Absstract of: US20260256860A1
A traditional Chinese medicine composition for treating amyotrophic lateral sclerosis and the use thereof. The composition is prepared by means of combining Ginseng radix et rhizoma and Cistanches herba.
Absstract of: WO2026179739A1
Disclosed in the present invention are an aminothiazole compound and use thereof as a brain-permeable histamine H2 receptor agonist, which belong to the field of medicine. The aminothiazole compound provided in the present invention has a general structural formula represented by formula (I) and comprises a pharmaceutically acceptable salt. The aminothiazole compound of the present invention has histamine H2 receptor agonist activity and excellent blood-brain barrier permeability. Therefore, the aminothiazole compound can be used in the preparation of a medicament for treating schizophrenia, mania, attention deficit hyperactivity disorder, binge eating disorder or bulimia nervosa, autism, Parkinson's disease, ischemic brain injury, and other diseases.
Absstract of: US2025127867A1
0000 The application describes a phosphorylated tau targeted active immunotherapy to treat preclinical Alzheimer's Disease.
Absstract of: WO2025089370A1
Disclosed are pyrazolopyridine or pyrazolopyrimidine compounds, or salts thereof, with colony-stimulating factor-1 receptor (CSF1R) inhibitory activity, medical use thereof for treating, preventing, and/or diagnosing diseases associated with CSF1R, and methods of preparing said compounds, or salts thereof. Provided include a compound represented by Formula I, or a salt thereof, wherein R1 is hydrogen, etc.; R21 and R22 are hydrogen, or R21 and R22 together with the adjacent heterocyclic form a bridged bicyclic ring; R3 is -L31-R31 optionally substituted with one or more R32, or R31 optionally substituted with one or more R32; L31 is -C(=O)-, etc.; R31 is C1-6 alkyl, etc.; R32 is each independently halogen, etc.; and X is CR1 or N; medical use thereof, and methods of preparation thereof.
Absstract of: WO2025087971A1
The invention relates to the treatment of Alzheimer's disease in a human patient, said treatment comprising administration of an anti-Aβ antibody component and co-administration of edaravone, the anti-Aβ antibody component being selected from anti-Aβ antibody, an Aβ- binding fragment of an Aβ antibody, a vectorised anti-Aβ antibody and a vectorised Aβ- binding fragment of an Aβ antibody.
Absstract of: EP4799693A2
The present invention provides methods for treating OFF episodes in a Parkinson's Disease patient comprising administering levodopa to the pulmonary system of a patient wherein after administration, the patient's Unified Parkinson's Disease Rating Scale (UPDRS) Part 3 score is improved by, for example, at least about 5 points compared to placebo control and/or as compared to the patient's UDPRS Part 3 score prior to administration. The invention also provides methods of reducing mean daily OFF time in a Parkinson's patient.
Absstract of: US20260248883A1
0000 Disclosed is a pharmaceutical composition for preventing or treating a brain disease, containing as an active ingredient an ErbB3-binding protein 1 (EBP1) protein or a polynucleotide sequence encoding the EBP1 protein, wherein an EBP1 N84A/N204A protein, which is an EBP1 mutant in an asparagine endopeptidase (AEP)-uncleavable form, and a polynucleotide encoding the same can reduce amyloid beta production and enhance cognitive function in the early stages of Alzheimer's disease, and thus the composition can be advantageously used as a therapeutic agent for brain diseases including sporadic Alzheimer's disease.
Absstract of: US20260248941A1
0000 Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a microtubule associated protein tau (MAPT) gene. The MAPT RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a MAPT gene. The MAPT RNAi agents are conjugated to an antigen binding protein that may enable subcutaneous delivery of the RNAi agents by facilitating crossing of the blood brain barrier (BBB). Pharmaceutical compositions that include one or more MAPT RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MAPT RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of MAPT gene expression and a reduction in MAPT activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including Alzheimer's disease, Frontotemporal lobar degeneration dementia (FTLD), Progressive supranuclear palsy, and other tauopathies.
Absstract of: WO2026178556A1
Among other things, the present disclosure provides oligonucleotides, compositions, and methods useful for targeting HTT. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties and activities. In some embodiments, the present disclosure provides oligonucleotides, compositions and methods for reducing HTT levels. In some embodiments, the present disclosure provides oligonucleotides, compositions and methods for treating conditions, disorders or diseases such as Huntington's disease.
Absstract of: US20260250396A1
Provided herein are therapies involving the use of antibodies that bind to Gal-3. Such therapies can include, but are not limited to, disorders such as Alzheimer's disease. Such therapies can also include treatments focused on increasing a subject's test scores under a variety of metrics.
Absstract of: US20260250715A1
The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.
Absstract of: US20260248855A1
Use of a transforming growth factor beta 1 (TGF-β1)-overexpressing olfactory mucosa mesenchymal stem cell (OM-MSC) in preparation of a drug for preventing and/or treating Parkinson's disease (PD) is provided, belonging to the technical field of drug preparation. The TGF-β1-overexpressing OM-MSCs can be used to better conduct a neural repair treatment of the PD. By verifying effects of the TGF-β1-overexpressing OM-MSCs in a PD cell model and a PD animal model, it is proved that the TGF-β1-overexpressing OM-MSCs do have a therapeutic effect on PD.
Absstract of: US20260250256A1
0000 Disclosed are compounds of Formulas (I), (Ia), (Ib), (II), (IIa), (III), (IIIa), and (IIIb), as well as pharmaceutical compositions thereof. The compounds can be used to improve proteostasis and enhance clearance of protein accumulation events by positively modulating the autophagy-lysosomal pathway, including augmenting the activity of cathepsin enzymes, and/or to treat neurological diseases, disorders and conditions, such as, but not limited to, Alzheimer's disease, Parkinson's disease, Huntington's disease, mild cognitive impairment, frontotemporal dementia, amyotrophic lateral sclerosis, Lewy body dementias, chronic traumatic encephalopathy, traumatic brain injury, and α-synucleinopathies.
Absstract of: US20260248877A1
0000 The present invention provides inhibitory peptides for use in the diagnostic and/or treatment of tauopathies, in particular Alzheimer's Disease and Pick's Disease. The inhibitory peptides comprise a hexapeptide sequence that specifically inhibits interactions of the PHF6 sequence within pathological Tau protein.
Absstract of: EP4796178A2
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).
Absstract of: WO2025083630A1
The present invention relates to heteroaromatic compounds of formula (I), or an isotopic form, a stereoisomer, or a pharmaceutically acceptable salt thereof as muscarinic M4 receptor positive allosteric modulators (M4 PAMs). The present invention also relates to pharmaceutical compositions comprising such compounds, chemical processes of preparation of such compounds and use of such compounds in the treatment of psychiatric and/or neurological disorders.
Absstract of: WO2025085704A1
This disclosure relates to vectors, compositions, pharmaceutical compositions, and kits that provide for brain cell-specific expression of reprogramming genes such as the Yamanaka factors Oct4, Sox2, Klf4 and c-Myc (OSKM). Also provided are methods and uses comprising the same for treating Alzheimer' s disease and progeria through brain cell-specific expression of reprogramming genes such as OSKM.
Absstract of: US20260240851A1
0000 Methods for preventing, limiting or delaying clinical motor progression in a subject with Parkinson's disease with low GCase activity, such as a PD patient with a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD) is provided, said methods comprising administering a therapeutically effective amount of 5,7-dimethyl-N-((1R,4R)-4-(pentyloxy)cyclohexyl)pyrazolol1,5-apyrimidine-3-carboxamide (Compound A), or a pharmaceutically acceptable salt thereof, to said subject.
Absstract of: US20260240872A1
The present invention provides a pharmaceutical composition comprising metformin and glibenclamide for use in the treatment of Parkinson's disease. The invention also comprises a combined administration of metformin and glibenclamide. In a preferred embodiment, the administration is made through oral route.
Absstract of: US20260242469A1
0000 Provided herein is an anti-CD2 antibody or antigen binding fragment thereof for treating and/or preventing ALS in a subject in need thereof.
Absstract of: WO2026174298A1
Provided here are methods for treating or reducing the symptoms of insomnia, and/or cognitive disease or disorder, for example Alzheimer's disease or mild cognitive impairment (MCI) in a subject in need thereof comprising administering to the subject an effective amount of one or more activators of norepinephrine (NE) signaling. Also provided here are compositions comprising one or more activators of norepinephrine (NE) signaling.
Absstract of: WO2026172356A1
The present disclosure relates to therapies for neurodegeneration and, in particular, the use of phosphoinositide 3-kinase (PI3K) inhibitors for treating alpha-synuclein (alpha-Syn)-related pathologies, including Parkinson's disease and related synucleinopathies. The disclosure further provides methods and uses of PI3K inhibitors that cross the blood-brain barrier (BBB) for treating synucleinopathies.
Absstract of: WO2026174035A1
Disclosed are N-terminal modified peptides, and their use in the treatment of diseases such as diabetes, obesity, Alzheimer's disease, liver disease, substance addiction, traumatic brain injury, chronic kidney disease, inflammation and cardiovascular diseases.
Absstract of: WO2026171281A1
Provided are MAPT-targeting siRNAs for preventing or treating neurological diseases or conditions, such as those associated with abnormal MAPT expression or tauopathies, and a range of neurodegenerative disorders including Alzheimer's disease.
Nº publicación: WO2026170996A1 20/08/2026
Applicant:
UNIV EAST CHINA NORMAL [CN]
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Absstract of: WO2026170996A1
Disclosed in the present invention are a lysosome-targeting protein degradation chimera CytoTAC and the use thereof. The CytoTAC consists of three functional protein domains, comprising: a lysosome-targeting domain, a target protein-targeting domain and a linker. The CytoTAC has a degradation rate of target proteins as high as about 80% and has the flexibility of multi-target design. By means of tandem design, the CytoTAC can simultaneously degrade 2-3 target proteins. In terms of the use, the CytoTAC exhibits a significant therapeutic potential against diseases comprising tumors and Alzheimer's disease. The present invention innovatively combines high specificity, rapid degradation and modular design, and breaks through the limitations of traditional protein degradation technology. The CytoTAC has significant application value in the fields of cell-targeted therapy, disease mechanism research, genetic engineering and the like, and provides a novel technical solution for disease treatment.