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158
resultados
Última actualización
30/11/2025 [07:23:00]
Resumen de: WO2025245500A1
The invention described herein provides eIF2B agonist (e.g., COMPOUND 1 ) and uses thereof for treatment of diseases, especially neurodegenerative diseases, including vanishing white matter (VWM), Huntington's disease (HD), Charcot Marie Tooth syndrome (CMT), and Amyotropic Lateral Sclerosis (ALS).
Resumen de: US2025361252A1
The present invention relates to a compound of formula (I), which is a phosphate ester of apomorphine, or a pharmaceutically acceptable salt thereof. The apomorphine phosphate ester according to the invention exhibits remarkably advantageous properties as a therapeutic, including a favorable tolerability, an improved side effect profile, particularly a reduced occurrence of skin nodule formation and panniculitis when administered subcutaneously, as well as pharmacokinetic and metabolic properties rendering it particularly well-suited as an apomorphine prodrug. The invention further relates to the compound of formula (I) for use as a medicament, particularly for use in the treatment of Parkinson's disease.
Resumen de: US2025361292A1
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: US2025361237A1
The present invention relates to compounds of formula (I) that are capable of modulating, e.g., inhibiting or activating, one or more kinases, especially LRRK2 and/or NUAK1 and/or TYK2 or mutants thereof. The compounds are useful for treating diseases, such as autoimmune diseases, inflammatory diseases, bone diseases, metabolic diseases, neurological and neurodegenerative diseases, cancer, cardiovascular diseases, allergies, asthma, Alzheimer's disease, Parkinson's disease, skin disorders, eye diseases, infectious diseases and hormone-related diseases. The present description discloses the synthesis and characterisation of exemplary compounds as well as pharmacological data thereof (e.g. pages 40 to 146; examples 1 to 63; compounds 1 to 248; tables 1 to 3). Preferred compounds are e.g. 1,4-dihydrobenzodpyrazolo3,4-f1,3diazepine derivatives and related compounds. An exemplary compound is e.g. 5-(2,6-difluorophenyl)-8-methoxy-1,4-dihydrobenzodpyrazolo3,4-f1,3diazepine (example 49). (Formula (II):
Resumen de: WO2025243064A1
The main problem with treating Alzheimer' s is that there is no certain medicine for its treatment, or that there is medical resistance to it in the patient's body. By using the WJ-MSCs exosomes from the steam cells grade, the exosomes are extracted with the ultracentrifuge method and confirmed in the size of SEM and TEM exosomes in the range of less than 100nm. then the mir-299-5p is transfected to the cell WJ- MSCs, with the help of a vector, and the exosomes are then extracted, gathered, and formulated. Since the vesicles are under 100 nanometers (exo som es) and could enter the cell by passing the brain blood barrier, what is inside them is easily transferred into the neuronic cell and treats them. Also, it decreases the drug resistance in the body, and the existence of the mir-299-5p is targeted, has pharmaceutical effects, and recovers the neuronic cells that were injured.
Resumen de: AU2023449890A1
Opicapone for use as adjunctive therapy to preparations of levodopa and a DOPA decarboxylase inhibitor (DDCI) in the treatment of Parkinson's disease; characterised in that a patient with Parkinson's disease is treatable with preparations of levodopa and a DDCI without clinically diagnosed motor complications.
Resumen de: AU2024250257A1
Disclosed is a method for the treatment of a neurological or movement disorder, e.g., Parkinson's disease, in a patient in need thereof, by parenteral administration of levodopa and a dopa decarboxylase inhibitor (DDCI), such as carbidopa, benserazide or any combination thereof.
Resumen de: US2025360121A1
A method of treating a human patient afflicted with Huntington's disease, comprising periodically orally administering to the patient a pharmaceutical composition comprising pridopidine, its analog or a pharmaceutically acceptable salt thereof.
Resumen de: WO2025243901A1
Provided is an injection preparation for the treatment of Parkinson's disease or restless legs syndrome, the injection preparation comprising microparticles each containing a free dopamine receptor agonist and a biodegradable polymer. The volume average particle diameter D50 of the microparticles is 50 μm or less, and the concentration of the dopamine receptor agonist in plasma of a subject to which the injection preparation has been administered is retained for 1 month or longer.
Resumen de: WO2025240956A1
Described is small molecule 6-methoxy-7-(3-(pyrrolidin-1-yl)propoxy)-4-(tetrahydro-2H-pyran-4-yl)quinolin-2-amine (MS152) that inhibits methyltransferases G9a/EHMT2. This inhibitor can be used for the treatment of patients with G9a/EHMT2 related diseases such as Prader-Willi Syndrome and Alzheimer's Disease. Formula (I).
Resumen de: US2025354188A1
The present invention provides a method of synthesizing celosianin II, a method of synthesizing a betaxanthin, an amyloid-β polymerization inhibitor or a therapeutic or preventive agent for Alzheimer's, an amyloid peptide aggregation inhibitor, and an HIV-1 protease activity inhibitor. A gene having a celosianin II synthesis ability has been isolated from quinoa, and a method of synthesizing celosianin II of the present invention has been constructed. Besides, it has been recognized that celosianin II or the like serves as an active ingredient of each of an amyloid-β polymerization inhibitor or a therapeutic or preventive agent for Alzheimer's, an amyloid peptide aggregation inhibitor, and an HIV-1 protease activity inhibitor.
Resumen de: US2025353904A1
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: US2025353841A1
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: US2025352533A1
A method of treating a human patient afflicted with Huntington's disease, comprising periodically orally administering to the patient a pharmaceutical composition comprising pridopidine, its analog or a pharmaceutically acceptable salt thereof.
Resumen de: WO2025239904A1
The application relates to monoclonal antibody clone 1A11, which is highly specific for a C-terminal sequence of human alpha-synuclein (hαSyn) containing three nitrated tyrosines (3NYαSyn) and does not bind to the corresponding unmodified sequence. A CAR specific for 3NYαSyn has been produced, comprising the antigen-recognizing site of the mAb 1A11 fused to an intracellular region containing the signaling motifs of CD3zeta and CD28. Tregs expressing said anti-3NYαSyn CAR have been shown to attenuate neurodegeneration and neuroinflammation in a mouse model for Parkinson's disease. The T reg-response specific for 3YNαSyn exerts a therapeutic effect attenuating the loss of dopaminergic neurons in a preclinical model and represents a promising therapeutic strategy to treat Parkinson's disease patients..
Resumen de: US2025352509A1
Compositions and methods for treating amyotrophic lateral sclerosis. A method of treating amyotrophic lateral sclerosis comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid. Also disclosed are methods of reducing, slowing or abating the progression of amyotrophic lateral sclerosis or a symptom thereof, comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid.
Resumen de: WO2025240469A1
The invention provides, in some embodiments, a potency assay matrix for assessing potency of dopaminergic neuronal progenitor cells (DANPCs) that are intended for use in treating a neurodegenerative disease such as Parkinson's Disease. The potency assay matrix can include a bioassay for L-DOPA decarboxylase (DDC) activity. In addition to the DDC activity assay matrix, the potency assay matrix may also include one or more additional mechanism of action-based assays such as bioinformatics-based assays for determining whether the DANPCs are dopaminergic neuronal progenitor cells, for determining whether the DANPCs are likely to engraft when implanted into a subject brain, and for determining whether neuronal cells that are derived from the DANPCs are likely to produce dopamine after implantation.
Resumen de: WO2025239588A1
The present invention relates to: a fusion protein comprising a mitochondrial outer membrane anchoring peptide and a cerebrovascular endothelial cell surface protein binding site; modified mitochondria to which the fusion protein is bound; and a pharmaceutical composition comprising the modified mitochondria as an active ingredient. The modified mitochondria comprising the cerebrovascular endothelial cell surface protein binding site according to the present invention can pass through the blood-brain barrier at the cellular and animal level. In addition, administering the modified mitochondria to a Parkinson's disease mouse model can alleviate movement disorders. Therefore, the modified mitochondria according to the present invention can be used as a therapeutic agent for brain diseases caused by mitochondrial dysfunction.
Resumen de: US2025352609A1
Disclosed herein are methods of treating or preventing Alzheimer's disease or dementia and concurrently treating hyperparathyrodism by administering a blood-brain barrier (BBB)-impermeable calcimimetic and/or a BBB-permeable calcilytic along with the administration of anti-amyloid-beta therapies. Also disclosed herein are methods of increasing calcium-sensing receptor (CaSR) homodimer formation, increasing expression or activity of the CaSR homodimer and reducing CaSR/GABA-B1 receptor heterodimer formation, reducing expression or activity of the CaSR/GABA-B1 receptor heterodimer in both peripheral tissues and central nervous system (CNS) in a subject.
Resumen de: US2025352494A1
Disclosed herein are patches, methods, devices, and systems for delivering a non-aggregating peptide, such as alcadein and its fragments, into a subject. In some aspects, the patch includes a backing, a matrix comprising a non-aggregating peptides disposed within the matrix, and a release liner. In other aspects, the method includes opening at least one channel in the subject's skin, applying the patch described herein, thereby treating a disease or disorder associated with the brain, such as Alzheimer's disease.
Resumen de: US2024310389A1
This document provides methods and materials related to screening for and treating Alzheimer's disease (AD), including late-onset Alzheimer's disease (LOAD).
Resumen de: AU2025256148A1
The present disclosure relates to compositions of levodopa 4’-monophosphate and carbidopa 4’-monophosphate having a weight by weight ratio of about 20:1 and methods of treating Parkinson's disease and associated conditions by subcutaneous administration of such compositions. The present disclosure relates to compositions of levodopa 4'-monophosphate and carbidopa 4'-monophosphate having a weight by weight ratio of about 20:1 and methods of treating Parkinson's disease and associated conditions by subcutaneous administration of such compositions. ct c t
Resumen de: AU2024283314A1
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: AU2024271182A1
The present invention relates to the use of the HIP/PAP protein, or a derivative thereof, in the treatment and prevention, and in particular in the treatment, of a cognitive disorder associated with anxiety disorder(s) in an individual in need thereof, as well as for improving cognition in an individual affected by a neurological disorder associated with anxiety disorder(s) or for alleviating cognitive deficit in an individual affected by a disorder selected from the group consisting of Obsessive-compulsive disorder, Attention deficit disorder, Dementia with Lewy bodies disease, Early onset dementia, Epilepsy-related cognitive dysfunction, Fronto-temporal dementia, Posterior cortical atrophy, Huntington's disease (HD), Parkinson's disease, bipolar disorder, substance abuse, attention deficit disorders, psychotic disorders and a sars-cov-2 infection, and also in the prevention and/or treatment of diet induced cognitive and anxiety deficits in an individual in need thereof, and in particular high fat diet induced cognitive and anxiety deficits.
Nº publicación: AU2024267789A1 13/11/2025
Solicitante:
UNIV OF PITTSBURGH OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION,
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Resumen de: AU2024267789A1
Provided are heterocyclic compounds of Formula I or Formula II or their pharmaceutically pharmaceutically acceptable salts, solvates, hydrates, tautomers or stereoisomers for imaging tau aggregates. Compounds of Formula I or Formula II may be used for the detection of tau aggregates in the diagnosis or monitoring of the progression of a disease or disorder such as Alzheimer's disease, corticobasal degeneration and progressive supranuclear palsy.
BOPI
Sede Electrónica
