3. Such antibodies are used in methods of treating epilepsy and related neurological disorders, such as Alzheimer's disease (AD) and Parkinson's disease (PD).
Resumen de: US20260034143A1
Compositions of Allopregnanolone (Allo), and methods of use thereof for treating and preventing Alzheimer's Disease (AD) or dementia have been developed. In some embodiments, the amount of Allo effective to treat AD or dementia is between about 2 mg and about 10 mg, preferably 4 mg per dose. Methods for identifying subjects for treatment of AD or dementia are also provided. The methods include selecting a subject having one or more Apo E4 gene alleles. Methods of treating a human subject having AD or at risk of AD OR DEMENTIA are provided. The methods include administering a dosage of from 2 mg to 6 mg to the subject once within a 24 hour period. The dosing is repeated every seven days, or less frequently. The methods stimulate mitosis of neural progenitor cells, stimulate neurite growth and organization, protect against neural loss, or one or more of these neural processes.
Resumen de: US20260034121A1
Provided herein are compounds, compositions, and methods for inhibiting fibril growth. Compositions include at least one alpha-Synuclein (aSyn) inhibiting agent in the form of a compound including a dimethyoxyphenyl piperazine group. Methods include inhibiting aSyn fibril growth and treating a neurodegenerative disease in a subject in need thereof, including Parkinson's Disease and Lewy Body disease (LBD). Methods including administering a composition of the present disclosure. Further provided is a system for detecting aSyn fibril growth in a subject in need thereof, the system including a fluorescence screening assay of the present disclosure.
Resumen de: WO2026030590A1
The present disclosure provides recombinantly manufactured fusion proteins comprising a HERV-K (HML-2) Env protein fragment or an analog thereof linked to a human Fc fragment. Embodiments include the administration of the fusion proteins to patients having a disease or a disorder with the intention of mitigating and/or reducing the duration of symptoms associated with the condition or disease (for example but not limited to muscular weakness, paralysis and respiratory failure), and/or preventing symptoms associated with the condition or disease, for example, by preventing motor neuron degeneration and cell death in ALS patients associated with the condition or disease. Accordingly, "treatment" generally means both therapeutic treatment and prophylactic or preventative measures. Improvement after treatment may be manifested as a decrease or elimination of such symptoms, e.g., by a decrease or elimination of symptoms associated with ALS, and/or by a decrease in the duration of such symptoms.
Resumen de: WO2026030341A1
The present disclosure provides a method of treating or inducing cognitive preservation in a patient with Alzheimer's disease, comprising administering an effective amount of CT1812. The effective amount of CT1812 is 100 mg or 300 mg administered orally once daily. The method demonstrates improvements in cognitive outcomes compared to placebo across multiple measures including ADAS-Cog11, MMSE, ADCS-ADL, and ADCS-CGIC. The method demonstrates improvements in cognitive outcomes in patients with low plasma levels of phosphorylated tau 217 prior to administration.
Resumen de: WO2026028189A1
The present invention relates to a mixture, compositions comprising said mixture, and their use in the treatment and/or prevention of neurodegenerative diseases affecting motor neurons and/or due to demyelination processes, such as Amyotrophic Lateral Sclerosis (ALS) and Multiple Sclerosis (MS). In greater detail, the present invention relates to a mixture comprising or, alternatively, consisting of: i) an extract of chamomile; ii) an extract of turmeric; iii) L-acetyl carnitine or a salt thereof; iv) a blueberry extract; and v) lipoic acid, or a salt thereof, and/or a bergamot extract.
Resumen de: WO2026028165A1
The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a C9Orf72 gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a C9Orf72 gene in a subject, or for treatment of amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD).
Resumen de: US20260035387A1
The present disclosure provides a compound of Formula (I′), or a pharmaceutically acceptable salt thereof and its use in, e.g. treating a condition, disease, or disorder in which lowering mutant huntingtin protein (“mHTT”) in a subject is of therapeutic benefit, specifically in treating Huntington disease (“HD”). This disclosure also features a composition containing the same as well as methods of using and making the same.
Resumen de: WO2026027658A1
The invention refers to the field of neurodegenerative diseases including Alzheimer's Disease and a treatment thereof with a Benzoxazepine compound. Benzoxazepine compounds are e.g. known in the treatment of breast cancer and according to the use of the invention are a first therapeutic and prophylactic treatment for neurodegenerative diseases, including Alzheimer's Disease. The invention further relates to test systems for identifying, mapping, elaborating and evaluating said and further therapeutic and prophylactic uses of compounds of interest and/or other pharmaceutical compositions for the treatment of neurodegenerative diseases, including Alzheimer's Disease. (AD)
Resumen de: EP4686476A1
The invention refers to the field of neurodegenerative diseases including Alzheimer's Disease and a treatment thereof with a Benzoxazepine compound. Benzoxazepine compounds are e.g. known in the treatment of breast cancer and according to the use of the invention are a first therapeutic and prophylactic treatment for neurodegenerative diseases, including Alzheimer's Disease.The invention further relates to test systems for identifying, mapping, elaborating and evaluating said and further therapeutic and prophylactic uses of compounds of interest and/or other pharmaceutical compositions for the treatment of neurodegenerative diseases, including Alzheimer's Disease. (AD)
Resumen de: NZ812405A
A levodopa derivative including a compound or pharmaceutically acceptable salt, hydrate, and/or solvate thereof, wherein the compound includes substituents which, in aggregate, contain at least 6 carbon atoms which are only bonded to either other carbon atoms or to hydrogen atoms. The levodopa derivative may be formulated as a composition including one or more pharmaceutically acceptable carriers or excipients. The levodopa derivative may be part of a pharmaceutical composition including micro or nano particles in which the levodopa derivative is encapsulated in the pharmaceutically acceptable polymer. The levodopa derivative can be used to treat Parkinson’s disease by administering to a mammal an amount sufficient to treat Parkinson’s disease.
Resumen de: AU2024307361A1
Described herein are crystalline forms of 6-(6-(((1R,2R,3S,5S)-2-fluoro-9-azabicyclo3.3.1nonan-3-yl)(methyl)amino)pyridazin-3-yl)-2-methylbenzodoxazol-5-ol (compound A), a small molecule splicing modulator (SMSM) of mRNA, such as pre-mRNA, encoded by genes, for the treatment of Huntington's disease.
Resumen de: NZ758484A
The present disclosure relates to novel compounds, pharmaceutical compositions containing the compounds and methods of using the compounds and pharmaceutical compositions for treating neurodegerative diseases, including Alzheimer’s disease and cognitive decline. Methods for inhibiting synapse number decline or membrane trafficking abnormalities associated with exposure of a neuronal cell to Abeta species are also disclosed.
Nº publicación: NZ799802A 30/01/2026
Solicitante:
COGNITION THERAPEUTICS INC
COGNITION THERAPEUTICS INC
Resumen de: NZ799802A
The present disclosure relates to novel compounds, pharmaceutical compositions containing the compounds and methods of using the compounds and pharmaceutical compositions for treating neurodegerative diseases, including Alzheimer’s disease and cognitive decline. Methods for inhibiting synapse number decline or membrane trafficking abnormalities associated with exposure of a neuronal cell to Abeta species are also disclosed.
BOPI
Sede Electrónica
