Resumen de: US20260116932A1
Disclosed herein are improved methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. Among other things, provided herein is a gene therapy construct encoding a non-naturally occurring codon-optimized transcription factor (ZFP-TF) comprising a zinc-finger protein (ZFP) sequence and a sequence encoding a transcriptional repression domain, wherein the ZFP-TF expression is driven by a phosphoglycerate kinase 1 (PGK), ubiquitin C (UBC), an EFS, or an EF1alpha promoter.
Resumen de: US20260117234A1
0000 A gene sequence construct used for the treatment of central nervous system diseases: by means of the construction of an auto-processing expression vector, tyrosine hydroxylase (TH), GTP-cyclohydrolase I (GCH1), aromatic amino acid dopa decarboxylase (AADC), and so on may be simultaneously expressed; proteins are connected by means of an auto-processing unit (APU); the use of a viral vector to introduce the construct into a target cell may ultimately result in the high-efficiency expression of tyrosine hydroxylase (TH), GTP-cyclohydrolase I (GCH1), aromatic amino acid dopa decarboxylase (AADC), and so on having independent functions, being used in the prevention or treatment of Parkinson's disease, Alzheimer's disease and other neurodegenerative diseases.
Resumen de: US20260115318A1
The present invention relates to: a gene editing composition comprising a guide RNA for editing mutated CAG repeat sequences of neural stem cells (ciNSCs) induced from somatic cells of a Huntington's disease patient; the edited ciNSCs; and a cell therapeutic agent for Huntington's disease, comprising same, wherein, in the edited ciNSCs, Huntington gene mutations are removed by the guide RNA of the present invention, and thus treatment for Huntington's disease and neuronal proliferation can be improved.
Resumen de: WO2026090552A1
Methods and compositions are provided for treating Alzheimer's disease by administering interleukin-33 (IL-33) to a subject. Methods of gene therapy are also provided for expressing IL-33 in vivo in the brain of a subject in effective amounts sufficient to treat amyloid plaque pathology and reduce numbers of plaques in the brain.
Resumen de: WO2026090369A1
A composition and method of using the composition to treat neurological disorders including Alzheimer's disease are disclosed wherein the composition includes an elastin-like polypeptide (ELP) and a drug. The ELP undergoes a transition from a solution phase to a gel phase as the temperature is raised above a transition temperature, and in doing so sequesters at least a portion of the drug into the gel phase. The composition is configured such that intranasal delivery of the composition results in gelation and sequestration of the drug by the ELP, thereby allowing the drug to traverse olfactory mucosa into brain tissue in order to treat neurological disorders, including Alzheimer's disease.
Resumen de: WO2026088070A1
The present invention relates to novel compounds and pharmaceutical compositions. More particularly, the present invention provides novel compounds which are useful for the treatment of and prevention of neurodegenerative disorders including, but not limited to, Alzheimer's Disease (AD), Parkinson's Disease (PD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and Nasu-Hakola Disease (NHD).
Resumen de: US20260115160A1
The present invention provides methods of providing rapid relief of motor fluctuations in a Parkinson's disease patient. The methods of the invention comprise pulmonary administration of levodopa by inhalation at therapeutically effective concentrations such that the patient's plasma levodopa concentration increases by at least about 200 ng/ml within 10 minutes or less post inhalation as compared to the concentration of levodopa in the patient's plasma prior to inhalation of the levodopa and wherein the patient's plasma concentration remains increased by at least about 200 ng/ml for a time period of at least 15 minutes after inhalation. The methods of the invention are particularly useful for treatment of motor fluctuations which arise as a result of poorly controlled levodopa plasma levels in a patient.
Resumen de: US20260116878A1
Indazole compounds for treating various diseases and pathologies are disclosed. More particularly, the present invention concerns the use of an indazole compound or analogs thereof, in the treatment of disorders characterized by the activation of Wnt pathway signaling (e.g., cancer, abnormal cellular proliferation, angiogenesis, Alzheimer's disease, lung disease and osteoarthritis), the modulation of cellular events mediated by Wnt pathway signaling, as well as genetic diseases and neurological conditions/disorders/diseases due to mutations or dysregulation of the Wnt pathway and/or of one or more of Wnt signaling components. Also provided are methods for treating Wnt-related disease states.
Resumen de: WO2026088136A1
The present disclosure provides a method of treating a neurodegenerative disease such as Alzheimer's disease in a subject. The method includes administering to the subject about 30 mg of AR1001 orally once daily, or a pharmaceutically acceptable salt thereof, and monitoring plasma phosphorylated tau 181 (pTau181) or phosphorylated tau 217 (pTau217) levels in the subject during treatment. The method may further include continuing AR1001 treatment if the subject's plasma pTau181 or pTau217 levels decrease during treatment. The subject may have mild cognitive impairment due to Alzheimer's disease or mild Alzheimer's disease dementia. The method may also include assessing the subject's cognitive function using one or more standardized tests during treatment. AR1001 may be administered in a pharmaceutical composition in the form of a tablet or capsule.
Resumen de: WO2026087399A1
The invention relates to a compound of formula (I) that reduce the protein level of huntingtin (HTT) and which are useful in the treatment of Huntington's disease.
Resumen de: WO2026086833A1
Disclosed in the present invention are a protein for inhibiting the conserved helix of TDP-43 and the use thereof. The protein contains an amino acid sequence as shown in SEQ ID NO: 1 or SEQ ID NO: 2. Further disclosed in the present invention are the use of the protein in the preparation of a drug for diagnosing, preventing and/or treating neurodegenerative diseases, and the use in the preparation of an inhibitor of a target protein containing the conserved helix of TDP-43 LCD. In the present invention, by means of using artificial intelligence-assisted protein design techniques, a high-affinity non-natural protein that precisely binds to the conserved helix of a TDP-43 protein is designed, which is used for inhibiting the participation of the conserved helix region in the formation of a β-sheet aggregation core, is mainly used in the treatment of diseases targeting the TDP-43 protein, such as amyotrophic lateral sclerosis, and is used as a research tool for TDP-43 protein phase separation experiments.
Resumen de: WO2024261329A1
The present invention relates to a compound of formula (I) or a pharmaceutically acceptable salt and/or solvate thereof, wherein Y1 is a 9- or 10-membered bicyclic heteroaryl, Y2 is a 5- or 6-membered heteroaryl or 6-membered aryl, L is a linker, Z1' is selected from -NR23' R24' and -(C3-C7) heterocyclo alkyl comprising at least one nitrogen atom, and R23' and R24' may be various groups. The present invention further relates to a compound of formula (I) as HDAC6 inhibitor, typically for use in the treatment and/or the prevention of an HDAC6-associated disease, such as cancers, neurodegenerative diseases, neuropathies, cardiovascular diseases, or metabolic or hormonal disorders.
Resumen de: AU2024342582A1
The present disclosure provides antibodies that specifically bind to human TDP-43 and methods of using these antibodies to treat patients with TDP-43-related diseases, including Amyotrophic Lateral Sclerosis (ALS).
Resumen de: US20260108494A1
0000 The purpose of the present invention is to provide: a therapeutic agent or a recurrence preventive agent for serotonergic system- or dopaminergic system-related diseases, in particular, mental dysfunction symptoms and other non-motor symptoms of Parkinson's disease; and a method for treating the aforesaid symptoms or preventing the recurrence of the same. A compound represented by formula (1) wherein each symbol is as defined in the description or a pharmaceutically acceptable salt thereof can exhibit an effect of treating serotonergic system- or dopaminergic system-related diseases, in particular, mental dysfunction symptoms and other non-motor symptoms of Parkinson's disease and/or an effect of preventing the recurrence of the same.
0000
Resumen de: AU2024357688A1
This invention relates to the treatment of a neuropsychiatric disorder, such as schizophrenia or Parkinson's disease, by administration (for example, transdermally) of tilivapram, zatolmilast, roflumilast, or a pharmaceutically acceptable salt thereof.
Resumen de: US20260108568A1
0000 Provided are a Bifidobacterium longum subsp. infantis for alleviating Parkinson's disease and a use thereof, and the Bifidobacterium longum subsp. infantis for alleviating Parkinson's disease is named as Bifidobacterium longum subsp. infantis BI03 strain, with a deposit number of CGMCC No. 24473 and deposit date of Mar. 7, 2022. The strain can significantly alleviate the symptoms of Parkinson's disease, specifically manifested in: alleviating Parkinson's disease-related dyskinesia and corticosterone elevation; weakening the neuroinflammation associated with Parkinson's disease; promoting glutathione and weakening brain oxidative stress damage.
Resumen de: US20260108572A1
0000 The objective of the present invention is to provide an Alzheimer-type dementia suppressant that is safe and thus can be administered daily and utilized as a health food and by which Alzheimer-type dementia can be suppressed, and a method for producing the Alzheimer-type dementia suppressant. In addition, the objective of the present invention is also to provide a use of an aboveground part of Sesamum indicum or an extract thereof for suppressing Alzheimer-type dementia, and a method for suppressing Alzheimer-type dementia. The Alzheimer-type dementia suppressant according to the present invention is characterized in comprising an aboveground part of Sesamum indicum or an extract thereof as an active ingredient.
Resumen de: WO2026085054A1
The invention is directed to immunological compositions of one or more peptides containing epitopes of PGN, LTA and LPS molecules that induce an immunological response in a mammal, and to multiple antibodies that bind to these epitopes. Immunological compositions and antibodies disclosed herein can be used in the treatment and/or prevention of human health disorders such as bacterial sepsis, inflammation, cancers, tumors, inflammatory diseases and disorders, and neurodegenerative disorders such as, but not limited to Alzheimer's disease, frontotemporal dementia, chronic traumatic encephalopathy (CTE), Lewy body dementia and/or limbic predominant age-related TDP-43 encephalopathy (LATE).
Resumen de: WO2026082154A1
Deposition of N-terminally truncated and pyroglutamate-modified Abeta peptides (AβpE3) play an important role in the aggregation of the amyloid-β (Aβ) protein in the brain. The N-terminally truncated and pyroglutamate-modified Abeta peptides, therefore, can be a suitable target for the prevention and treatment of Alzheimer's Disease (AD). Disclosed herein are antibodies and fragments having excellent specificity to N-terminally truncated and pyroglutamate-modified Abeta peptides, as well as uses of these antibodies and fragments for the prevention and treatment of AD.
Resumen de: WO2026081146A1
A composition containing a brain protein hydrolysate, a preparation method, and a use. The composition comprises the following raw materials: porcine brain protein hydrolysate, deer brain protein hydrolysate, bovine collagen peptide powder, Gastrodia elata powder, sea buckthorn powder, hawthorn powder, chicken gizzard membrane powder, vitamin C, vitamin B1, vitamin B3, vitamin B5, vitamin B6, and vitamin B12. The composition has anti-inflammatory and antioxidant effects, and can also be used for preparing a medicament for preventing Alzheimer's disease.
Resumen de: WO2026085233A1
The disclosure relates to formulations of double stranded ribonucleic acid (dsRNAi) agents targeting a MAPT gene, as well as methods of inhibiting expression of a MAPT gene and methods of treating subjects having a MAPT-associated neurodegenerative disease or disorder, e.g., Alzheimer's disease, FTD, PSP, or other tauopathy, using such dsRNAi agent formulations.
Resumen de: WO2026085535A2
Disclosed is a method for treating for treating pathologies associated with Down syndrome (e.g., symptoms of Alzheimer's disease and metabolic abnormalities), preventing the onset of pathologies associated with Down syndrome in patients with Down syndrome, reducing cognitive or functional decline, or reducing symptom load in a subject having Down Syndrome, in need or at risk thereof, comprising administering to said subject a therapeutically effective amount of an anti-Follicle Stimulating Hormone (FSH) antibody, or an antigen-binding portion thereof.
Resumen de: WO2026082004A1
The present invention relates to a fused heteroaryl derivative, a preparation method therefor, and an application thereof. The fused heteroaryl derivative can be used in a sodium channel inhibitor, and/or in a GABA receptor agonist, or in treating neuropsychiatric disorders such as epilepsy, Parkinson's, schizophrenia, or depression.
Resumen de: DE102024130715A1
Die vorliegende Offenbarung bezieht sich auf eine pflanzliche Mundspüllösung, die Ruta graveolens und Saxifraga enthält und speziell zur Vorbeugung von neurologischen Erkrankungen wie Parkinson, Alzheimer, Lähmungen und Nervenschäden entwickelt wurde. Verfahren zur Herstellung der pflanzlichen Mundspüllösung durch Einweichen einer fein gemahlenen Mischung aus Ruta graveolens und Saxifraga in Alkohol. Die resultierende Lösung wird als Mundspülung verabreicht und zum Spülen und Gurgeln verwendet. Die Erfindung bietet eine wirksame, natürliche therapeutische Alternative zur Behandlung neurodegenerativer Erkrankungen, zur Förderung des Neuroschutzes und zur möglichen Unterstützung der Regeneration geschädigter Nervenzellen.
Nº publicación: WO2026082108A1 23/04/2026
Solicitante:
QINGDAO BORSON TAI TECH CO LTD [CN]
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Resumen de: WO2026082108A1
The present invention relates to use of an NLRP3 inhibitor for treating Parkinson's disease or Parkinsonism. Specifically, the present invention relates to use of a compound represented by formula (I) or a pharmaceutically acceptable salt thereof in the preparation of a drug for treating Parkinson's disease or Parkinsonism, wherein each substituent is as defined in the specification.