Resumen de: MX2025013613A
Disclosed are methods of treating amyotrophic lateral sclerosis (ALS). Also disclosed are methods of treating C9orf72 amyotrophic lateral sclerosis (C9-ALS).
Resumen de: MX2025007688A
Compounds, compositions, uses, and methods for increasing cell viability of a dopaminergic neuron, or for preventing or treating dopaminergic neuronal death, are provided herein. In certain examples, methods for reducing symptoms and/or for preventing or treating Parkinson's disease in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1a<sup>GDP</sup>), one or more expressible nucleic acids encoding Rab1a<sup>GDP</sup>, or a combination thereof.
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: CN121428061A
The invention belongs to the technical field of medicines, and particularly relates to a group of butyrylcholine esterase (BChE) inhibitors with high selectivity and a screening method thereof, in particular to two BChE specific inhibitors, namely methyl 4-hydroxybenzyl carbamate and moringa seed extract, which are screened from a moringa seed extract. The invention also relates to application of the BChE specific inhibitor in preparation of drugs for preventing or treating Alzheimer's disease. The invention provides a brand new strategy for efficiently screening the anti-Alzheimer disease medicine.
Resumen de: CN121422231A
The invention discloses a multi-target compound preparation for targeted neuron autophagy repair as well as a preparation method and application thereof, and belongs to the technical field of neuropharmacology and pharmaceutical preparations. The compound preparation comprises an mTOR inhibitor Torrin 1, a lysosome function enhancer ursodesoxycholic acid, a neuroprotective agent coenzyme Q10 and alpha-lipoic acid according to a mass ratio of 1: 10: 5, and by synergistically adjusting autophagy initiation, autophagosome-lysosome fusion and neuron metabolism homeostasis, systematically repairing autophagy flux, efficiently removing beta amyloid protein and tau protein aggregates, and improving the autophagy efficiency. The mitochondrial function is improved. Sustained release is realized by adopting a sustained release microsphere technology, and the drug effect can be maintained for 14 days. Animal experiments show that protein aggregates in brains of mice suffering from the Alzheimer disease can be reduced by 70% or above, the cognitive function is remarkably improved, the safety is good, and a new choice is provided for treatment of neurodegenerative diseases.
Resumen de: CN121443318A
The present invention provides a method of treating a neurodegenerative disease. The method comprises the step of administering to a subject in need thereof an effective amount of a polymer-flavonoid conjugate or nanocomposite, the nanocomposites have an outer shell comprising one or more polymer-flavonoid conjugates and, optionally, an inner shell comprising one or more flavonoid oligomers, as well as drugs, such as anti-CD3 or anti-CD33, encapsulated within these shells. The methods of the invention allow therapeutically effective substances to pass through the blood-brain barrier to treat neurodegenerative diseases. The methods of the invention are effective in the treatment of neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease, dementia with Lewy bodies, and Huntington's disease.
Resumen de: NZ792513A
Aspects of the disclosure relate to compositions and methods useful for treating Huntington’s disease. In particular, the disclosure provides interfering nucleic acids (e.g., artificial mature miRNAs flanked by a miR-155 or a miR-30 backbone sequence) targeting the huntingtin gene (HTT) and methods of treating Huntington’s disease using the same.
Resumen de: CN121422010A
The invention relates to the field of natural product pharmacy, in particular to an acetylcholin esterase inhibitor sourced from a natural product and application of the acetylcholin esterase inhibitor. Although a chemical synthetic acetylcholin esterase inhibitor used in clinical treatment at present achieves a certain curative effect, the chemical synthetic acetylcholin esterase inhibitor still has the problems of side effects, drug resistance and the like. Therefore, the invention provides the acetylcholin esterase inhibitor, the inhibitor is a single-drug or double-drug composition derived from natural products, the single drug is grifolin isoflavone (TI) or isoeugenol acetate (IA), and the double drug is prepared from TI and IA in proportion. The acetylcholin esterase inhibitor disclosed by the invention has good AChE inhibitory activity, anti-Abeta aggregation effect, antioxidant activity and good neuroprotection effect, and the TI and/or IA can be used as a novel acetylcholin esterase inhibitor to be applied to the development and use of medicines for clinically treating and/or preventing related diseases such as Alzheimer'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.
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.
Resumen de: US20260029411A1
The invention provides methods and compositions for accurate identification and determination of Parkinson's disease ante-mortem tissue samples. The determination of Parkinson's disease is based on the binding of localized phosphorylated alpha-synuclein with the nerve feature. The methods disclosed in the invention may be used on myriad tissue types and could be manual or automated.
Resumen de: US20260028347A1
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
Resumen de: US20260028625A1
The present disclosure relates to an sgRNA and its application in the preparation of a product for the treatment of Huntington's disease. The present disclosure was designed and screened to obtain an sgRNA targeting exon 1 of the human HTT gene as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The CRISPR/Cas9 system mediated HTT gene knockout strategy based on this sgRNA and its high homologue sgRNA can efficiently knock out the human Huntingtin gene and achieve gene therapy for Huntington's disease.
Resumen de: US20260028623A1
Provided are RNAi agents, pharmaceutical compositions, and methods for reducing the amount or activity of PRNP RNA in a cell or a subject, and in certain instances reducing the amount of prion protein in a cell or a subject. Such RNAi agents, pharmaceutical compositions, and methods are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such neurodegenerative diseases include prion diseases, such as Creutzfeldt-Jakob disease (CJD) (e.g., variant Creutzfeldt-Jakob Disease (vCJD), classic Creutzfeldt-Jakob Disease (cCJD), familial Creutzfeldt-Jakob Disease (fCJD), or sporadic Creutzfeldt-Jakob Disease (sCJD)), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, or kuru; synucleinopathies such as Alzheimer's disease, Parkinson's disease, or dementia with Lewy bodies; or tauopathies such as frontal temporal dementia associated with a Tau mutation, Pick's disease, progressive supranuclear palsy, corticobasal neurodegeneration, or chronic traumatic encephalopathy (CTE).
Resumen de: US20260027234A1
The present disclosure provides recombinant vectors encoding a choline acetyltransferase (ChAT) polypeptide, compositions thereof, and methods of use thereof.
Resumen de: US20260027178A1
The present disclosure generally relates to the treatment of subjects having Parkinson's disease or at risk of developing Parkinson's disease by administering a SIM BHLH Transcription Factor 2 (SIM2) agonist to the subject.
Resumen de: US20260027179A1
Disclosed is a method of treating a neurodegenerative disease such as Parkinson's disease, diffuse Lewy body disease, transitional Lewy body dementia, and multiple system atrophy in a subject. The method comprises administering to the subject a therapeutically effective amount of a peptide comprising an α-synuclein binding domain operably linked to a protein transduction domain and a proteasomal targeting domain, wherein the α-synuclein binding domain is derived from a reversed sequence of β-synuclein. Other methods, as well as uses and compositions, are disclosed.
Resumen de: WO2026022191A1
The present invention relates to the field of neurodegenerative diseases, in particular Alzheimer's disease. The present invention further relates to fibrillary Apolipoprotein E (ApoE) for use in a method of treatment and/or prevention of a neurodegenerative disease and methods of producing said fibrillary ApoE. Moreover, the present invention relates to an antigen-binding peptide specifically binding to fibrillary ApoE, preferably human ApoE, a method of generating said antigen-binding peptide, and its use in a method of treatment and/or prevention and/or diagnosis of a neurodegenerative disease in a patient in need thereof.
Resumen de: WO2026021131A1
Disclosed are an aromatic alkylamine ferroptosis inhibitor based on a butylphthalide structure, a preparation method therefor, and an application thereof. The chemical structural formula of the ferroptosis inhibitor is as shown in formula (1) or formula (2). The ferroptosis inhibitor is capable of inhibiting ferroptosis caused by a ferroptosis inducer, and reduces the level of intracellular reactive oxygen species. The ferroptosis inhibitor reduces neurological damage caused by cerebral ischemia-reperfusion, and alleviates symptoms of neurological disorders such as Alzheimer's disease and Parkinson's disease. Compared to the ferroptosis inhibitor Ferrostatin-1, the arylalkylamine compound exhibits better metabolic stability and is suitable for in-vivo efficacy evaluation. Therefore, the novel arylalkylamine compound provided by the present invention demonstrates great application value in the treatment of ferroptosis-related neurological disorders.
Resumen de: WO2026021591A1
The present application relates to a novel micropeptide MP29 and a use thereof and relates to a use of the micropeptide in the preparation of a reagent or drug for preventing, treating, or alleviating diseases related to abnormal mitochondrial energy metabolism in cells, wherein the diseases include Alzheimer's disease, Parkinson's disease, Huntington's disease, schizophrenia, aging, photoaging, fatty liver disease, liver fibrosis, liver cirrhosis, liver cancer, diabetic nephropathy, cardiovascular diseases such as heart failure, etc. By means of endogenous overexpression or exogenous synthesis, the reducing equivalents NADH in the tricarboxylic acid cycle are up-regulated to promote intracellular ATP production, thereby significantly enhancing the proliferation of high-energy-demanding cardiac cells and brain tissue cells, or suppressing oxidative damage and apoptosis of cells in Alzheimer's disease models and Parkinsonism, or alleviating hypertrophy and aging of myocardial cells in heart failure models, or ameliorating the photodamage of cells in photoaging models. These results indicate that the micropeptide MP29 has an application value in preventing or treating diseases related to abnormal mitochondrial energy metabolism.
Resumen de: WO2026024717A1
Provided herein are methods and compositions for treating neurological diseases (e.g., neurodegenerative disorders (e.g., Alzheimer's disease, Parkinson's disease, dementia, a tauopathy, chronic traumatic encephalopathy (CTE), traumatic brain injury (TBI), mild cognitive impairment); psychiatric disorders (e.g., bipolar disorder, schizophrenia, depression, anxiety, post-traumatic stress disorder, obsessive compulsive disorder); inflammation in the central nervous system) using lithium salts, activators that increase the expression or activity of the RE1 silencing transcription factor (REST), or a combination thereof, or in combination with an additional agent.
Resumen de: WO2026024812A1
The present disclosure provides substituted 2,3,4,5-tetrahydro-1H-benzo e 1,4 diazepin-l-yl compounds, pharmaceutically acceptable salts thereof, or a pharmaceutical composition thereof, utilized in the manufacture of a medicament for the inhibition of F-ATP hydrolase and for treating diseases, disorders or conditions associated with F-ATP hydrolase, including Alzheimer's disease, Parkinson's Disease, amyotrophic lateral sclerosis, Friedreich's ataxia and cancer.
Resumen de: WO2026024121A1
The present disclosure relates to a bicyclic fused-ring (for example, pteridine, pyridopyrimidine, pyridopyrazine, quinazoline, naphthyridine, or quinoxaline) derivative or a pharmaceutically acceptable salt thereof inducing activation of a triggering receptor expressed on myeloid cells 2 (TREM2), a pharmaceutical composition including the same, and a use thereof. The bicyclic fused-ring derivative or a pharmaceutically acceptable salt thereof may be useful in treatment and prevention of TREM2-mediated neurodegenerative diseases such as Alzheimer's disease (AD), frontotemporal lobar degeneration (FTLD), FTLD-like syndrome, Parkinson's disease, Huntington's disease and Nasu-Hakola disease.
Nº publicación: WO2026024901A1 29/01/2026
Solicitante:
VIGIL NEUROSCIENCE INC [US]
VIGIL NEUROSCIENCE, INC
Resumen de: WO2026024901A1
The present disclosure provides methods of using a small molecule TREM2 agonist or a pharmaceutically acceptable salt thereof to treat Alzheimer's Disease in a human subject.