Resumen de: US2025059233A1
Peptides and pharmaceutically acceptable salts thereof and peptide conjugates and pharmaceutically acceptable salts thereof having 5 to 15 amino acids and having an amino acid sequence comprising at least 5 consecutive amino acids from the amino acid sequence LSSTQAQQSX1 (SEQ ID NO:1) for use in treating a subject suffering from or at risk of an inflammatory disease or disorder, schizophrenia or psychosis, a neurodegenerative disease such as Parkinson's disease. Alzheimer's disease, or ALS, a gastrointestinal disease or disorder such as irritable bowel syndrome, inflammatory bowel disease. Crohn's disease, or constipation, pain (e.g., visceral pain), rheumatoid arthritis, migraine, headache, substance abuse (e.g., substance use disorder such as drug use disorder, polysubstance use disorder, alcohol use disorder, or nicotine use disorder, tobacco use disorder), drug addiction, a seizure disorder, major depressive disorder, atypical depression, a major depressive episode (MDE) (e.g., atypical MDE), treatment resistant depression, depression in the presence of a neurodegenerative disease, cognitive impairment, COVID-19 related cognitive impairment and/or depression, ADHD, an autism spectrum disorder, a pervasive developmental disorder, atypical autism, multiple sclerosis, PTSD, a sleep disorder such as insomnia, daytime fatigue, or REM sleep behavior disorder, or for use in improving sleep in a subject.
Resumen de: WO2025038979A1
Material compositions and/or methods useful for the prophylaxis and/or treatment of protein depletion (proteinopenia) are provided, including material compositions that retains native function of a peptide/protein while limiting and/or preventing amyloid formation and/or aggregation of said peptide/protein. Material compositions and formulations for enhancing peptide/protein solubility, stability, circulation time, receptor interaction, brain penetrance, CSF half-life, facilitating peptide/protein synthesis and purification are also provided.
Resumen de: AU2023321711A1
Among other things, the present disclosure provides various oligonucleotide technologies including chirally controlled oligonucleotide compositions and technologies for manufacturing such oligonucleotide compositions. In some embodiments, a method is a method of treatment or prevention of Huntington's Disease in a subject in need thereof; a method of allele-specific knockdown of a mutant Huntingtin transcript in a subject; a method for delaying the onset of and/or reducing the severity of at least one symptom of Huntington's Disease in a subject with Huntington's Disease; a method of reducing the expression, level, amount and/or activity of a mutant Huntingtin gene or a gene product thereof; and/or a method of preparation of a medicament for treatment of Huntington's Disease, wherein the method pertains to the use of an oligonucleotide described herein, administered at a dose described herein. In some embodiments, the present disclosure provides doses, dosages, and formulations of an oligonucleotide described herein.
Resumen de: AU2023301953A1
Provided in the present invention are an α,β-unsaturated amide compound, and a preparation method therefor, and a pharmaceutical composition and the use thereof. Specifically, provided in the present invention is a compound as represented by formula I, wherein the definition of each group is as described in the description. The compound can be used as a compound for improving cerebral blood flow and is used for preparing a pharmaceutical composition for treating neurodegenerative diseases such as Alzheimer's disease and vascular dementia and strokes.
Resumen de: US2025059518A1
The compositions contain a novel aldehyde dehydrogenase derived from a novel mutant yeast, that improve memory and cognitive function. The embodiments relate to a food and pharmaceutical composition for preventing Alzheimer's disease and Huntington disease by reducing the accumulation of lesional proteins in brain tissue. The food or pharmaceutical compositions contain a lysate of any one or a mixture thereof selected from Saccharomyces cerevisiae, KCTC13925BP, KCTC14122BP, KCTC14123BP, KCTC14983BP, KCTC14984BP and KCTC14985BP.
Resumen de: WO2025038296A2
Pyrvinium compounds, or a pharmaceutically acceptable salt thereof, used in methods for reducing tauopathy in a subject in need thereof. Pyrvinium compounds, or a pharmaceutically acceptable salt thereof, used in methods for treating a disease associated with accumulation of misfolded proteins (e.g., proteinopathies, Alzheimer's disease, frontotemporal dementia, Parkinson's disease, retinitis pigmentosa such as retinitis pigmentosa with rhodopsin mutations, aging, neurodegenerative diseases, or metabolic disorders).
Resumen de: WO2025036214A1
The present invention belongs to the field of gene therapy, and in particular relates to a gene therapy for treating neuropathy and a use thereof. The present invention provides a gene therapy, said gene therapy involving a recombinant nucleic acid molecule encoding human glucocerebrosidase (GCase) and a neurotrophic factor. The present invention further provides a recombinant vector comprising the recombinant nucleic acid molecule, a recombinant adeno-associated virus, a host cell and a pharmaceutical composition, which are used to treat neuropathy caused by reduced GCase activity, such as Parkinson's disease and type II and type III Gaucher's disease. It can effectively increase GCase activity and the anti-damage and anti-apoptosis abilities of neurons in a subject by means of systemic injection or local injection.
Resumen de: US2025057796A1
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: US2025059541A1
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SNCA gene, particularly in a CNS tissue, as well as methods of inhibiting expression of a SNCA gene and methods of treating subjects having a SNCA-associated neurodegenerative disease or disorder, e.g., Parkinson's Disease (PD), multiple system atrophy (MSA), Lewy body dementia (LBD), among other synucleinopathies, using such dsRNAi agents and compositions.
Resumen de: EP4509504A1
The present invention aims to provide a medicament capable of treating and/or preventing diseases associated with oxidative stress by inhibiting the protein-protein interaction between Keapl and Nrf2 and activating Nrf2. The present invention relates to a compound represented by the following formula (1):wherein each symbol is as described in the DESCRIPTION,or a pharmaceutically acceptable salt thereof. In addition, the present invention also relates to a medicament containing the aforementioned compound, for the prophylaxis and/or treatment of diseases involving oxidative stress selected from the group consisting of chronic kidney disease, non-alcoholic steatohepatitis, chronic obstructive pulmonary disease, radiation skin disorder, radiation mucosal disorder, cardiac failure, pulmonary arterial hypertension, Parkinson's disease, Friedreich's ataxia, multiple sclerosis, age-related macular degeneration, retinitis pigmentosa and glaucoma.
Resumen de: WO2023201182A1
The invention relates generally to the treatment of Parkinson's Disease (PD), including Parkinson's Disease psychosis (PDP) with iloperidone.
Resumen de: WO2023201133A1
Provided are engineered cells that include a T cell receptor (TCR) or antigen-binding fragment thereof that binds to amyloid beta, and methods of engineering and using such cells, such as in methods of treatment, diagnosis, and monitoring of therapeutic effectiveness, of diseases or conditions, such as those associated with amyloid beta, e.g., Alzheimer's Disease.
Resumen de: US2025051750A1
Provided are an aromatic L-amino acid decarboxylase (AADC)/glial cell line-derived neurotrophic factor (GDNF) polynucleotide, and a use thereof in treating Parkinson's disease. Specifically, provided are a method for and a use in treating neurodegenerative diseases (such as Parkinson's disease) by delivering AADC and a GDNF to specific areas of the brain by a gene delivery system using AAV as a vector.
Resumen de: US2025051427A1
Disclosed herein are antibodies, pharmaceutical formulations for treating or preventing Alzheimer's disease, methods of treating or preventing Alzheimer's disease, and kits comprising pharmaceutical formulations for treating or preventing Alzheimer's Disease comprising an anti-Aβ protofibril antibody and anti-tau antibody.
Resumen de: US2025051345A1
The present disclosure provides for a compound according to formula (I) or a pharmaceutically acceptable salt thereof as Cav1.2 activators for the treatment of schizophrenia, bipolar disorder, major depressive disorder, substance use disorder. ADHD, Phelan-Mc-Dermid Syndrome, autism spectrum disorder, multiple sclerosis, frontotemporal dementia, Alzheimer's disease. Brugada Syndrome. Short QT syndrome, or early repolarization syndrome.
Resumen de: AU2023303333A1
The present invention relates to a pharmaceutical composition that contains sustained-release microspheres composed of semaglutide or a pharmacologically acceptable salt thereof, a bioavailability enhancer, and a biodegradable polymer and which is designed to avoid rapid initial drug release (initial burst), contain a high concentration of the drug relative to particle size, have high bioavailability, and minimize patient discomfort and inflammatory reactions when administered to the human body, whereby the composition is useful in the prevention or treatment of diabetes, hypertension, hyperlipidemia, obesity, non-alcoholic steatohepatitis, or degenerative neurological diseases such as Alzheimer's and Parkinson's disease and in the preservation of beta-cell functions.
Resumen de: AU2023319981A1
The invention provides methods of treating patients with Parkinson's disease (PD) associated with wild-type LRRK2. The invention recognizes that analysis of biomarkers in such patients allows identification of those patients who will respond to LRRK2 inhibitors. Thus, the invention provides methods of identifying PD patients who will respond to LRRK2 inhibitors and methods of treating such patients.
Resumen de: WO2025030709A1
Disclosed in the present invention is a β-carboline compound having a structure as represented by formula (I) or a pharmaceutically acceptable salt thereof, wherein R1 is halogen; X is O or NH; n is 1 or 2; ring A is 3- to 6-membered cycloalkyl, 4- to 6-membered heterocyclyl, aryl or heteroaryl; and R2 is H, halogen, cyano, nitro, C1-C3 alkyl, C2-C3 alkenyl or C2-C3 alkynyl. The compound or the salt thereof of the present invention has a relatively high selective inhibitory effect on MAO-B, has a significantly higher in-vitro MAO-B inhibitory activity and selectivity than safinamide, and has a reversible inhibitory effect on MAO-B. The compound or the salt thereof of the present invention can be used for treating neurodegenerative diseases such as Parkinson's disease and Alzheimer's disease. Further disclosed in the present invention is the use of the β-carboline compound or the pharmaceutically acceptable salt thereof in the preparation of an MAO-B inhibitor or in the preparation of a drug for treating and/or preventing diseases regulated by a monoamine oxidase B inhibitor.
Resumen de: WO2025030991A1
The present application relates to Bifidobacterium longum subsp. infantis capable of relieving Parkinson's disease and the use thereof. Said strain is named as Bifidobacterium longum subsp. infantis BI03 strain, with an accession number of CGMCC No. 24473, the preservation date thereof being March 07, 2022. Said strain can significantly relieve the symptoms of Parkinson's disease, specifically embodied in: relieving Parkinson's disease related dyskinesia and increased corticosterone levels; alleviating neuroinflammation related to Parkinson's disease; promoting glutathione and reducing brain oxidative stress injuries, etc.
Resumen de: US2025051766A1
Disclosed herein are UNC13A oligonucleotides with one or more spacers or without a spacer. In various embodiments, UNC13A oligonucleotides with spacer(s) reduce mis-spliced UNC13A transcripts and increase full length UNC13A transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).
Resumen de: US2025049741A1
The present invention relates to a composition for preventing or treating Alzheimer's disease, comprising a phospholipase C (PLC) activator as an active ingredient. A composition comprising the PLC activator of the present invention as an active ingredient restores the S-eCB mobilization suppressed by AβO, recovers the synaptic plasticity impaired by AβO, and not only recovers PLCβ1 protein levels to normal levels in AβO-treated mouse hippocampal slices and 5XFAD mouse hippocampal slices in the chronic stage of AD, but also recovers contextual fear memory impairment in AD mice, and thus is expected to be usefully used for preventing or treating Alzheimer's disease.
Resumen de: US2025049740A1
The present disclosure relates to a compound and the use thereof in the preparation of a medicament for treating Alzheimer's disease. The present disclosure provides a method for constructing an animal model of diseases with N-oleoyl-dopamine (OLDA) and the use of the model in drug screening and animal experimental research, wherein the animal model includes at least one of an animal model of neurodegenerative diseases, an animal model of intestinal permeability diseases, and an animal model of inflammatory diseases. The present disclosure is based on the discovery that OLDA has a new use for a neurodegenerative disease, an intestinal permeability disease, and an inflammatory disease, and based on OLDA, compounds that have a therapeutic effect on the above diseases are identified.
Resumen de: US2025051264A1
Disclosed in the present invention are a cannabidiol derivative, a preparation method therefor and an application thereof, particularly an application in preventing and treating nervous system diseases (such as Parkinson's disease). The cannabidiol derivative is obtained by screening from a series of synthetic derivatives. An animal testing result shows that the cannabidiol derivative can reduce the balance beam score of a Parkinson's model animal and improve the dopamine level and the tyrosine hydroxylase (TH) cell positive rate in substantia nigra (SubN), can be used for drug development and research of various diseases such as epilepsy and Parkinson's disease, and has better application value.
Resumen de: AU2023241739A1
The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease.
Nº publicación: EP4506011A1 12/02/2025
Solicitante:
SAISEIKEN CO LTD [JP]
Saiseiken Co., Ltd
Resumen de: EP4506011A1
This application provides compositions for the treatment of intractable neurological diseases and methods for their production, enabling the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer's disease. The composition for treating intractable neurological diseases is characterized in that it contains culture supernatant obtained by culturing deciduous dental pulp stem cells in serum-free medium under ultrasound irradiation. A pharmaceutical product for the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer's disease, characterized in that it contains the composition for the treatment of intractable neurological diseases. A method for producing a composition for treating intractable neurological diseases, characterized in that the composition contains a culture supernatant obtained by culturing deciduous dental pulp stem cells under ultrasound irradiation using serum-free medium. The method of manufacturing the composition for the treatment of intractable neurological diseases.