Resumen de: 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.
Resumen de: 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.
Resumen de: 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).
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: US20260242796A1
Provided are oligonucleotides that increase the expression of glucocerebrosidase (GBA) in cells; conjugates, salts and pharmaceutical compositions thereof; and methods for treatment of diseases associated with reduced expression of GBA, including Gaucher's disease and/or Parkinson's disease. The oligonucleotides may comprise a contiguous sequence complementary to contiguous bases in the 3′ untranslated region (UTR) of the GBA mRNA transcript.
Resumen de: 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.
Resumen de: 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.
Resumen de: US20260240880A1
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: US20260242463A1
The invention provides antibodies or binding fragments thereof directed against citrulline-containing epitopes for use in treating or preventing diseases associated with extracellular trap release from cells, such as Neutrophil Extracellular Trap (NET)-associated pathologies (NET associated pathology) or Eosinophil Extracellular Trap (EET)-associated pathologies (EET-associated pathology), wherein the methods comprising administering at least one dose of the antibody at a specific concentration. The invention also provides the methods themselves. The NET-associated pathologies include systemic lupus erythematosus (SLE), lupus, sepsis, vasculitis, inflammatory arthritis, rheumatoid arthritis and osteoarthritis, psoriasis, Alzheimer's disease, autoimmune hepatitis, juvenile idiopathic arthritis, myositis (polymyositis and dermatomyositis), Sjögren's disease, Anti-phospholipid Syndrome, Bechet'disease, spondylitis, spondyloarthropathy, multiple system atrophy, Parkinson's disease, Lewy body dementia, asthma, allergic rhinovirus exacerbated asthma, allergic asthma, acute respiratory distress syndrome, cystic fibrosis, fibrosis and idiopathic pulmonary fibrosis, heart failure, atherosclerosis, dry eye disease, uveitis, nongranulomatous uveitis, granulomatous uveitis, dermatitis, atopic dermatitis, COPD, bronchitis, or other NET-associated pathologies such as wound healing in diabetes, cancer, cancer metastasis, and transplant organ health in vivo or ex vivo. The invention also pr
Resumen de: 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.
Resumen de: 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.
Resumen de: US20260240775A1
0000 The present disclosure relates to a pharmaceutical composition useful for the prevention or treatment of diabetes, preservation of beta-cell function, hypertension, hyperlipidemia, obesity, non-alcoholic steatohepatitis, or neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease, which includes a sustained-release microsphere containing semaglutide or a pharmaceutically acceptable salt thereof, a bioavailability enhancer and a biodegradable polymer, so that the pharmaceutical composition do not have a high initial burst of drug, contain a high content of drug relative to the particle size and have a high bioavailability, and thus, can minimize pain and inflammatory response of patient that may occur when administered to the human body.
Resumen de: AU2025206887A1
The present invention is directed to methods and compositions for the administration of sodium pentosan polysulfate and related glycosaminoglycans, particularly by oral administration, particularly in combination with administration of an intestinal penetration agent. The methods and compositions are suitable for treatment of neurodegenerative diseases such as, but not limited to, Alzheimer's disease. Methods and compositions according to the present invention can be used together with other agents suitable for treatment of neurodegenerative diseases such as, but not limited to, Alzheimer's disease.
Resumen de: AU2026210879A1
The present invention includes pharmaceutical composition comprising (2R,2R')-3,3'- disulfanediyl bis(2-acetamidopropanamide)(diNACA) or D3-N-acetyl cysteine amide, or a physiologically acceptable salt thereof, having a deuterium enrichment above the natural abundance of deuterium, and derivatives or solids thereof, and methods of using diNACA to treat eye diseases and other diseases associated with oxidative damage including, e.g., antivenom, beta-thallassemia, cataract, chronic obstructive pulmonary disease, macular degeneration, contrast-induced nephropathy, asthma, lung contusion, methamphetamine- induced oxidative stress, multiple sclerosis, Parkinson's disease, platelet apoptosis, Tardive dyskinesia, Alzheimer disease, HIV-1-associated dementia, mitochondrial diseases, myocardial myopathy, neurodegenerative diseases, pulmonary fibrosis, skin pigmentation, skin in need of rejuventation, antimicrobial infection, Friedreich's ataxia. ug u g
Resumen de: 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.
Resumen de: 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.
Resumen de: AU2026208118A1
Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS). This data, for application number 2019268063, is current as of 2022-01-02 21:00 AEST Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS). This data, for application number 2019268063, is current as of 2022-01-02 21:00 AEST pr This data, for application number 2024202423, is current as of 2026-07-27 09:38 AEST ul
Resumen de: US20260242440A1
0000 The present invention relates to the field of neurodegenerative processes and means to provide protection against the same. In particular, the present invention relates to polypeptides, fusion proteins, and other compounds interacting with the N-terminal domain of transient receptor potential melastatin subfamily member 4 (TRPM4), which are capable of interfering with NMDA receptor mediated neurotoxicity. The present invention also relates to nucleic acids encoding the aforementioned polypeptides or fusion proteins, compositions comprising the same and the use of said polypeptides, fusion proteins, and other compounds in methods for treating or preventing a disease of the human or animal body, for example in a method of treating diseases like Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS), Huntington's disease (HD) or stroke.
Resumen de: US20260240820A1
The present invention pertains to a method for controlling a membrane potential-dependent ion channel (VGSC or the like) through a type I taste receptor present in a nerve cell or the like. In the present invention, it has been found that an Aβ peptide, or a sweet amino acid or an umami substance specifically binds to a type I taste receptor on the surface of a nerve cell to exert an agonist-like or antagonist-like action, thereby amplifying or suppressing a VGSC active current.Moreover, with the binding of an Aβ peptide or the like to a type I taste receptor, the amplification of a VGSC active current occurs, the overactivity of nerve cells causing epileptiform attack occurs, and a large number of substances, which can effectively suppress the amplification of the VGSC active current, among ligand substances that specifically bind to the type I taste receptor, can be found.The present invention provides: a type I taste receptor-specific ligand substance that can control the amplification or suppression of a VGSC active current; and a pharmaceutical composition for preventing or treating various neurodegenerative diseases, such as Alzheimer's disease (AD), due to the amplification of a VGSC active current caused by the binding of an Aβ peptide or the like to a type I taste receptor. Moreover, a method for using, as a target receptor, a type I taste receptor present in a nerve cell or the like to screen a ligand substance for controlling a VGSC or the like in the cell is al
Resumen de: 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.
Resumen de: US20260242352A1
0000 The general field of the present disclosure are novel approaches to the treatment of Alzheimer's and other neurodegenerative disorders using novel therapeutics comprising SHIP1 phosphatase inhibitors. Specifically, the disclosure provides compounds, pharmaceutical compositions and methods of treating and preventing such disorders.
Resumen de: US20260240812A1
0000 Present invention relates to the development of therapeutic compound for the treatment of Amyotrophic lateral sclerosis (ALS). Specifically, the present invention provides a NLRP3 inhibitors or its pharmaceutically acceptable salt or suitable composition useful in the treatment of Amyotrophic lateral sclerosis (ALS) and other related disorders including progressive bulbar palsy, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, Kennedy's disease, and post-polio syndrome. Formula (I)
0000
Nº publicación: EP4791421A1 19/08/2026
Solicitante:
ALPHABETA AB [SE]
Alphabeta AB
Resumen de: WO2025078660A1
An isolated protein comprising (i) a first protein moiety selected from the group of proteins comprising an amino acid sequence having at least 70% identity to residues 113-231 of Bri2 from human (SEQ ID NO: 2); and proteins comprising an amino acid sequence having at least 70% identity to any one of the BRICHOS domains of Bri2 from human (SEQ ID NO: 5), chimpanzee (SEQ ID NO: 6), bovine (SEQ ID NO: 7), pig (SEQ ID NO: 8), mouse (SEQ ID NO: 9) and rat (SEQ ID NO: 10); and and optionally (ii) a second protein or polypeptide moiety, preferably containing at least 50 amino acid residues, wherein the second protein or polypeptide moiety is selected from the group consisting of protein drugs, polypeptide drugs, antibodies and/or neurotrophic factors; wherein the second protein or polypeptide moiety is effective for treatment of Parkinson's Disease; for use as a medicament, in particular for treatment of Parkinson's Disease.